Amicus Therapeutics (NASDAQ: FOLD) has announced that the EMA has approved its Marketing Authorization Application for AT-GAA, its experimental two-component therapy for Pompe disease treatment.
EMA approves Amicus’s AT-GAA
The EMA’s centralized approach with the Committee for Medicinal Products for Human Use (CHMP) assessment begins after the application is validated. The MAAs were filed to the EMA based on an assessment of AT-GAA effects and safety profile in adults with Pompe disease, including data from the Phase 1/2 and Phase 3 PROPEL trials and data from a long-term open-label extension study.
CEO John Crowley said, “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 US Food and Drug Administration previously designated AT-GAA as a Breakthrough Therapy and accepted the Biologics License Application (BLA) and the New Drug Application (NDA) for evaluation (NDA). The FDA has set a May 29, 2022 action date for the NDA and July 29, 2022, for the BLA under the Prescription Drug User Fee Act.
AT-GAA comprises a recombinant human acid alpha-glucosidase
AT-GAA is a two-component investigational therapy consisting of cipaglucosidase alfa (ATB200), a recombinant human acid alpha-glucosidase (rhGAA) enzyme with enhanced carbohydrate structures, especially bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to improve absorption into cells, and miglustat (AT2221), a cipa AT-GAA was linked to higher amounts of the mature lysosomal form of GAA and lower glycogen levels in muscle in preclinical trials, as well as improved muscular strength and alleviation of the autophagic deficiency.
Deficiency of the enzyme acid alpha-glucosidase causes Pompe disease, a hereditary lysosomal condition (GAA). Reduced or missing GAA levels cause glycogen buildup in cells, which is thought to cause the clinical signs of Pompe disease.