Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase. It affects approximately 5,000 to 10,000 people worldwide. Amicus Therapeutics developed AT-GAA, a two-component therapy consisting of an optimized form of the GAA enzyme called cipaglucosidase alfa (ATB200), which is administered with an oral small molecule called miglustat (ATB2221) that acts as a pharmacological chaperone.

The MAA was 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 I/II and phase III Propel studies, as well as data from the long-term, open-label extension study.

The U.S. Food and Drug Administration previously granted a breakthrough therapy designation for AT-GAA and accepted for review the biologics license application and the new drug application.

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