FOLD - Amicus Therapeutics announces pre-BLA meeting with FDA for AT-GAA in Pompe disease

Amicus Therapeutics (FOLD) has successfully completed a Type B Pre-Biologics License Application ((BLA)) meeting with the FDA for AT-GAA (cipaglucosidase alfa co-administered with miglustat), its investigational two-component therapy for the treatment of Pompe disease.Based on this pre-BLA meeting, the company intends to complete the rolling BLA submission for cipaglucosidase alfa and submit a New Drug Application for miglustat. The filings will be based on the evaluation of the effects of AT-GAA in late-onset Pompe patients and its safety profile and data from both the Phase 1/2 and Phase 3 PROPEL studies as well as open label extension study.As part of the rolling BLA, FOLD previously submitted the nonclinical component of the cipaglucosidase alfa BLA and is on-track to submit all of the remaining modules of the application by the end of Q2.FOLD shares up 4% premarket trading at $10.23.

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Amicus Therapeutics announces pre-BLA meeting with FDA for AT-GAA in Pompe disease