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  • EMA validate filing of AT GAA for Pompe disease.

EMA validate filing of AT GAA for Pompe disease.

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Published:6th Dec 2021
Amicus Therapeutics announced that the European Medicines Agency validated the Marketing Authorization Applications (MAA) for AT GAA (cipaglucosidase alfa + miglustat), 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 I/II and Phase III PROPEL studies, as well as data from the long-term open-label extension study.

PROPEL study was published in The Lancet Neurology : See- "Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase III trial."- The Lancet Neurology.Vol. 20 No. 12 p1027–1037 Published: December, 2021. Benedikt Schoser,Mark Roberts,Barry J Byrne,Sheela Sitaraman,Hai Jiang,Pascal Laforêt,and others.

Condition: Pompe Disease
Type: drug

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