Amicus Therapeutics Concludes Pre-BLA Meeting With FDA For AT-GAA In Pompe Disease - Amicus Therapeutics (NASDAQ:FOLD)

Amicus Therapeutics Inc FOLD has completed a Type B Pre-Biologics License Application (BLA) meeting with the FDA for AT-GAA (cipaglucosidase alfa co-administered with miglustat) for Pompe disease.
Based on this formal pre-BLA meeting and the final written communication received from the FDA, Amicus intends to complete the rolling submission for cipaglucosidase alfa and submit a marketing application for miglustat.
Amicus intends to base its filings on evaluating the effects of AT-GAA in late-onset Pompe patients and its safety profile, which will include data from both the Phase 1/2 and Phase 3 PROPEL studies as well as data from the open-label extension study.
As part of the rolling BLA, Amicus previously submitted the non-clinical component of the cipaglucosidase alfa application and is on track to submit all of the remaining modules of the application by the end of the second quarter.
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), an enzyme replacement therapy administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.
Marketing applications for AT-GAA are expected to be submitted with the European Medicines Agency (EMA) in the second half of 2021.
Price Action: FOLD shares are up 4.4% at $10.27 in the premarket session on the last check Monday.