PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD) announced that the U.S. Food and Drug Administration (FDA) has 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).
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.
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