FOLD - EMA validates Amicus' marketing applications for AT-GAA in Pompe disease

Amicus Therapeutics (NASDAQ:FOLD) announces that the EMA validated the Marketing Authorization Applications (MAA) for AT-GAA, an investigational two-component therapy for the treatment of Pompe disease, an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase. Validation confirms the submission is accepted, and the EMA’s centralized procedure with Committee for Medicinal Products for Human Use's assessment begins. The MAAs were submitted based on the data from Phase 1/2 and Phase 3 PROPEL studies, as well as long-term open-label extension study. The FDA has previously granted Breakthrough Therapy designation for AT-GAA and accepted for review the BLA and the NDA. The Agency has set a PDUFA action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA.

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EMA validates Amicus' marketing applications for AT-GAA in Pompe disease