UNITED STATES
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FORM
CURRENT REPORT PURSUANT TO
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Item 8.01 – Other Events
On December 3, 2021, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing that the European Medicines Agency validated the Marketing Authorization Applications for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. 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 December 3, 2021 | |
| 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: December 3, 2021 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
European Medicines Agency Validates Amicus
Therapeutics Marketing Authorization Applications
for AT-GAA for the treatment of Pompe Disease
PHILADELPHIA, PA, December 3, 2021 – Amicus Therapeutics (Nasdaq: FOLD) today announced that the European Medicines Agency (EMA) validated the Marketing Authorization Applications (MAA) for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Validation of the application confirms the submission is accepted, and the EMA’s centralized procedure with Committee for Medicinal Products for Human Use (CHMP)’s assessment begins.
The MAAs were submitted to the EMA based on the evaluation of the effects of AT-GAA in adults living with Pompe disease and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies, as well as data from the long-term open-label extension study.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics Inc., stated, “The acceptance of these filings is an important step forward for people living with Pompe disease and their families in Europe. Patients need new medicines as soon as possible. We will work with great urgency with the EMA as they review the applications over the course of the coming months. With today’s announcement, we remain confident in the potential of this medicine to become the next standard of care in Pompe disease.”
The U.S. Food and Drug Administration (FDA) previously granted Breakthrough Therapy designation for AT-GAA and accepted for review the Biologics License Application (BLA) and the New Drug Application (NDA). The FDA has set a Prescription Drug User Fee Act action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA.
About AT-GAA
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a 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.
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 us on Twitter and LinkedIn.
Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to regulatory submissions for AT-GAA and the status of those submissions. There can be no assurance that the FDA or EMA 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, 2020 and 10-Q for the quarter ending Sept. 31, 2021. 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.
CONTACT:
Investors:
Andrew Faughnan
Executive Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Diana Moore
Head of Global Corporate Affairs & Communications
dmoore@amicusrx.com
(609) 662-5079
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