PHILADELPHIA – 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.
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 a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to the 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 on 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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