0001178879-23-000004 | 10-K | XBRL Viewer

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Cover - USD ($)	12 Months Ended
Cover - USD ($)	Dec. 31, 2022	Feb. 13, 2023	Jun. 30, 2022
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2022
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-33497
Entity Registrant Name	Amicus Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	71-0869350
Entity Address, Address Line One	3675 Market Street,
Entity Address, City or Town	Philadelphia,
Entity Address, State or Province	PA
Entity Address, Postal Zip Code	19104
City Area Code	(215)
Local Phone Number	921-7600
Title of 12(b) Security	Common Stock, par value $0.01 per share
Trading Symbol	FOLD
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Entity Shell Company	false
Entity Public Float			$ 2,979,823,386
Entity Common Stock, Shares Outstanding		282,714,738
Documents Incorporated by Reference	Portions of the Proxy Statement for the registrant's 2022 Annual Meeting of Stockholders which is to be filed subsequent to the date hereof are incorporated by reference into Part III of this Annual Report on Form 10-K.
Entity Central Index Key	0001178879
Amendment Flag	false
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	FY

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2022
Audit Information [Abstract]
Auditor Firm ID	42
Auditor Name	Ernst & Young LLP
Auditor Location	Iselin, New Jersey

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 148,813	$ 245,197
Investments in marketable securities	144,782	237,299
Accounts receivable	66,196	52,672
Inventories	23,816	26,818
Prepaid expenses and other current assets	40,209	34,848
Total current assets	423,816	596,834
Operating lease right-of-use assets, net	29,534	20,586
Property and equipment, less accumulated depreciation of $22,281 and $19,882 at December 31, 2022 and 2021, respectively	30,778	42,496
In-process research & development	23,000	23,000
Goodwill	197,797	197,797
Other non-current assets	19,242	24,427
Total Assets	724,167	905,140
Current liabilities:
Accounts payable	15,413	21,513
Accrued expenses and other current liabilities	93,636	98,153
Contingent consideration payable	21,417	18,900
Operating lease liabilities	8,552	7,409
Total current liabilities	139,018	145,975
Long-term debt	391,990	389,357
Operating lease liabilities	51,578	43,363
Deferred income taxes	4,939	4,930
Deferred reimbursements	4,656	5,906
Other non-current liabilities	8,939	8,240
Total liabilities	601,120	597,771
Commitments and contingencies
Stockholders' equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 281,108,273 and 278,912,800 shares issued and outstanding at December 31, 2022 and 2021, respectively	2,815	2,808
Additional paid-in capital	2,664,744	2,595,419
Accumulated other comprehensive (loss) gain:
Foreign currency translation adjustment	(11,989)	5,251
Unrealized loss on available-for-sale securities	(116)	(270)
Warrants	83	83
Accumulated deficit	(2,532,490)	(2,295,922)
Total stockholders' equity	123,047	307,369
Total Liabilities and Stockholders' Equity	$ 724,167	$ 905,140

Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Statement of Financial Position [Abstract]
Accumulated depreciation of property and equipment	$ 22,281	$ 19,882
Common stock, par value (in dollars per share)	$ 0.01	$ 0.01
Common stock, shares authorized (in shares)	500,000,000	500,000,000
Common stock, shares issued (in shares)	281,108,273	278,912,800
Common stock, shares outstanding (in shares)	281,108,273	278,912,800

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Income Statement [Abstract]
Net product sales	$ 329,233	$ 305,514	$ 260,886
Cost of goods sold	38,599	34,466	31,044
Gross profit	290,634	271,048	229,842
Operating expenses:
Research and development	276,677	272,049	308,443
Selling, general, and administrative	213,041	192,710	156,407
Changes in fair value of contingent consideration payable	1,078	6,514	3,144
Loss on impairment of assets	6,616	0	0
Depreciation and amortization	5,342	6,209	8,846
Total operating expenses	502,754	477,482	476,840
Loss from operations	(212,120)	(206,434)	(246,998)
Other income (expense):
Interest income	3,024	509	3,226
Interest expense	(37,119)	(32,471)	(22,425)
Loss on extinguishment of debt	0	(257)	(7,276)
Other income (expense)	4,176	(2,901)	(781)
Loss before income tax	(242,039)	(241,554)	(274,254)
Income tax benefit (expense)	5,471	(8,906)	(2,598)
Net loss attributable to common stockholders	$ (236,568)	$ (250,460)	$ (276,852)
Net loss attributable to common stockholders per common share - basic (in dollars per share)	$ (0.82)	$ (0.92)	$ (1.07)
Net loss attributable to common stockholders per common share - diluted (in dollars per share)	$ (0.82)	$ (0.92)	$ (1.07)
Weighted-average common shares outstanding - basic (in shares)	289,057,198	271,421,986	258,867,380
Weighted-average common shares outstanding - diluted (in shares)	289,057,198	271,421,986	258,867,380

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Statement of Comprehensive Income [Abstract]
Net loss	$ (236,568)	$ (250,460)	$ (276,852)
Other comprehensive (loss) gain:
Foreign currency translation adjustment (loss) gain	(17,240)	(3,161)	5,627
Unrealized gain (loss) on available-for-sale securities	154	(85)	(225)
Other comprehensive (loss) gain	(17,086)	(3,246)	5,402
Comprehensive loss	$ (253,654)	$ (253,706)	$ (271,450)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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+ Details

Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-In Capital	Warrants	Other Comprehensive Gain (Loss)	Accumulated Deficit
Balance (in shares) at Dec. 31, 2019		255,417,869
Balance at Dec. 31, 2019	$ 476,425	$ 2,598	$ 2,227,225	$ 12,387	$ 2,825	$ (1,768,610)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	5,243,000	5,233,672
Stock options exercised, net	$ 42,282	$ 52	42,230
Vesting of restricted stock units, net of taxes (in shares)		1,411,920
Vesting of restricted stock units, net of taxes	(10,028)		(10,028)
Share-based compensation	49,151		49,151
Unrealized gain (loss) on available-for-sale securities	(225)				(225)
Foreign currency translation adjustment	5,627				5,627
Net loss	(276,852)					(276,852)
Balance (in shares) at Dec. 31, 2020		262,063,461
Balance at Dec. 31, 2020	$ 286,380	$ 2,650	2,308,578	12,387	8,227	(2,045,462)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	1,483,000	1,461,189
Stock options exercised, net	$ 10,228	$ 15	10,213
Common stock issued from equity financing and pre-funded warrants (in shares)		11,296,660
Common stock issued from equity financing and pre-funded warrants	199,747	$ 112	199,552	83
Vesting of restricted stock units, net of taxes (in shares)		1,064,135
Vesting of restricted stock units, net of taxes	(15,009)		(15,009)
Share-based compensation	57,838		57,838
Warrants exercised (in shares)		2,554,999
Warrants exercised	19,230	$ 26	31,591	(12,387)
Equity component of the convertible notes	2,661	$ 5	2,656
Unrealized gain (loss) on available-for-sale securities	(85)				(85)
Foreign currency translation adjustment	(3,161)				(3,161)
Net loss	$ (250,460)					(250,460)
Balance (in shares) at Dec. 31, 2021	278,912,800	278,912,800
Balance at Dec. 31, 2021	$ 307,369	$ 2,808	2,595,419	83	4,981	(2,295,922)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	660,000	656,377
Stock options exercised, net	$ 4,310	$ 7	4,303
Vesting of restricted stock units, net of taxes (in shares)		1,539,096
Vesting of restricted stock units, net of taxes	(11,490)		(11,490)
Share-based compensation	76,512		76,512
Unrealized gain (loss) on available-for-sale securities	154				154
Foreign currency translation adjustment	(17,240)				(17,240)
Net loss	$ (236,568)					(236,568)
Balance (in shares) at Dec. 31, 2022	281,108,273	281,108,273
Balance at Dec. 31, 2022	$ 123,047	$ 2,815	$ 2,664,744	$ 83	$ (12,105)	$ (2,532,490)

Consolidated Statements of Cash Flows - USD ($)	12 Months Ended
Consolidated Statements of Cash Flows - USD ($)	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Operating activities
Net loss	$ (236,568,000)	$ (250,460,000)	$ (276,852,000)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing	2,634,000	2,490,000	1,794,000
Depreciation and amortization	5,342,000	6,209,000	8,846,000
Share-based compensation	76,512,000	57,838,000	49,151,000
Loss on extinguishment of debt	0	257,000	7,276,000
Non-cash changes in the fair value of contingent consideration payable	1,078,000	6,514,000	3,144,000
Foreign currency remeasurement loss	6,121,000	3,565,000	5,471,000
Non-cash deferred taxes	9,000	34,000	(155,000)
Asset impairment charges and other asset write-offs	18,177,000	0	99,000
Changes in operating assets and liabilities:
Accounts receivable	(17,330,000)	(8,189,000)	(11,219,000)
Inventories	(5,343,000)	(7,790,000)	(4,639,000)
Prepaid expenses and other current assets	(6,194,000)	(5,919,000)	(8,766,000)
Accounts payable, accrued expenses, and other current liabilities	(6,377,000)	7,430,000	(10,610,000)
Other non-current assets and liabilities	(4,636,000)	(4,117,000)	3,170,000
Payment of contingent consideration	0	(10,353,000)	0
Net cash used in operating activities	(166,575,000)	(202,491,000)	(233,290,000)
Investing activities
Sale and redemption of marketable securities	335,926,000	424,043,000	354,826,000
Purchases of marketable securities	(243,255,000)	(341,398,000)	(365,178,000)
Capital expenditures	(3,766,000)	(3,884,000)	(3,227,000)
Proceeds from sale of assets	3,411,000	0	0
Net cash provided by (used in) investing activities	92,316,000	78,761,000	(13,579,000)
Financing activities
Proceeds from issuance of common stock from equity financing and pre-funded warrants, net of issuance costs	0	199,750,000	0
Payment of long-term debt	0	0	(155,249,000)
Proceeds from long-term debt, net of issuance costs	0	0	385,929,000
Payment of finance leases	(283,000)	(479,000)	(76,000)
Purchase of vested restricted stock units, net of taxes	(11,490,000)	(15,009,000)	(10,028,000)
Proceeds from stock options exercised, net	4,310,000	10,228,000	42,282,000
Proceeds from warrants exercised, net	0	19,230,000	0
Payment of contingent consideration	0	(1,647,000)	0
Net cash (used in) provided by financing activities	(7,463,000)	212,073,000	262,858,000
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	(14,619,000)	(5,049,000)	3,830,000
Net increase in cash, cash equivalents, and restricted cash	(96,341,000)	83,294,000	19,819,000
Cash, cash equivalents, and restricted cash at the beginning of the year	249,456,000	166,162,000	146,343,000
Cash, cash equivalents, and restricted cash at the end of the year	153,115,000	249,456,000	166,162,000
Supplemental disclosures of cash flow information
Tenant improvements paid through lease incentive	0	300,000	470,000
Cash paid during the period for interest	34,358,000	30,468,000	24,683,000
Capital expenditures unpaid at the end of period	1,141,000	1,448,000	985,000
Cash paid for income taxes	$ 1,609,000	$ 20,032,000	$ 10,371,000

Description of Business	12 Months Ended
Description of Business	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Description of Business	Description of Business Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company has a portfolio including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease, that is under review with the U.S. Food and Drug Administration ("FDA"), European Medicines Agency ("EMA"), and the United Kingdom ("U.K.") Medicines and Healthcare products Regulatory Agency ("MHRA"). The Company is committed to discovering and developing next generation therapies in Fabry and Pompe diseases. The cornerstone of the Company's portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the United States ("U.S."), European Union ("E.U."), U.K., and Japan, with multiple additional approvals granted and applications pending in several geographies around the world. The lead biologics program of the Company's pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, two-component, potential best-in-class treatment for Pompe disease. In February 2019, the FDA granted Breakthrough Therapy designation ("BTD") to AT-GAA for the treatment of late onset Pompe disease. In September 2021, the FDA set the Prescription Drug User Fee Act ("PDUFA") target action date of May 29, 2022 for the New Drug Application ("NDA") for miglustat and July 29, 2022 for the Biologics License Application ("BLA") for cipaglucosidase alfa. The EMA validated the Marketing Authorization Application (“MAA”) in the fourth quarter of 2021. In May 2022, the FDA extended the review period for the NDA for miglustat and the BLA for cipaglucosidase alfa resulting in revised PDUFA action dates of August 29, 2022 and October 29, 2022, respectively. In October 2022, the FDA deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing facility inspection prior to the PDUFA action date. The Company is actively engaged with the FDA and an inspection has been scheduled. In December 2022, the Committee for Medicinal Products for Human Use ("CHMP") of the EMA adopted a positive opinion recommending market authorization of cipaglucosidase alfa, or Pombiliti™. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022. The Company continues to monitor the novel coronavirus (“COVID-19”) pandemic. The Company's commercial operations have not been significantly impacted by the COVID-19 pandemic and the Company gradually continues to see an improvement in patient identification and Galafold® initiation. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, as well as maintained the operational integrity of its clinical trials, with minimum disruptions. The Company has been able to continue to meet required commercial demand for Galafold® as well as supply its ongoing Pompe disease clinical studies and access programs including the Early Access to Medicines Scheme ("EAMS") without interruption. In regard to the Company’s regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a facility inspection was necessary, however, could not be completed by the PDUFA action date due to COVID-19 related travel restrictions. The facility inspection has subsequently been scheduled. Per FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval. The Company had an accumulated deficit of $2.5 billion as of December 31, 2022 and anticipates incurring losses through the fiscal year ending December 31, 2023. The Company has historically funded its operations through stock offerings, Galafold® revenues, debt issuances, collaborations, and other financing arrangements. Based on its current operating model, the Company believes that the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company's future capital requirements.

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Basis of Presentation

The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented.

Consolidation

The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.

Foreign Currency Transactions

The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of December 31, 2022 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive (loss) gain in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.

Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other current assets and other non-current assets on the Company's Consolidated Balance Sheets.

Concentration of Credit Risk

The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.

The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company's accounts receivable at December 31, 2022 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of December 31, 2022, the Company's allowance for doubtful accounts was $0.2 million.

Property and Equipment

Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three to five years, or the lesser of the related initial term of the lease or useful life for leasehold improvements.

The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are charged to income in the period in which the costs are incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy.

The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the long-lived asset over its estimated fair value in the period at which such a determination is made.

Revenue Recognition

The Company’s net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the years ended December 31, 2022, 2021 and 2020 were $329.0 million, $305.5 million and $260.9 million, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2022, one customer accounted for 27% of net product sales and 14% of accounts receivable from product sales. In 2021, one customer accounted for 24% of net product sales and 14% of accounts receivable from product sales.

The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.

The following table summarizes the Company's net product sales disaggregated by geographic area:


	For the Year
(in thousands)	2022		2021		2020
U.S.	$	115,946	$	95,387	$	80,046
Ex-U.S.	213,287		210,127		180,840
Total net product sales	$	329,233	$	305,514	$	260,886

Inventories and Cost of Goods Sold

Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Consolidated Statements of Operations.

Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable.

Fair Value Measurements

The Company records certain asset and liability balances under the fair value measurements as defined by the Financial Accounting Standard Board ("FASB") guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participant's assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy).

Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability.

Contingent Liabilities

On an ongoing basis, the Company may be involved in various claims and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company's best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in material adverse adjustments to the Company's operating results.

Research and Development Costs

Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development.

Interest Income and Interest Expense

Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases.

Income Taxes

The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized.

The Company’s tax returns are subject to examination by U.S. Federal, state, and foreign taxing jurisdictions. The impact of an uncertain tax position taken or expected to be taken on an income tax return must be recognized in the financial statements at the largest amount that is more likely than not to be sustained. An uncertain income tax position will not be recognized in the financial statements unless it is more likely than not to be sustained.

Other Comprehensive (Loss) Gain

Components of other comprehensive (loss) gain include unrealized gains and losses on available-for-sale securities and (loss) gain on foreign currency transactions and are included in the Consolidated Statements of Comprehensive Loss.

Leases

The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets.

ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments.

Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories.

The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor.

Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.

Nonqualified Cash Deferral Plan

The Company's Cash Deferral Plan (the "Deferral Plan"), provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code. All of the investments held in the Deferral Plan are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets.

Equity-based Compensation

At December 31, 2022, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 8. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award.

Loss per Common Share

The Company calculates net loss per share as a measurement of the Company's performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company had a net loss for all periods presented; accordingly, the inclusion of common stock options, unvested restricted stock units, and certain warrants would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same. See "— Note 15. Basic and Diluted Net Loss per Common Share" for further discussion on net loss per share.

Segment Information

The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker who comprehensively manages the entire business. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reporting unit.

Business Combinations

The Company assigns fair value to the tangible and intangible assets acquired and liabilities assumed based upon their estimated fair values on the acquisition date from acquired businesses. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and in-process research and development ("IPR&D"). In connection with the purchase price allocations for acquisitions, the Company estimates the fair value of contingent payments utilizing a probability-based income approach inclusive of an estimated discount rate.

Contingent Consideration Payable

Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable. This does not apply in circumstances when the contingent consideration meets the definition of a derivative, in which case the amount becomes part of the basis in the asset acquired. Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets. For contingent consideration payments in business combinations, the Company determines the fair value of contingent acquisition consideration payable on the acquisition date using a probability-based income approach utilizing an appropriate discount rate. The short term and long term portions of the contingent consideration payable is shown on the Company's Consolidated Balance Sheets. The fair value of the contingent consideration payable will be determined each period end and the resulting change will be recorded on the Consolidated Statements of Operations.

Intangible Assets and Goodwill

The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Purchased IPR&D is accounted for as an indefinite lived intangible asset until the underlying project is completed, at which point the intangible asset will be accounted for as a definite lived intangible asset, or abandoned, at which point the intangible asset will be written off or partially impaired. Goodwill and indefinite lived intangible assets are assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill or IPR&D impairment test is conducted. If it is determined the full carrying amount of IPR&D is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference. No indicators of impairment were noted during the years ended December 31, 2022 and 2021.

Recent Accounting Developments

The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

Goodwill and Intangible Assets

12 Months Ended

Dec. 31, 2022

Goodwill and Intangible Assets Disclosure [Abstract]

Goodwill and Intangible Assets

As of December 31, 2022, in connection with the acquisitions, the Company had goodwill of $197.8 million and IPR&D of $23.0 million, which are reported at fair value on the Company's Consolidated Balance Sheets. Intangible assets related to IPR&D assets are considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts. During the period the assets are considered indefinite-lived, they will not be amortized but will be tested for impairment on an annual basis and between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate a reduction in the fair value of the IPR&D assets below their respective carrying amounts.

Goodwill and intangible assets are assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. A goodwill impairment loss, if any, is measured as the amount by which the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value. An IPR&D impairment loss, if any, is measured as the amount by which the carrying amount of the asset exceeds its fair value.

The following table represents the changes in IPR&D for the years ended December 31, 2022 and 2021, respectively:


	(in millions)
Balance at December 31, 2020	$	23.0
Change in IPR&D	—
Balance at December 31, 2021	$	23.0
Change in IPR&D	—
Balance at December 31, 2022	$	23.0

The following table represents the changes in Goodwill for the years ended December 31, 2022 and 2021, respectively:


	(in millions)
Balance at December 31, 2020	$	197.8
Change in goodwill	—
Balance at December 31, 2021	$	197.8
Change in goodwill	—
Balance at December 31, 2022	$	197.8

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

12 Months Ended

Dec. 31, 2022

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

As of December 31, 2022, the Company held $148.8 million in cash and cash equivalents and $144.8 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive (loss) gain in the Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss, such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss the Company would recognize an allowance and the corresponding credit loss would be included in earnings.

The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as many of these securities are either government backed or of the highest credit rating. Investments that have original maturities greater than three months but less than one year are classified as current.

Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:


	As of December 31, 2022
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Commercial paper	144,299		82		—		144,381
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	293,513	$	82	$	—	$	293,595
Included in cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Included in marketable securities	144,700		82		—		144,782
Total cash, cash equivalents, and marketable securities	$	293,513	$	82	$	—	$	293,595


	As of December 31, 2021
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Commercial paper	174,578		7		(54)		174,531
Corporate debt securities	32,322		—		(11)		32,311
Asset-backed securities	30,070		—		(14)		30,056
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	482,568	$	7	$	(79)	$	482,496
Included in cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Included in marketable securities	237,371		7		(79)		237,299
Total cash, cash equivalents, and marketable securities	$	482,568	$	7	$	(79)	$	482,496

For both the years ended December 31, 2022 and 2021, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.

The Company had no marketable securities in an unrealized loss position as of December 31, 2022. Unrealized loss positions in the marketable securities as of December 31, 2021 reflect temporary impairments and were not a result of credit loss. Additionally, as these positions were in a loss position for less than twelve months and the Company did not intend to sell these securities before recovery, the losses were recognized in other comprehensive (loss) gain. The fair value of these marketable securities in unrealized loss positions was $173.4 million as of December 31, 2021.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows.


(in thousands)	December 31, 2022		December 31, 2021		December 31, 2020
Cash and cash equivalents	$	148,813	$	245,197	$	163,240
Restricted cash	4,302		4,259		2,922
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	153,115	$	249,456	$	166,162

Inventories

12 Months Ended

Dec. 31, 2022

Inventory Disclosure [Abstract]

Inventories

Inventories consist of raw materials, work in process, and finished goods related to the manufacture of Galafold®. The following table summarizes the components of inventories:


(in thousands)	December 31, 2022		December 31, 2021
Raw materials	$	10,054	$	12,289
Work-in-process	9,615		10,699
Finished goods	4,147		3,830
Total inventories	$	23,816	$	26,818

The Company's reserve for inventory was $0.4 million and $1.1 million as of December 31, 2022 and 2021, respectively.

Property and Equipment

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

Property and Equipment

Property and equipment consist of the following:


	December 31,
(in thousands)	2022		2021
Leasehold improvements	$	24,162	$	24,168
Research equipment	16,345		16,663
Computer equipment	4,486		5,720
Construction in progress	4,160		8,229
Furniture and fixtures	2,734		3,163
Computer software	1,106		1,174
Vehicles	66		71
Land	—		3,190
Gross property and equipment	53,059		62,378
Less accumulated depreciation	(22,281)		(19,882)
Net property and equipment	$	30,778	$	42,496

Depreciation expense was $4.8 million and $5.7 million for the years ended December 31, 2022 and 2021, respectively. Additionally, during the year ended December 31, 2022, in connection with the strategic prioritization of its gene therapy portfolio, the Company performed an assessment of its fixed assets. As a result, the Company recognized an impairment charge of $6.6 million.

Accrued Expenses and Other Current Liabilities

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

Accrued Expenses and Other Current Liabilities

Accrued expenses and other current liabilities consist of the following:


	December 31,
(in thousands)	2022		2021
Accrued compensation and benefits	$	25,701	$	24,258
Accrued sales rebates and discounts	21,886		15,989
Accrued program fees	10,515		13,294
Accrued contract manufacturing & contract research costs	8,230		20,361
Accrued royalties	6,908		4,522
Accrued professional fees	6,868		9,377
Accrued taxes	5,938		6,154
Other	7,590		4,198
	$	93,636	$	98,153

Stockholders' Equity	12 Months Ended
Stockholders' Equity	Dec. 31, 2022
Equity [Abstract]
Stockholders' Equity	Stockholders' Equity Common Stock and Warrants As of December 31, 2022, the Company was authorized to issue 500 million shares of common stock. Dividends on common stock will be paid when, and if, declared by the board of directors. Each holder of common stock is entitled to vote on all matters that are appropriate for stockholder voting and is entitled to one vote for each share held. In November 2022, the Company entered into a Sales Agreement with The Goldman Sachs & Co. LLC to create an at-the-market ("ATM") equity program, pursuant to which the Company may offer to sell shares of its common stock having an aggregate offering gross proceeds of up to $250.0 million. At December 31, 2022, no shares have been issued under the ATM equity program. In September 2021, the Company entered into a securities purchase agreement with certain entities, the (“Purchase Agreements”) for the private placement of an aggregate of 11,296,660 shares of the Company’s common stock, at a purchase price of $10.18 per share, and pre-funded warrants to purchase an aggregate of 8,349,705 shares of common stock, at a purchase price of $10.17 per pre-funded warrant. Proceeds from the private placement, net of offering costs, were $199.8 million. Each pre-funded warrant has an initial exercise price of $0.01 per share and is exercisable at any time after its original issuance, subject generally to the lock-up period, at the option of each holder, in such holder’s discretion, by (i) payment in full in immediately available funds of the initial exercise price for the number of shares of common stock purchased upon such exercise or (ii) a cashless exercise, in which case the holder would receive upon such exercise the net number of shares of common stock determined according to the formula set forth in the pre-funded warrant. Certain of the Purchase Agreements provide for a lock-up period of either 60 days or nine months based on the individual agreements. During the first quarter of 2021, 1,294,999 and 1,260,000 warrants were exercised at $7.98 and $7.06 per share of common stock, respectively, resulting in gross cash proceeds of $19.2 million. During the first and third quarters of 2021, the Company entered into separate, privately negotiated exchange agreements with the holders of the Convertible Notes (the "Holders"). Under the terms of the exchange agreements, the Holders agreed to exchange the remaining aggregate principal amount of $2.8 million of Convertible Notes held by them in exchange for an aggregate of approximately 472,356 shares of Company common stock, par value $0.01 per share. This transaction resulted in an increase of $2.7 million and five thousand dollars to additional paid-in-capital and common stock, respectively. Nonqualified Cash Deferral Plan The Company's Deferral Plan provides certain key employees and members of the Board of Directors, as selected by the Compensation Committee, with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code of 1986 as amended. The Company had a deferred compensation investment balance of $5.5 million and $4.8 million as of December 31, 2022 and 2021, respectively, with corresponding approximate amounts of liability. Deferral Plan investment assets are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. Deferred compensation liability amounts under the Deferral Plan are included in other long-term liabilities. Equity Incentive Plan The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. Under the provisions of the Plan, no option will have a term in excess of 10 years. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. Options granted pursuant to the Plan generally vest 25% on the first year anniversary date of grant plus an additional 1/48th for each month thereafter and may be exercised in whole or in part for 100% of the shares vested at any time after the date of grant. As of December 31, 2022, the Company has reserved up to 15,472,672 shares for issuance under the Plan.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

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+ Details

Share based Compensation

12 Months Ended

Dec. 31, 2022

Share-Based Payment Arrangement [Abstract]

Share based Compensation

The Plan provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors. advisors, and consultants at a price to be determined by the Company’s Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company’s business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.

The Plan provides for certain benefits to qualifying Plan participants who separate from service with the Company due to death, disability or "retirement" (as such term is defined under the Plan) ("Qualified Participants"). Options granted under the Plan to a Qualified Participant shall continue to vest until the 2nd anniversary of the Qualified Participant’s separation and all vested options held by such Qualified Participant shall remain exercisable until the earlier of the 4th anniversary of the Qualified Participant’s separation or the original expiration date of the option. Options that are not exercised during this exercise period shall be forfeited. Time-based restricted stock units and restricted stock granted to a Qualified Participant under the Plan that was scheduled to vest within the two year period following the Qualified Participant's separation shall accelerate and be delivered upon such separation. Any time-based restricted stock units or restricted stock that would have vested after such two year period will be forfeited upon the Qualified Participant's separation. Also, per the Amendment, any performance-based restricted stock units under the Plan ("PRSUs") received by the Qualified Participant, shall remain eligible to vest after the Qualified Participant’s separation based on the actual performance of the Company through the end of the performance period applicable to any such PRSUs.

Stock Option Grants

The Company uses the fair value method of measuring share-based compensation, using the fair value of each equity award granted. The Company chose the ‘‘straight-line’’ attribution method for allocating compensation costs and recognized the fair value of each stock option on a straight-line basis over the vesting period of the related awards.

The Company uses the Black-Scholes option pricing model when estimating the grant date fair value for share-based awards. Use of a valuation model requires management to make certain assumptions with respect to selected model inputs. Expected volatility is based on the Company's historical volatility. The average expected life is determined using the Company's actual historical data. The risk-free interest rate is based on U.S. Treasury, zero-coupon issues with a remaining term equal to the expected life assumed at the date of grant. Forfeitures are estimated based on voluntary termination behavior, as well as a historical analysis of actual option forfeitures.

The fair value of the stock options granted was estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:


	Years Ended December 31,
	2022			2021			2020
Expected stock price volatility	62.1		%	65.4		%	75.1		%
Risk free interest rate	1.7		%	0.6		%	1.6		%
Expected life of options (years)	5.34			5.40			5.67
Expected annual dividend per share	$	—		$	—		$	—

The weighted average grant-date fair value per share of options granted during 2022, 2021, and 2020 were $6.11, $9.08, and $6.40, respectively.

A summary of the Company's stock options for the year ended December 31, 2022 were as follows:


	Number of Shares	Weighted Average Exercise Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Options outstanding, December 31, 2019	16,724	$	9.15
Granted	4,362	$	9.98
Exercised	(5,243)	$	8.11
Forfeited	(1,376)	$	10.42
Expired	(435)	$	13.33
Options outstanding, December 31, 2020	14,032	$	9.54
Granted	3,262	$	16.53
Exercised	(1,483)	$	7.05
Forfeited	(844)	$	12.97
Expired	(236)	$	13.28
Options outstanding, December 31, 2021	14,731	$	11.08
Granted	5,733	$	11.53
Exercised	(660)	$	6.58
Forfeited	(495)	$	12.57
Expired	(245)	$	12.84
Options outstanding, December 31, 2022	19,064	$	11.31	6.6	$	36.7
Vested and unvested expected to vest, December 31, 2022	17,492	$	11.22	6.4	$	35.1
Exercisable at December 31, 2022	10,938	$	10.59	5.0	$	29.0

The aggregate intrinsic value of options exercised during the years ended December 31, 2022, 2021 and 2020 was $2.5 million, $8.5 million, and $40.9 million, respectively. Cash proceeds from stock options exercised during the years ended December 31, 2022, 2021, and 2020 were $4.3 million, $10.2 million, and $42.3 million, respectively. As of December 31, 2022, the total unrecognized compensation cost related to non-vested stock options granted was $31.1 million and is expected to be recognized over a weighted average period of three years.

Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")

RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the year ended December 31, 2022 is as follows:


	Number of Shares	Weighted Average Grant Date Fair Value		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Non-vested units as of December 31, 2019	5,792	$	11.18
Granted	4,692	$	11.29
Vested	(2,164)	$	10.70
Forfeited	(1,240)	$	11.14
Non-vested units as of December 31, 2020	7,080	$	11.35
Granted	3,191	$	16.94
Vested	(1,863)	$	15.77
Forfeited	(1,067)	$	12.82
Non-vested units as of December 31, 2021	7,341	$	13.90
Granted	5,048	$	11.93
Vested	(2,251)	$	12.48
Forfeited	(421)	$	12.06
Non-vested units as of December 31, 2022	9,717	$	13.07	2.1	$	118.6

As of December 31, 2022, there was $50.4 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years.

Compensation Expense Related to Equity Awards

The following table summarizes information related to compensation expense recognized in the Consolidated Statements of Operations related to the equity awards:


	Years Ended December 31,
(in thousands)	2022		2021		2020
Research and development expense	$	25,089	$	17,340	$	20,817
Selling, general, and administrative expense	51,423		40,498		28,334
Total equity compensation expense	$	76,512	$	57,838	$	49,151

Assets and Liabilities Measured at Fair Value

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

Assets and Liabilities Measured at Fair Value

The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy which is defined as follows:

Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.

Level 3 — Inputs that are unobservable for the asset or liability.

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables:


(in thousands)	Level 2		Total
Assets:
Commercial paper	$	144,381	$	144,381
Money market	5,808		5,808
	$	150,189	$	150,189


(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	21,417	$	21,417
Deferred compensation plan liability	5,458		—		5,458
	$	5,458	$	21,417	$	26,875


(in thousands)	Level 2		Total
Assets:
Commercial paper	$	174,531	$	174,531
Corporate debt securities	32,311		32,311
Asset-backed securities	30,056		30,056
Money market	5,150		5,150
	$	242,048	$	242,048


(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	20,339	$	20,339
Deferred compensation plan liability	4,800		—		4,800
	$	4,800	$	20,339	$	25,139

The Company's Senior Secured Term Loan due 2026 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan due 2026 approximates the fair value.

The Company did not have any Level 3 assets as of December 31, 2022 or 2021.

Cash, Money Market Funds and Marketable Securities

The Company classifies its cash within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. No changes in valuation techniques or inputs occurred during the year ended December 31, 2022. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the year ended December 31, 2022.

Contingent Consideration Payable

The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. ("Callidus") in November 2013. The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Gains and losses are included in the Consolidated Statements of Operations.

The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined.

The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:

Contingent Consideration Liability

Fair Value as of December 31, 2022

Valuation Technique

Unobservable Input

Range

(in thousands)

Discount rate

10.2%

Clinical and regulatory milestones

21,417

Probability weighted discounted cash flow

Probability of achievement of milestones

88%-98%

Projected year of payments

2023

Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment, and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met.

The Company reached a regulatory milestone in September 2021 associated with FDA approval of the first BLA and in November 2021 associated with EMA validation of the MAA, both related to the contingent consideration of Callidus for the ATB200 Pompe disease program. The satisfaction of these milestones resulted in the collective milestone payment of $12.0 million.

The following table shows the change in the balance of contingent consideration payable for the year ended December 31, 2022 and 2021, respectively:


	Years ended December 31,
(in thousands)	2022		2021
Balance, beginning of the period	$	20,339	$	25,825
Changes in fair value during the period, included in the Consolidated Statements of Operations	1,078		6,514
Payment of contingent consideration in cash	—		(12,000)
Balance, end of the period(1)	$	21,417	$	20,339

______________________________

(1) As certain milestones are expected to be reached within the next twelve months, the December 31, 2022 balance was recorded as a current liability in the Consolidated Balance Sheets.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

Debt

12 Months Ended

Dec. 31, 2022

Debt Disclosure [Abstract]

Debt

The Company's debt consists of the following:


(in thousands)	December 31, 2022		December 31, 2021
Senior Secured Term Loan due 2026:
Principal	$	400,000	$	400,000
Less: debt discount (1)	(4,571)		(6,074)
Less: deferred financing (1)	(3,439)		(4,569)
Net carrying value of Long-term debt	$	391,990	$	389,357

_____________________________

(1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan using the effective interest rate method.

Senior Secured Term Loan due 2026

In July 2020, the Company entered into a definitive agreement for a $400 million credit facility with Hayfin Capital Management (“Senior Secured Term Loan due 2026”) with an interest rate equal to 3-month LIBOR, subject to a 1% floor, plus 6.5% per annum and requires interest-only payments until mid-2024. The Senior Secured Term Loan due 2026 will be repaid in nine quarterly payments of $44.4 million, starting on July 2024 with the final balance due on the maturity date in July 2026. This transaction resulted in net proceeds of $385.9 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2026. The Company used the proceeds to voluntarily settle the Senior Secured Term Loan with BioPharma Credit PLC, which included the $150 million principal, $1.1 million accrued interest, and $5.2 million in early settlement premiums. As a result of this early extinguishment, the Company recognized a loss on extinguishment of debt of $7.3 million in the Consolidated Statements of Operations.

The Senior Secured Term Loan due 2026 is subject to mandatory prepayment provisions that require prepayment upon a change of control, the incurrence of certain additional indebtedness, asset sale, or an event of loss, subject to certain conditions set forth in the Senior Secured Term Loan due 2026. The Company may prepay the Senior Secured Term Loan due 2026 in whole, at its option at any time. Any prepayment of the Senior Secured Term Loan due 2026 is subject to certain make-whole premiums and prepayment premiums, the latter of which decrease until the fourth anniversary of the transaction date at which point no prepayment penalty shall exist. The obligations under the Senior Secured Term Loan due 2026 are secured by a first lien security interest in certain assets of the Company. The Senior Secured Term Loan due 2026 contains certain customary representations and warranties, affirmative and negative covenants and events of default applicable to the Company. The Senior Secured Term Loan due 2026 also contains a minimum liquidity covenant of $75 million, and an incremental minimum consolidated revenue covenant, measured as of the previous four consecutive fiscal quarters. The minimum consolidated revenue covenant ranges from $140 million, beginning March 31, 2021, and peaks at $225 million by June 30, 2023, continuing at that level until the Senior Secured Term Loan due 2026 is repaid. If an event of default occurs and is continuing, Hayfin Capital Management may declare all amounts outstanding under the Senior Secured Term Loan due 2026 to be immediately due and payable.

Interest Expense

The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2022 and 2021, respectively:


(in thousands, except interest rate amounts)	December 31, 2022			December 31, 2021
Contractual interest expense	$	34,446		$	30,473
Amortization of debt discount	$	1,503		$	1,462
Amortization of deferred financing	$	1,131		$	1,028
Effective interest rate of the liability component, Senior Secured Term Loan due 2026	12.1		%	8.4		%

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
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-Topic 470
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-Subparagraph (SX 210.4-08(c))
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (g)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
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-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

+ Details

Leases

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

Leases

The Company currently has operating leases for office and research laboratory space, equipment, and vehicles under agreements expiring at various dates through 2044, which include renewal options on leases which the Company is reasonably certain to exercise.

For the years ended December 31, 2022, and 2021, operating lease expense was $9.8 million and $10.0 million and variable lease expense was $1.7 million and $2.1 million, respectively. For the years ended December 31, 2022, and 2021, the Company paid $8.3 million and $8.4 million, respectively, for amounts included in the measurement of operating lease liabilities and recorded $8.9 million and $0.3 million, respectively, of right-of-use assets. For the year ended December 31, 2022, there were no tenant improvements paid through lease incentives in exchange for new operating lease liabilities. For the year ended December 31, 2021, there were $0.3 million of tenant improvements paid through lease incentives in exchange for new operating lease liabilities.

Commitments under finance leases are not significant for the year ended December 31, 2022.

Supplemental balance sheet information related to operating leases were as follows:


(in thousands, except year and discount rate amounts)	December 31, 2022			December 31, 2021
Operating lease ROU assets, net	$	29,534		$	20,586

Current portion of the operating lease liabilities	$	8,552		$	7,409
Non-current portion of the operating lease liabilities	51,578			43,363
Total operating lease liability	$	60,130		$	50,772

Weighted-average remaining lease terms (years)	17.0			19.6
Weighted-average discount rate	12.2		%	13.1		%

At December 31, 2022, the future minimum operating lease payments were as follows:


(in thousands)	Operating Lease
2023	$	9,466
2024	8,624
2025	8,419
2026	8,571
2027	8,754
Thereafter	124,979
Total lease payments	168,813
Less lease incentives	(22,299)
Less imputed interest	(86,384)
Total operating lease liability	$	60,130

Income Taxes

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

Income Taxes

For financial reporting purposes, income (loss) before income taxes includes the following components:


	Years Ended December 31,
(in thousands)	2022		2021		2020
United States	$	(343,424)	$	(333,571)	$	(365,332)
Foreign	101,385		92,017		91,078
Total	$	(242,039)	$	(241,554)	$	(274,254)

Following were the components of income tax expense (benefit) for the years ended December 31, 2022, 2021, and 2020:


	Years Ended December 31,
(in thousands)	2022		2021		2020
Current
Federal	$	—	$	—	$	—
State	6		15		11
Foreign	(5,760)		8,857		4,163
Deferred
Federal	274		—		(1,421)
State	9		34		(155)
Foreign	—		—		—
Total	$	(5,471)	$	8,906	$	2,598

A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2022, 2021, and 2020 are as follows:


	Years Ended December 31,
	2022		2021		2020
Statutory rate	(21)	%	(21)	%	(21)	%
Tax credits	(11)		(9)		(7)
Impact of foreign operations	18		3		4
Other	3		3		2
Valuation allowance	9		28		23
Net	(2)	%	4	%	1	%

On December 22, 2017, the U.S. government enacted the Tax Cuts and Jobs Act ("Tax Act"). The Tax Act significantly revises U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Tax Act also introduced an additional U.S. tax on certain non-U.S. subsidiaries’ earnings which are considered to be Global Intangible Low Taxed Income (referred to as "GILTI"). After consideration of the relevant guidance and completing the accounting for the tax effects of the Tax Act, the Company has elected to treat GILTI as a period cost.

Beginning in 2022, the Tax Act eliminated the right to deduct research and development expenditures for tax purposes in the period the expenses were incurred and instead requires all U.S. and foreign research and development expenditures to be amortized over five and fifteen tax years, respectively.

Tax returns for years 2017 through 2022 are open to examination by tax authorities. The Company is also subject to examination in any period for which it has net operating losses.

Deferred income taxes reflect the net effect of temporary difference between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The significant components of the deferred tax assets and liabilities are as follows:


	Years Ended December 31,
(in thousands)	2022		2021
Deferred tax assets
Intellectual property	$	68,567	$	105,453
Research tax credit	223,366		205,095
Capitalized research and development costs	29,317		—
Net operating loss carry forwards	315,444		334,762
Share-based compensation	16,417		11,779
Interest carry forward limitation	12,558		8,285
Lease liability	11,428		9,105
Inventory	10,400		—
Other	20,109		17,748
Gross deferred tax assets	707,606		692,227
Deferred tax liabilities
Business acquisition	(4,939)		(4,930)
Royalty payable	(68,567)		(105,453)
Other	(6,806)		(4,932)
Total net deferred tax assets	627,294		576,912
Less: valuation allowance	(632,233)		(581,842)
Net deferred tax liability	$	(4,939)	$	(4,930)

The Company records a valuation allowance for temporary differences for which it is more likely than not that the Company will not receive future tax benefits. At December 31, 2022 and 2021, the Company recorded valuation allowances of $632.2 million and $581.8 million, respectively, representing an increase in the valuation allowance of $50.4 million in 2022, due to the uncertainty regarding the realization of such deferred tax assets, to offset the benefits of net operating losses generated during those years. The deferred tax liability related to business acquisitions pertains to the basis difference in IPR&D acquired by the Company. The Company's policy is to record a deferred tax liability related to acquired IPR&D that may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful.

As of December 31, 2022, the Company had federal and state net operating loss carry forwards ("NOLs") of approximately $1,183 million and $992 million, respectively. The federal carry forward for losses generated prior to 2018 will expire in 2029 through 2037. Federal net operating losses incurred in 2018 and onward have an indefinite expiration under the Tax Act. Most of the state carry forwards generated prior to 2009 have expired through 2016. The remaining state carry forwards including those generated in 2009 through 2022 will expire in 2029 through 2040. Utilization of NOLs may be subject to a substantial limitation pursuant to Section 382 of the Internal Revenue Code of 1986, as amended as well as similar state statutes in the event of an ownership change. Such ownership changes have occurred in the past and could occur again in the future. Under Section 382 of the Internal Revenue Code of 1986, as amended, or Section 382, if a corporation undergoes an "ownership change," generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation's ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income may be limited. The Company may experience ownership changes in the future as a result of shifts in the stock ownership some of which are outside the Company's control. The Company completed a detailed study of the NOLs for the tax year 2022 and determined that there was not an ownership change in excess of 50%. Ownership changes in future periods may place additional limits on the Company's ability to utilize net operating loss and tax credit carry forwards. In addition, at the state level, there may be periods during which the use of NOLs is suspended or otherwise limited, which could accelerate or permanently decrease the amount of state attributes and increase state taxes owed.

The Company also has research and experimentation and orphan drug credit carryforwards of approximately $36.4 million and $187.0 million, respectively, which will expire in the years 2030 through 2040. Deferred tax assets for these carryforwards are subject to a full valuation allowance.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 21
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32857-109319

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 11.C)
-URI https://asc.fasb.org/extlink&oid=122134291&loc=d3e330215-122817

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 17
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32809-109319

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB TOPIC 6.I.5.Q1)
-URI https://asc.fasb.org/extlink&oid=122134291&loc=d3e330036-122817

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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-Section 50
-Paragraph 14
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32705-109319

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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-SubTopic 270
-Section 50
-Paragraph 1
-URI https://asc.fasb.org/extlink&oid=6424409&loc=d3e44925-109338

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-URI https://asc.fasb.org/topic&trid=2144680

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 30
-Section 50
-Paragraph 2
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=6424122&loc=d3e41874-109331

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
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-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-URI https://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

+ Details

Collaborative Agreements	12 Months Ended
Collaborative Agreements	Dec. 31, 2022
Collaborative Agreements
Collaborative Agreements	Collaborative Agreements University of Pennsylvania In October 2018, as amended, the Company entered into a collaboration agreement with the University of Pennsylvania ("Penn") to pursue research and development of novel gene therapies. The Company's gene therapy portfolio pipeline expanded to include Pompe disease, Fabry disease and other rare diseases. In December 2022, the Company entered into a mutual termination agreement (the "Termination Agreement") pursuant to which the Company and Penn mutually agreed to terminate the collaboration agreement, as amended. In connection with the Termination Agreement, the Company agreed to pay Penn an aggregate of $23.7 million in connection with an unpaid portion of the discovery support payments, research program wind-down activities, and outstanding patent costs which was recorded as a component of research and development expense within the Consolidated Statements of Operations. Concurrently, the Company entered into a license agreement with Penn pursuant to which it obtained a license with respect to the pre-clinical research and development of next generation parvovirus gene therapy products for the treatment of Pompe disease and Fabry disease. Under the agreement, the Company will be responsible for clinical development and commercialization of the licensed products for the indications and Penn is eligible to receive certain milestone and royalty payments with respect to licensed products for each indication, up to an aggregate of $86.5 million per indication. Royalty payments are based on net sales of licensed products on a licensed product-by-licensed product and country-by-country basis. GlaxoSmithKline In July 2012, as amended in November 2013, the Company entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which Amicus obtained global rights to develop and commercialize Galafold® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S. The contingent milestone payments due to GSK are recorded within the other current and other non-current liabilities accounts on the Consolidated Balance Sheets. Sales based tiered royalties due to GSK are recorded within the cost of goods sold within the Consolidated Statements of Operations. For the year ended December 31, 2022, under the GSK collaboration agreements, the Company incurred approximately $25.1 million of royalty expenses. As of December 31, 2022, $7.4 million was recorded as deferred reimbursements and the Company recognized a liability of $6.9 million related to royalties payable to GSK within accrued expenses in the Consolidated Balance Sheets.

Legal Proceedings	12 Months Ended
Legal Proceedings	Dec. 31, 2022
Commitments and Contingencies Disclosure [Abstract]
Legal Proceedings	Legal Proceedings In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware for infringement of its Orange Book-listed patents and will vigorously enforce its Galafold® intellectual property rights.

Basic and Diluted Net Loss per Common Share

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

Basic and Diluted Net Loss per Common Share

The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:


	Years Ended December 31,
(in thousands, except per share amounts)	2022		2021		2020
Numerator:
Net loss attributable to common stockholders	$	(236,568)	$	(250,460)	$	(276,852)
Denominator:
Weighted average common shares outstanding — basic and diluted	289,057,198		271,421,986		258,867,380

Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options, unvested RSUs, outstanding warrants for common stock equivalents, and convertible debt units. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect.

The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:


	Years ended December 31,
(in thousands)	2022	2021	2020
Options to purchase common stock	19,064	14,731	14,032
Unvested restricted stock units	9,717	7,341	7,080
Outstanding warrants, convertible to common stock	—	—	2,555
Convertible notes	—	—	462
Total number of potentially issuable shares	28,781	22,072	24,129

Summary of Significant Accounting Policies (Policies)	12 Months Ended
Summary of Significant Accounting Policies (Policies)	Dec. 31, 2022
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented.
Consolidation	Consolidation The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
Foreign Currency Transactions	Foreign Currency Transactions The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of December 31, 2022 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash	Cash, Cash Equivalents, Marketable Securities, and Restricted Cash The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive (loss) gain in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs. Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other current assets and other non-current assets on the Company's Consolidated Balance Sheets.
Concentration of Credit Risk	Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities. The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company's accounts receivable at December 31, 2022 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis.
Property and Equipment	Property and Equipment Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three to five years, or the lesser of the related initial term of the lease or useful life for leasehold improvements. The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are charged to income in the period in which the costs are incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy. The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the long-lived asset over its estimated fair value in the period at which such a determination is made.
Revenue Recognition	Revenue Recognition The Company’s net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the years ended December 31, 2022, 2021 and 2020 were $329.0 million, $305.5 million and $260.9 million, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2022, one customer accounted for 27% of net product sales and 14% of accounts receivable from product sales. In 2021, one customer accounted for 24% of net product sales and 14% of accounts receivable from product sales. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
Inventories and Cost of Goods Sold	Inventories and Cost of Goods Sold Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Consolidated Statements of Operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable.
Fair Value Measurements	Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the Financial Accounting Standard Board ("FASB") guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participant's assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability.
Contingent Liabilities	Contingent Liabilities On an ongoing basis, the Company may be involved in various claims and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company's best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in material adverse adjustments to the Company's operating results.
Research and Development Costs	Research and Development Costs Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development.
Interest Income and Interest Expense	Interest Income and Interest Expense Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases.
Income Taxes	Income Taxes The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized. The Company’s tax returns are subject to examination by U.S. Federal, state, and foreign taxing jurisdictions. The impact of an uncertain tax position taken or expected to be taken on an income tax return must be recognized in the financial statements at the largest amount that is more likely than not to be sustained. An uncertain income tax position will not be recognized in the financial statements unless it is more likely than not to be sustained.
Other Comprehensive (Loss) Gain	Other Comprehensive (Loss) Gain Components of other comprehensive (loss) gain include unrealized gains and losses on available-for-sale securities and (loss) gain on foreign currency transactions and are included in the Consolidated Statements of Comprehensive Loss.
Leases	Leases The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets. ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories. The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.
Nonqualified Cash Deferral Plan	Nonqualified Cash Deferral Plan The Company's Cash Deferral Plan (the "Deferral Plan"), provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code. All of the investments held in the Deferral Plan are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets.
Equity-based Compensation	Equity-based Compensation At December 31, 2022, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 8. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award.
Loss per Common Share	Loss per Common ShareThe Company calculates net loss per share as a measurement of the Company's performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company had a net loss for all periods presented; accordingly, the inclusion of common stock options, unvested restricted stock units, and certain warrants would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same.
Segment Information	Segment Information The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker who comprehensively manages the entire business. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reporting unit.
Business Combinations	Business Combinations The Company assigns fair value to the tangible and intangible assets acquired and liabilities assumed based upon their estimated fair values on the acquisition date from acquired businesses. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and in-process research and development ("IPR&D"). In connection with the purchase price allocations for acquisitions, the Company estimates the fair value of contingent payments utilizing a probability-based income approach inclusive of an estimated discount rate.
Contingent Consideration Payable	Contingent Consideration Payable Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable. This does not apply in circumstances when the contingent consideration meets the definition of a derivative, in which case the amount becomes part of the basis in the asset acquired. Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets. For contingent consideration payments in business combinations, the Company determines the fair value of contingent acquisition consideration payable on the acquisition date using a probability-based income approach utilizing an appropriate discount rate. The short term and long term portions of the contingent consideration payable is shown on the Company's Consolidated Balance Sheets. The fair value of the contingent consideration payable will be determined each period end and the resulting change will be recorded on the Consolidated Statements of Operations.
Intangible Assets and Goodwill	Intangible Assets and GoodwillThe Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Purchased IPR&D is accounted for as an indefinite lived intangible asset until the underlying project is completed, at which point the intangible asset will be accounted for as a definite lived intangible asset, or abandoned, at which point the intangible asset will be written off or partially impaired. Goodwill and indefinite lived intangible assets are assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill or IPR&D impairment test is conducted. If it is determined the full carrying amount of IPR&D is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.
Recent Accounting Developments	Recent Accounting Developments The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Revenue Disaggregated by Geographical Area

The following table summarizes the Company's net product sales disaggregated by geographic area:


	For the Year
(in thousands)	2022		2021		2020
U.S.	$	115,946	$	95,387	$	80,046
Ex-U.S.	213,287		210,127		180,840
Total net product sales	$	329,233	$	305,514	$	260,886

Goodwill and Intangible Assets (Tables)

12 Months Ended

Dec. 31, 2022

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of Changes in IPR&D

The following table represents the changes in IPR&D for the years ended December 31, 2022 and 2021, respectively:


	(in millions)
Balance at December 31, 2020	$	23.0
Change in IPR&D	—
Balance at December 31, 2021	$	23.0
Change in IPR&D	—
Balance at December 31, 2022	$	23.0

Schedule of Changes in Goodwill

The following table represents the changes in Goodwill for the years ended December 31, 2022 and 2021, respectively:


	(in millions)
Balance at December 31, 2020	$	197.8
Change in goodwill	—
Balance at December 31, 2021	$	197.8
Change in goodwill	—
Balance at December 31, 2022	$	197.8

- Definition

Tabular disclosure of goodwill by reportable segment and in total which includes a rollforward schedule.

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (h)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (g)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (e)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (d)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1A
-URI https://asc.fasb.org/extlink&oid=121556970&loc=SL108378252-109267

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (f)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 2
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13854-109267

Reference 11: http://www.xbrl.org/2003/role/exampleRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 55
-Paragraph 24
-URI https://asc.fasb.org/extlink&oid=122137925&loc=d3e14258-109268

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 350
-SubTopic 20
-Section 50
-Paragraph 1
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=121556970&loc=d3e13816-109267

+ Details

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables)

12 Months Ended

Dec. 31, 2022

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Schedule of Cash and Available for Sale Securities

Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:


	As of December 31, 2022
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Commercial paper	144,299		82		—		144,381
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	293,513	$	82	$	—	$	293,595
Included in cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Included in marketable securities	144,700		82		—		144,782
Total cash, cash equivalents, and marketable securities	$	293,513	$	82	$	—	$	293,595


	As of December 31, 2021
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Commercial paper	174,578		7		(54)		174,531
Corporate debt securities	32,322		—		(11)		32,311
Asset-backed securities	30,070		—		(14)		30,056
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	482,568	$	7	$	(79)	$	482,496
Included in cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Included in marketable securities	237,371		7		(79)		237,299
Total cash, cash equivalents, and marketable securities	$	482,568	$	7	$	(79)	$	482,496

Schedule of Reconciliation of Cash and Cash Equivalents and Restricted Cash


(in thousands)	December 31, 2022		December 31, 2021		December 31, 2020
Cash and cash equivalents	$	148,813	$	245,197	$	163,240
Restricted cash	4,302		4,259		2,922
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	153,115	$	249,456	$	166,162

Inventories (Tables)

12 Months Ended

Dec. 31, 2022

Inventory Disclosure [Abstract]

Schedule of Inventories for the Period

The following table summarizes the components of inventories:


(in thousands)	December 31, 2022		December 31, 2021
Raw materials	$	10,054	$	12,289
Work-in-process	9,615		10,699
Finished goods	4,147		3,830
Total inventories	$	23,816	$	26,818

Property and Equipment (Tables)

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

Schedule of Property and Equipment

Property and equipment consist of the following:


	December 31,
(in thousands)	2022		2021
Leasehold improvements	$	24,162	$	24,168
Research equipment	16,345		16,663
Computer equipment	4,486		5,720
Construction in progress	4,160		8,229
Furniture and fixtures	2,734		3,163
Computer software	1,106		1,174
Vehicles	66		71
Land	—		3,190
Gross property and equipment	53,059		62,378
Less accumulated depreciation	(22,281)		(19,882)
Net property and equipment	$	30,778	$	42,496

Accrued Expenses and Other Current Liabilities (Tables)

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

Schedule of Accounts Payable and Accrued Expenses

Accrued expenses and other current liabilities consist of the following:


	December 31,
(in thousands)	2022		2021
Accrued compensation and benefits	$	25,701	$	24,258
Accrued sales rebates and discounts	21,886		15,989
Accrued program fees	10,515		13,294
Accrued contract manufacturing & contract research costs	8,230		20,361
Accrued royalties	6,908		4,522
Accrued professional fees	6,868		9,377
Accrued taxes	5,938		6,154
Other	7,590		4,198
	$	93,636	$	98,153

Share based Compensation (Tables)

12 Months Ended

Dec. 31, 2022

Share-Based Payment Arrangement [Abstract]

Schedule of Fair Value of Options

The fair value of the stock options granted was estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:


	Years Ended December 31,
	2022			2021			2020
Expected stock price volatility	62.1		%	65.4		%	75.1		%
Risk free interest rate	1.7		%	0.6		%	1.6		%
Expected life of options (years)	5.34			5.40			5.67
Expected annual dividend per share	$	—		$	—		$	—

Schedule of Stock Options Activity

A summary of the Company's stock options for the year ended December 31, 2022 were as follows:


	Number of Shares	Weighted Average Exercise Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Options outstanding, December 31, 2019	16,724	$	9.15
Granted	4,362	$	9.98
Exercised	(5,243)	$	8.11
Forfeited	(1,376)	$	10.42
Expired	(435)	$	13.33
Options outstanding, December 31, 2020	14,032	$	9.54
Granted	3,262	$	16.53
Exercised	(1,483)	$	7.05
Forfeited	(844)	$	12.97
Expired	(236)	$	13.28
Options outstanding, December 31, 2021	14,731	$	11.08
Granted	5,733	$	11.53
Exercised	(660)	$	6.58
Forfeited	(495)	$	12.57
Expired	(245)	$	12.84
Options outstanding, December 31, 2022	19,064	$	11.31	6.6	$	36.7
Vested and unvested expected to vest, December 31, 2022	17,492	$	11.22	6.4	$	35.1
Exercisable at December 31, 2022	10,938	$	10.59	5.0	$	29.0

Schedule of Non-Vested RSU Activity under the Plan

A summary of non-vested RSU activity under the Plan for the year ended December 31, 2022 is as follows:


	Number of Shares	Weighted Average Grant Date Fair Value		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Non-vested units as of December 31, 2019	5,792	$	11.18
Granted	4,692	$	11.29
Vested	(2,164)	$	10.70
Forfeited	(1,240)	$	11.14
Non-vested units as of December 31, 2020	7,080	$	11.35
Granted	3,191	$	16.94
Vested	(1,863)	$	15.77
Forfeited	(1,067)	$	12.82
Non-vested units as of December 31, 2021	7,341	$	13.90
Granted	5,048	$	11.93
Vested	(2,251)	$	12.48
Forfeited	(421)	$	12.06
Non-vested units as of December 31, 2022	9,717	$	13.07	2.1	$	118.6

Schedule of Equity Compensation Expenses

The following table summarizes information related to compensation expense recognized in the Consolidated Statements of Operations related to the equity awards:


	Years Ended December 31,
(in thousands)	2022		2021		2020
Research and development expense	$	25,089	$	17,340	$	20,817
Selling, general, and administrative expense	51,423		40,498		28,334
Total equity compensation expense	$	76,512	$	57,838	$	49,151

Assets and Liabilities Measured at Fair Value (Tables)

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

Summary of Assets and Liabilities Subject to Fair Value Measurements


(in thousands)	Level 2		Total
Assets:
Commercial paper	$	144,381	$	144,381
Money market	5,808		5,808
	$	150,189	$	150,189


(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	21,417	$	21,417
Deferred compensation plan liability	5,458		—		5,458
	$	5,458	$	21,417	$	26,875


(in thousands)	Level 2		Total
Assets:
Commercial paper	$	174,531	$	174,531
Corporate debt securities	32,311		32,311
Asset-backed securities	30,056		30,056
Money market	5,150		5,150
	$	242,048	$	242,048


(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	20,339	$	20,339
Deferred compensation plan liability	4,800		—		4,800
	$	4,800	$	20,339	$	25,139

Schedule of Significant Unobservable Inputs Used in the Valuation of the Contingent Consideration Payable

The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:

Contingent Consideration Liability

Fair Value as of December 31, 2022

Valuation Technique

Unobservable Input

Range

(in thousands)

Discount rate

10.2%

Clinical and regulatory milestones

21,417

Probability weighted discounted cash flow

Probability of achievement of milestones

88%-98%

Projected year of payments

2023

Schedule of Changes in Contingent Consideration Payable

The following table shows the change in the balance of contingent consideration payable for the year ended December 31, 2022 and 2021, respectively:


	Years ended December 31,
(in thousands)	2022		2021
Balance, beginning of the period	$	20,339	$	25,825
Changes in fair value during the period, included in the Consolidated Statements of Operations	1,078		6,514
Payment of contingent consideration in cash	—		(12,000)
Balance, end of the period(1)	$	21,417	$	20,339

______________________________

(1) As certain milestones are expected to be reached within the next twelve months, the December 31, 2022 balance was recorded as a current liability in the Consolidated Balance Sheets.

- Definition

Tabular disclosure of financial instruments measured at fair value, including those classified in shareholders' equity measured on a recurring or nonrecurring basis. Disclosures include, but are not limited to, fair value measurements recorded and the reasons for the measurements, level within the fair value hierarchy in which the fair value measurements are categorized and transfers between levels 1 and 2. Nonrecurring fair value measurements are those that are required or permitted in the statement of financial position in particular circumstances.

+ References + Details

Debt (Tables)

12 Months Ended

Dec. 31, 2022

Debt Disclosure [Abstract]

Schedule of Liability Components of the Convertible Notes

The Company's debt consists of the following:


(in thousands)	December 31, 2022		December 31, 2021
Senior Secured Term Loan due 2026:
Principal	$	400,000	$	400,000
Less: debt discount (1)	(4,571)		(6,074)
Less: deferred financing (1)	(3,439)		(4,569)
Net carrying value of Long-term debt	$	391,990	$	389,357

_____________________________

Components of Total Interest Expense Recognized Related to the Convertible Notes

The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2022 and 2021, respectively:


(in thousands, except interest rate amounts)	December 31, 2022			December 31, 2021
Contractual interest expense	$	34,446		$	30,473
Amortization of debt discount	$	1,503		$	1,462
Amortization of deferred financing	$	1,131		$	1,028
Effective interest rate of the liability component, Senior Secured Term Loan due 2026	12.1		%	8.4		%

Leases (Tables)

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

Schedule of Lease Cost

Supplemental balance sheet information related to operating leases were as follows:


(in thousands, except year and discount rate amounts)	December 31, 2022			December 31, 2021
Operating lease ROU assets, net	$	29,534		$	20,586

Current portion of the operating lease liabilities	$	8,552		$	7,409
Non-current portion of the operating lease liabilities	51,578			43,363
Total operating lease liability	$	60,130		$	50,772

Weighted-average remaining lease terms (years)	17.0			19.6
Weighted-average discount rate	12.2		%	13.1		%

Schedule of Future Minimum Operating Lease Payments

At December 31, 2022, the future minimum operating lease payments were as follows:


(in thousands)	Operating Lease
2023	$	9,466
2024	8,624
2025	8,419
2026	8,571
2027	8,754
Thereafter	124,979
Total lease payments	168,813
Less lease incentives	(22,299)
Less imputed interest	(86,384)
Total operating lease liability	$	60,130

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

Schedule of Income (Loss) Before Income Taxes

For financial reporting purposes, income (loss) before income taxes includes the following components:


	Years Ended December 31,
(in thousands)	2022		2021		2020
United States	$	(343,424)	$	(333,571)	$	(365,332)
Foreign	101,385		92,017		91,078
Total	$	(242,039)	$	(241,554)	$	(274,254)

Schedule of Income Tax Expense (Benefit)

Following were the components of income tax expense (benefit) for the years ended December 31, 2022, 2021, and 2020:


	Years Ended December 31,
(in thousands)	2022		2021		2020
Current
Federal	$	—	$	—	$	—
State	6		15		11
Foreign	(5,760)		8,857		4,163
Deferred
Federal	274		—		(1,421)
State	9		34		(155)
Foreign	—		—		—
Total	$	(5,471)	$	8,906	$	2,598

Schedule of Reconciliation of the Statutory Tax Rates and the Effective Tax Rates

A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2022, 2021, and 2020 are as follows:


	Years Ended December 31,
	2022		2021		2020
Statutory rate	(21)	%	(21)	%	(21)	%
Tax credits	(11)		(9)		(7)
Impact of foreign operations	18		3		4
Other	3		3		2
Valuation allowance	9		28		23
Net	(2)	%	4	%	1	%

Schedule of Significant Components of the Deferred Tax Assets and Liabilities

The significant components of the deferred tax assets and liabilities are as follows:


	Years Ended December 31,
(in thousands)	2022		2021
Deferred tax assets
Intellectual property	$	68,567	$	105,453
Research tax credit	223,366		205,095
Capitalized research and development costs	29,317		—
Net operating loss carry forwards	315,444		334,762
Share-based compensation	16,417		11,779
Interest carry forward limitation	12,558		8,285
Lease liability	11,428		9,105
Inventory	10,400		—
Other	20,109		17,748
Gross deferred tax assets	707,606		692,227
Deferred tax liabilities
Business acquisition	(4,939)		(4,930)
Royalty payable	(68,567)		(105,453)
Other	(6,806)		(4,932)
Total net deferred tax assets	627,294		576,912
Less: valuation allowance	(632,233)		(581,842)
Net deferred tax liability	$	(4,939)	$	(4,930)

Basic and Diluted Net Loss per Common Share (Tables)

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

Schedule of Reconciliation of the Numerator and Denominator Used in Computing Basic and Diluted Net Loss Per Common Share

The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:


	Years Ended December 31,
(in thousands, except per share amounts)	2022		2021		2020
Numerator:
Net loss attributable to common stockholders	$	(236,568)	$	(250,460)	$	(276,852)
Denominator:
Weighted average common shares outstanding — basic and diluted	289,057,198		271,421,986		258,867,380

Schedule of Potential Shares of Common Stock that were Excluded from the Computation as they were Anti-Dilutive Using the Treasury Stock Method

The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:


	Years ended December 31,
(in thousands)	2022	2021	2020
Options to purchase common stock	19,064	14,731	14,032
Unvested restricted stock units	9,717	7,341	7,080
Outstanding warrants, convertible to common stock	—	—	2,555
Convertible notes	—	—	462
Total number of potentially issuable shares	28,781	22,072	24,129

Description of Business (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Accumulated deficit	$ (2,532,490)	$ (2,295,922)

Summary of Significant Accounting Policies - Concentration of Credit Risk and Revenue Recognition (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Concentration Risk [Line Items]
Allowance for doubtful accounts receivable	$ 200
Net product sales	329,233	$ 305,514	$ 260,886
Galafold
Concentration Risk [Line Items]
Net product sales	$ 329,000	$ 305,500	$ 260,900
Net product sales \| Customer Concentration Risk \| Customer One
Concentration Risk [Line Items]
Concentration risk, percentage	27.00%	24.00%
Accounts receivable \| Customer Concentration Risk \| Customer One
Concentration Risk [Line Items]
Concentration risk, percentage	14.00%	14.00%

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 30
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8906-108599

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 40
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9031-108599

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 41
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9038-108599

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 4
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130543-203045

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 22
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8736-108599

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 22
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8736-108599

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 42
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9054-108599

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 5
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130545-203045

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 924
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
-URI https://asc.fasb.org/extlink&oid=6472922&loc=d3e499488-122856

+ Details

Summary of Significant Accounting Policies - Property and Equipment (Details)	12 Months Ended
	Dec. 31, 2022
Minimum
Property and Equipment
Estimated useful lives	3 years
Maximum
Property and Equipment
Estimated useful lives	5 years

Summary of Significant Accounting Policies - Revenue by Geographical Area (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Disaggregation of Revenue [Line Items]
Net product sales	$ 329,233	$ 305,514	$ 260,886
U.S.
Disaggregation of Revenue [Line Items]
Net product sales	115,946	95,387	80,046
Ex-U.S.
Disaggregation of Revenue [Line Items]
Net product sales	$ 213,287	$ 210,127	$ 180,840

- Definition

+ References

+ Details

Summary of Significant Accounting Policies - Equity Incentive Plan and Stock-Based Compensation (Details)	Dec. 31, 2022 plan
Equity-based Compensation
Number of equity-based employee compensation plans	1

Summary of Significant Accounting Policies - Segment Information and Recent Accounting Policies (Details)	12 Months Ended
	Dec. 31, 2022 segment unit
Accounting Policies [Abstract]
Number of business segments \| segment	1
Number of reporting units \| unit	1

Goodwill and Intangible Assets - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Goodwill and Intangible Assets Disclosure [Abstract]
Goodwill	$ 197,797	$ 197,797	$ 197,800
In-process research & development	$ 23,000	$ 23,000	$ 23,000

- Definition

Amount after accumulated impairment loss of an asset representing future economic benefits arising from other assets acquired in a business combination that are not individually identified and separately recognized.

+ References + Details

Goodwill and Intangible Assets - Changes in IPR&D (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Changes in IPR&D
Beginning balance	$ 23,000	$ 23,000
Change in IPR&D	0	0
Ending balance	23,000	23,000
Changes in goodwill
Beginning balance	197,797	197,800
Change in goodwill	0	0
Ending balance	$ 197,797	$ 197,797

- Definition

+ References + Details

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 148,813,000	$ 245,197,000	$ 163,240,000
Debt securities, available-for-sale	144,782,000	237,299,000
Debt securities, realized gain (loss)	0	0
Fair value of available-for-sale debt securities in unrealized loss positions	$ 0	$ 173,400,000

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash and Available for Sale Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Debt Securities, Available-for-sale [Line Items]
Cash and cash equivalents	$ 148,813	$ 245,197	$ 163,240
Fair value, cash balances	148,813	245,197
Cost, available-for-sale securities	144,700	237,371
Gross unrealized gain, available-for-sale securities	82	7
Gross unrealized loss, available-for-sale securities	0	(79)
Fair value, available-for-sale debt securities	144,782	237,299
Cost, cash balances and available-for-sale securities	293,513	482,568
Gross unrealized gain, cash balances and available-for-sale securities	82	7
Gross unrealized loss, cash balances and available-for-sale securities	0	(79)
Fair value, cash balances and available-for-sale securities	293,595	482,496
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	144,299	174,578
Gross unrealized gain, available-for-sale securities	82	7
Gross unrealized loss, available-for-sale securities	0	(54)
Fair value, available-for-sale debt securities	144,381	174,531
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Corporate debt securities, cost		32,322
Corporate debt securities, gross unrealized gain		0
Corporate debt securities, gross unrealized loss		(11)
Corporate debt securities, fair value		32,311
Asset-backed securities
Debt Securities, Available-for-sale [Line Items]
Corporate debt securities, cost		30,070
Corporate debt securities, gross unrealized gain		0
Corporate debt securities, gross unrealized loss		(14)
Corporate debt securities, fair value		30,056
Money market
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	350	350
Gross unrealized gain, available-for-sale securities	0	0
Gross unrealized loss, available-for-sale securities	0	0
Fair value, available-for-sale debt securities	350	350
Certificate of deposit
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	51	51
Gross unrealized gain, available-for-sale securities	0	0
Gross unrealized loss, available-for-sale securities	0	0
Fair value, available-for-sale debt securities	$ 51	$ 51

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Reconciliation of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 148,813	$ 245,197	$ 163,240
Restricted cash	4,302	4,259	2,922
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$ 153,115	$ 249,456	$ 166,162	$ 146,343

Inventories (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Inventory Disclosure [Abstract]
Raw materials	$ 10,054	$ 12,289
Work-in-process	9,615	10,699
Finished goods	4,147	3,830
Total inventories	23,816	26,818
Reserve for inventory	$ 400	$ 1,100

Property and Equipment (Details) - USD ($) $ in Thousands	12 Months Ended
Property and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Property and Equipment
Property and equipment, gross	$ 53,059	$ 62,378
Less accumulated depreciation	(22,281)	(19,882)
Net property and equipment	30,778	42,496
Depreciation expense	4,800	5,700
Asset impairment charges	6,616	0	$ 0
Leasehold improvements
Property and Equipment
Property and equipment, gross	24,162	24,168
Research equipment
Property and Equipment
Property and equipment, gross	16,345	16,663
Computer equipment
Property and Equipment
Property and equipment, gross	4,486	5,720
Construction in progress
Property and Equipment
Property and equipment, gross	4,160	8,229
Furniture and fixtures
Property and Equipment
Property and equipment, gross	2,734	3,163
Computer software
Property and Equipment
Property and equipment, gross	1,106	1,174
Vehicles
Property and Equipment
Property and equipment, gross	66	71
Land
Property and Equipment
Property and equipment, gross	$ 0	$ 3,190

Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Accounts payable and accrued expenses
Accrued compensation and benefits	$ 25,701	$ 24,258
Accrued sales rebates and discounts	21,886	15,989
Accrued program fees	10,515	13,294
Accrued contract manufacturing & contract research costs	8,230	20,361
Accrued royalties	6,908	4,522
Accrued professional fees	6,868	9,377
Accrued taxes	5,938	6,154
Other	7,590	4,198
Accrued expenses and other current liabilities	$ 93,636	$ 98,153

Stockholders' Equity - Common Stock and Warrants (Details) $ / shares in Units, $ in Thousands	1 Months Ended		3 Months Ended		12 Months Ended
	Nov. 30, 2022 USD ($)	Sep. 30, 2021 USD ($) $ / shares shares	Sep. 30, 2021 USD ($) $ / shares shares	Mar. 31, 2021 USD ($) $ / shares shares	Dec. 31, 2022 USD ($) vote $ / shares shares	Dec. 31, 2021 USD ($) $ / shares shares	Dec. 31, 2020 USD ($)
Convertible Debt
Common stock, shares authorized (in shares) \| shares					500,000,000	500,000,000
Voting right for each share held, number \| vote					1
Proceeds from issuance of common stock from equity financing and pre-funded warrants, net of issuance costs		$ 199,800			$ 0	$ 199,750	$ 0
Gross cash proceeds from warrant exercises				$ 19,200	$ 0	$ 19,230	$ 0
Common stock, par value (in dollars per share) \| $ / shares		$ 0.01	$ 0.01		$ 0.01	$ 0.01
Equity component of the convertible notes						$ 2,661
2016 Convertible Notes \| Convertible Notes
Convertible Debt
Debt conversion, converted instrument, amount			$ 2,800
At The Market Equity Program
Convertible Debt
Proceeds from issuance of common stock	$ 250,000
Private Placement
Convertible Debt
Stock issued (in dollars per share) \| $ / shares		$ 10.18	$ 10.18
Number of common shares called by warrants (in shares) \| shares		8,349,705	8,349,705
Exercise price of warrants (in dollars per share) \| $ / shares		$ 0.01	$ 0.01
Stock Exercises at $7.98
Convertible Debt
Exercise price of warrants (in dollars per share) \| $ / shares				$ 7.98
Stock Exercises at $7.06
Convertible Debt
Exercise price of warrants (in dollars per share) \| $ / shares				$ 7.06
Common Stock
Convertible Debt
Stock issued from equity financing (in shares) \| shares		11,296,660				11,296,660
Equity component of the convertible notes (in shares) \| shares			472,356
Equity component of the convertible notes			$ 5			$ 5
Additional Paid-In Capital
Convertible Debt
Equity component of the convertible notes			$ 2,700			$ 2,656
Warrants \| Private Placement
Convertible Debt
Stock issued (in dollars per share) \| $ / shares		$ 10.17	$ 10.17
Warrants \| Stock Exercises at $7.98
Convertible Debt
Stock issued from equity financing (in shares) \| shares				1,294,999
Warrants \| Stock Exercises at $7.06
Convertible Debt
Stock issued from equity financing (in shares) \| shares				1,260,000

Stockholders' Equity - Nonqualified Cash Plan and Equity Incentive Plans (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Stockholders' Equity
Deferred compensation investment	$ 5.5	$ 4.8
Expiration period	10 years
Vesting percentage	100.00%
Maximum \| Options to purchase common stock
Stockholders' Equity
Number of shares reserved for issuance (in shares)	15,472,672
First Anniversary
Stockholders' Equity
Vesting percentage	25.00%
Monthly vesting percentage	2.08%

Share based Compensation - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average grant-date fair value, options (in dollars per share)	$ 6.11	$ 9.08	$ 6.40
Aggregate intrinsic value of options exercised	$ 2,500	$ 8,500	$ 40,900
Proceeds from stock options exercised, net	4,310	$ 10,228	$ 42,282
Total unrecognized compensation costs	$ 31,100
Unvested restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Period following separation for accelerated vesting	2 years
Unrecognized compensation costs, period for recognition (in years)	2 years
Unrecognized compensation cost	$ 50,400
Options to purchase common stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation costs, period for recognition (in years)	3 years

Share based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected stock price volatility	62.10%	65.40%	75.10%
Risk free interest rate	1.70%	0.60%	1.60%
Expected life of options (years)	5 years 4 months 2 days	5 years 4 months 24 days	5 years 8 months 1 day
Expected annual dividend per share (in dollars per share)	$ 0	$ 0	$ 0

Share based Compensation - Stock Option Activity (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Number of Shares
Balance at the beginning of the period (in shares)	14,731	14,032	16,724
Options, Granted (in shares)	5,733	3,262	4,362
Options, Exercised (in shares)	(660)	(1,483)	(5,243)
Options, Forfeited (in shares)	(495)	(844)	(1,376)
Options, Expired (in shares)	(245)	(236)	(435)
Balance at the end of the period (in shares)	19,064	14,731	14,032
Vested and unvested expected to vest as of the end of the period (in shares)	17,492
Exercisable at the end of the period (in shares)	10,938
Weighted Average Exercise Price
Balance at the beginning of the period (in dollars per share)	$ 11.08	$ 9.54	$ 9.15
Options, Granted (in dollars per share)	11.53	16.53	9.98
Options, Exercised (in dollars per share)	6.58	7.05	8.11
Options, Forfeited (in dollars per share)	12.57	12.97	10.42
Options, Expired (in dollars per share)	12.84	13.28	13.33
Balance at the end of the period (in dollars per share)	11.31	$ 11.08	$ 9.54
Vested and unvested expected to vest as of the end of the period (in dollars per share)	11.22
Exercisable at the end of the period (in dollars per share)	$ 10.59
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Balance at the end of the period	6 years 7 months 6 days
Vested and unvested expected to vest at the end of the period	6 years 4 months 24 days
Exercisable at the end of the period	5 years
Aggregate intrinsic value of options outstanding	$ 36.7
Aggregate intrinsic value of options vested and unvested expected to vest	35.1
Aggregate intrinsic value of options exercisable	$ 29.0

Share based Compensation - RSUs and PBRSUs Summary (Details) - Unvested restricted stock units - USD ($) $ / shares in Units, shares in Thousands, $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Number of Shares
Non-vested units as of the beginning of the period (in shares)	7,341	7,080	5,792
Granted (in shares)	5,048	3,191	4,692
Vested (in shares)	(2,251)	(1,863)	(2,164)
Forfeited (in shares)	(421)	(1,067)	(1,240)
Non-vested units as of the end of the period (in shares)	9,717	7,341	7,080
Weighted Average Grant Date Fair Value
Non-vested units as of the beginning of the period (in dollars per share)	$ 13.90	$ 11.35	$ 11.18
Granted (in dollars per share)	11.93	16.94	11.29
Vested (in dollars per share)	12.48	15.77	10.70
Forfeited (in dollars per share)	12.06	12.82	11.14
Non-vested units as of the end of the period (in dollars per share)	$ 13.07	$ 13.90	$ 11.35
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Additional Disclosures [Abstract]
Non-vested units, weighted average remaining years	2 years 1 month 6 days
Non-vested units, aggregate intrinsic value	$ 118.6

Share based Compensation - Expense Summary (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 76,512	$ 57,838	$ 49,151
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	25,089	17,340	20,817
Selling, general, and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 51,423	$ 40,498	$ 28,334

Assets and Liabilities Measured at Fair Value - Summary of Fair Value of Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Assets:
Fair value of assets	$ 150,189	$ 242,048
Liabilities:
Contingent consideration payable	21,417	20,339
Deferred compensation plan liability	5,458	4,800
Fair value of liabilities	26,875	25,139
Commercial paper
Assets:
Fair value of assets	144,381	174,531
Corporate debt securities
Assets:
Fair value of assets		32,311
Asset-backed securities
Assets:
Fair value of assets		30,056
Money market
Assets:
Fair value of assets	5,808	5,150
Level 2
Assets:
Fair value of assets	150,189	242,048
Liabilities:
Contingent consideration payable	0	0
Deferred compensation plan liability	5,458	4,800
Fair value of liabilities	5,458	4,800
Level 2 \| Commercial paper
Assets:
Fair value of assets	144,381	174,531
Level 2 \| Corporate debt securities
Assets:
Fair value of assets		32,311
Level 2 \| Asset-backed securities
Assets:
Fair value of assets		30,056
Level 2 \| Money market
Assets:
Fair value of assets	5,808	5,150
Level 3
Liabilities:
Contingent consideration payable	21,417	20,339
Deferred compensation plan liability	0	0
Fair value of liabilities	$ 21,417	$ 20,339

Assets and Liabilities Measured at Fair Value - Significant Unobservable Inputs (Details) $ in Thousands	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)
Financial assets and liabilities subject to fair value measurements
Contingent consideration payable	$ 21,417	$ 20,339
Callidus \| Clinical and Regulatory Approval Milestones \| ATB-200 Pompe Program
Financial assets and liabilities subject to fair value measurements
Contingent consideration payable	$ 21,417
Callidus \| Clinical and Regulatory Approval Milestones \| Discount rate \| ATB-200 Pompe Program \| Probability weighted discounted cash flow
Financial assets and liabilities subject to fair value measurements
Contingent consideration, measurement input	0.102
Callidus \| Clinical and Regulatory Approval Milestones \| Probability of achievement of milestones \| ATB-200 Pompe Program \| Minimum
Financial assets and liabilities subject to fair value measurements
Contingent consideration, measurement input	0.88
Callidus \| Clinical and Regulatory Approval Milestones \| Probability of achievement of milestones \| ATB-200 Pompe Program \| Maximum
Financial assets and liabilities subject to fair value measurements
Contingent consideration, measurement input	0.98

Assets and Liabilities Measured at Fair Value - Level 3 Roll Forward (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Balance, beginning of the period	$ 20,339	$ 25,825
Changes in fair value during the period, included in the Consolidated Statements of Operations	1,078	6,514	$ 3,144
Payment of contingent consideration in cash	0	(12,000)
Balance, end of the period	$ 21,417	$ 20,339	$ 25,825

Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Debt Instruments
Net carrying value of Long-term debt	$ 391,990	$ 389,357
Senior Loans \| Senior Secured Term Loan due 2026:
Debt Instruments
Principal	400,000	400,000
Less: debt discount	(4,571)	(6,074)
Less: deferred financing	(3,439)	(4,569)
Net carrying value of Long-term debt	$ 391,990	$ 389,357

- Definition

Amount, excluding unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

+ References + Details

Debt - Narrative (Details)	1 Months Ended	12 Months Ended
Debt - Narrative (Details)	Jul. 31, 2020 USD ($) numberOfPayments	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Convertible Debt
Loss on extinguishment of debt		$ 0	$ (257,000)	$ (7,276,000)
Secured Debt \| Senior Loans
Convertible Debt
Maximum borrowing capacity	$ 400,000,000
Number of payments \| numberOfPayments	9
Periodic payment	$ 44,400,000
Proceeds from secured lines of credit	385,900,000
Minimum liquidity covenant	75,000,000
Secured Debt \| Senior Loans \| Minimum
Convertible Debt
Consolidated revenue covenant, amount	140,000,000
Secured Debt \| Senior Loans \| Maximum
Convertible Debt
Consolidated revenue covenant, amount	$ 225,000,000
Secured Debt \| Senior Loans \| LIBOR
Convertible Debt
Variable rate	1.00%
Basis spread on variable rate	6.50%
Secured Debt \| Senior Secured Term Loan due 2026:
Convertible Debt
Interest expense, debt	$ 1,100,000
Early settlement premiums	5,200,000
Loss on extinguishment of debt	(7,300,000)
Line of Credit
Convertible Debt
Maximum borrowing capacity	$ 150,000,000

Debt - Interest Expense (Details) - USD ($) $ in Thousands	12 Months Ended
Debt - Interest Expense (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Interest expense
Contractual interest expense	$ 34,446	$ 30,473
Amortization of debt discount	1,503	1,462
Amortization of deferred financing	$ 1,131	$ 1,028
Senior Notes \| Senior Secured Term Loan due 2026:
Interest expense
Effective interest rate of the liability component	12.10%	8.40%

Leases - Narrative (Details) - USD ($)	12 Months Ended
Leases - Narrative (Details) - USD ($)	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Leases [Abstract]
Operating lease cost	$ 9,800,000	$ 10,000,000
Variable lease cost	1,700,000	2,100,000
Cash paid for amounts included in operating lease liabilities	8,300,000	8,400,000
Right-of-use assets obtained in exchange for new operating lease liabilities	8,900,000	300,000
Tenant improvements paid through lease incentive	$ 0	$ 300,000	$ 470,000

Leases - Lease Cost (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Leases [Abstract]
Operating lease ROU assets, net	$ 29,534	$ 20,586
Current portion of the operating lease liabilities	8,552	7,409
Non-current portion of the operating lease liabilities	51,578	43,363
Total operating lease liability	$ 60,130	$ 50,772
Weighted-average remaining lease terms (years)	17 years	19 years 7 months 6 days
Weighted-average discount rate	12.20%	13.10%

Leases - Future Minimum Operating Lease Payments (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Operating Lease
2023	$ 9,466
2024	8,624
2025	8,419
2026	8,571
2027	8,754
Thereafter	124,979
Total lease payments	168,813
Less lease incentives	(22,299)
Less imputed interest	(86,384)
Total operating lease liability	$ 60,130	$ 50,772

Income Taxes - Statutory Rate (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Income (loss) before income taxes:
United States	$ (343,424)	$ (333,571)	$ (365,332)
Foreign	101,385	92,017	91,078
Loss before income tax	(242,039)	(241,554)	(274,254)
Current
Federal	0	0	0
State	6	15	11
Foreign	(5,760)	8,857	4,163
Deferred
Federal	274	0	(1,421)
State	9	34	(155)
Foreign	0	0	0
Total	$ (5,471)	$ 8,906	$ 2,598
Reconciliation of the statutory tax rates and the effective tax rates
Statutory rate	(21.00%)	(21.00%)	(21.00%)
Tax credits	(11.00%)	(9.00%)	(7.00%)
Impact of foreign operations	18.00%	3.00%	4.00%
Other	3.00%	3.00%	2.00%
Valuation allowance	9.00%	28.00%	23.00%
Net	(2.00%)	4.00%	1.00%

Income Taxes - Deferred and Valuation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Deferred tax assets
Intellectual property	$ 68,567	$ 105,453
Research tax credit	223,366	205,095
Capitalized research and development costs	29,317	0
Net operating loss carry forwards	315,444	334,762
Share-based compensation	16,417	11,779
Interest carry forward limitation	12,558	8,285
Lease liability	11,428	9,105
Inventory	10,400	0
Other	20,109	17,748
Gross deferred tax assets	707,606	692,227
Deferred tax liabilities
Business acquisition	(4,939)	(4,930)
Royalty payable	(68,567)	(105,453)
Other	(6,806)	(4,932)
Total net deferred tax assets	627,294	576,912
Less: valuation allowance	(632,233)	(581,842)
Net deferred tax liability	(4,939)	(4,930)
Valuation allowance	632,233	$ 581,842
Increase in the valuation allowance	$ (50,400)

Income Taxes - Loss Carryforwards (Details) $ in Millions	Dec. 31, 2022 USD ($)
Research and experimentation tax credit carryforward
Operating Loss Carryforwards [Line Items]
Tax credit carryforward amount	$ 36.4
Orphan drug tax credit carryforwards
Operating Loss Carryforwards [Line Items]
Tax credit carryforward amount	187.0
Federal
Operating Loss Carryforwards [Line Items]
Amount of net operating loss carry forwards	1,183.0
State
Operating Loss Carryforwards [Line Items]
Amount of net operating loss carry forwards	$ 992.0

Collaborative Agreements (Details) $ in Thousands	1 Months Ended		12 Months Ended
Collaborative Agreements (Details) $ in Thousands	Dec. 31, 2022 USD ($)	Nov. 30, 2013 USD ($) market	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Collaborative Agreements
Net product sales			$ 329,233	$ 305,514	$ 260,886
Deferred reimbursements	$ 4,656		4,656	5,906
Accrued expenses and other current liabilities	93,636		93,636	$ 98,153
Termination Agreement
Collaborative Agreements
Payments for collaborative agreement	23,700
Collaborative Arrangements
Collaborative Agreements
Conditional aggregate payments per indication	86,500		86,500
Revised Agreement \| GSK
Collaborative Agreements
Potential milestone payments upon achievement of post-approval and sales-based milestones		$ 40,000
Number of major markets outside the U.S. from whom parties to contractual arrangement is eligible to receive post-approval and sales-based milestones \| market		8
Net product sales			25,100
Deferred reimbursements	7,400		7,400
Accrued expenses and other current liabilities	$ 6,900		$ 6,900

Legal Proceedings (Details)	Nov. 30, 2022 lawsuit
Commitments and Contingencies Disclosure [Abstract]
Number of lawsuits	4

Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Numerator:
Net loss attributable to common stockholders	$ (236,568)	$ (250,460)	$ (276,852)
Denominator:
Weighted-average common shares outstanding - basic (in shares)	289,057,198	271,421,986	258,867,380
Weighted-average common shares outstanding - diluted (in shares)	289,057,198	271,421,986	258,867,380
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	28,781,000	22,072,000	24,129,000
Options to purchase common stock
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	19,064,000	14,731,000	14,032,000
Unvested restricted stock units
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	9,717,000	7,341,000	7,080,000
Outstanding warrants, convertible to common stock
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	0	0	2,555,000
Convertible notes
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	0	0	462,000