PHILADELPHIA, PA — Amicus Therapeutics (Nasdaq: FOLD) announced that the U.S. Food and Drug Administration (FDA) this week accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives. The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Pompe disease.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. The BLA and NDA are based on the evaluation of the effects of AT-GAA in Pompe disease patients and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies as well as data from the open-label extension study.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics Inc., stated, “The FDA’s acceptance of these filings is an immensely important step forward for people living with Pompe disease and their families in the United States. Patients need new medicines as soon as possible. We will work with great urgency with the FDA as they review the applications over the course of the coming months. In parallel, we are diligently working towards additional regulatory submissions outside of the U.S. With today’s announcement, we remain confident in the potential of this medicine to become the next standard of care in Pompe disease.”
Previously, the U.S. 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. The Marketing Authorization Application for AT-GAA is expected to be submitted in the EU in the fourth quarter of 2021. In June 2021, the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted AT-GAA a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
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