Posted by Michael Wonder on 04 Dec 2021
European Medicines Agency validates Amicus Therapeutics marketing authorisation applications for AT-GAA for the treatment of Pompe disease
3 December 2021 - Amicus Therapeutics today announced that the EMA validated the marketing authorisation applications for AT-GAA, the Company’s investigational two component therapy for the treatment of Pompe disease.
The marketing authorisation applications 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.
