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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT PURSUANT TO

SECTION 13 OR 15(d) OF THE

SECURITIES EXCHANGE ACT OF 1934

 

Date of Report (Date of earliest event reported): May 10, 2022

 

AMICUS THERAPEUTICS, INC.

(Exact Name of Registrant as Specified in Its Charter)

 

Delaware   001-33497   71-0869350

(State or Other Jurisdiction

of Incorporation)

 

(Commission

File Number)

 

(I.R.S. Employer

Identification No.)

 

3675 Market Street, Philadelphia, PA 19104

(Address of Principal Executive Offices, and Zip Code)

 

215-921-7600

Registrant’s Telephone Number, Including Area Code

 

(Former Name or Former Address, if Changed Since Last Report.)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

  ¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
     
  ¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
     
  ¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
     
  ¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

 

Securities registered pursuant to Section 12(b) of the Act:

Title of each class   Trading Symbol(s)  

Name of each exchange on which
registered

Common Stock Par Value $0.01   FOLD   NASDAQ

 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company ¨

 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨

 

 

 

 

 

Item 8.01 – Other Events

 

On May 10, 2022, Amicus Therapeutics, Inc. issued a press release announcing that the U.S. Food and Drug Administration has extended the review period by 90 days for the Biologics License Application for cipaglucosidase alfa and the New Drug Application for miglustat, the two components of AT-GAA. A copy of this press release is attached hereto as Exhibit 99.1 and incorporated herein by reference.

 

Item 9.01 Financial Statements and Exhibits

 

(d) Exhibits:

 

Exhibit No.   Description
99.1   Press Release dated May 10, 2022
104   Cover Page Interactive Data File (embedded within the Inline XBRL document)

 

 

 

Signature Page

 

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

  

  AMICUS THERAPEUTICS, INC.
   
Date: May 10, 2022 By: /s/ Ellen S. Rosenberg
  Name: Ellen S. Rosenberg
  Title: Chief Legal Officer and Corporate Secretary

 

 

Exhibit 99.1

 

 

 

Amicus Therapeutics Receives Notification of PDUFA Date Extensions for AT-GAA

 

PHILADELPHIA, PA, May 10, 2022Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period by 90 days for the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA. The revised PDUFA action dates for miglustat and cipaglucosidase alfa are August 29, 2022 and October 29, 2022, respectively. The Company continues to expect the FDA to approve the applications together.

 

The FDA extended the PDUFA dates to allow additional time to review information submitted by the Company as part of its ongoing reviews. The extension of the review timeline was not related to requests for any additional clinical data. The Company also expects that the additional time will allow for the completion of the pre-license approval inspections necessary at the WuXi Biologics manufacturing site in China.

 

“We continue to work collaboratively with the FDA as it completes its review of the AT-GAA applications,” said John F. Crowley, Chairman and Chief Executive Officer at Amicus Therapeutics. “We want to thank the FDA for its continued diligence during the review process. We remain deeply committed to bringing AT-GAA to as many people living with Pompe disease as quickly as possible and delivering on our promise to become the potential new standard of care.”

 

Previously, the FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease based on clinical efficacy results from the Phase 1/2 clinical study. In the European Union, the Marketing Authorization Applications were validated in the fourth quarter of 2021 and the Committee for Medicinal Products for Human Use (CHMP) opinion is expected in late 2022.

 

About AT-GAA

 

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength.

 

In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 0 to 18 years with LOPD (ATB200-04). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03911505

 

About Pompe Disease

 

Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

 

About Amicus Therapeutics

 

Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.

 

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Forward-Looking Statements

 

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to top-line data from a global Phase 3 study to investigate AT-GAA for the treatment of Pompe Disease, the potential implications on these data for the future advancement and development of AT-GAA, expectations regarding the FDA regulatory process, and the outcome of the FDA’s review. There can be no assurance that the FDA will grant approval for AT-GAA. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward-looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully manufacture and commercialize AT-GAA. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2021 and Quarterly Report 10-Q for the quarter ended March 31, 2022. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward-looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.

 

CONTACTS:

 

Investors: 

Amicus Therapeutics 

Andrew Faughnan

Executive Director, Investor Relations 

afaughnan@amicusrx.com

(609) 662-3809

 

Media: 

Amicus Therapeutics 

Diana Moore 

Head of Global Corporate Communications 

dmoore@amicusrx.com

(609) 662-5079

 

FOLD-G

 

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