Pompe Disease

The FDA has accepted for review Amicus Therapeutics Inc's (NASDAQ: FOLD) Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA. Also Read: Amicus's Fabry Disease Medicine Gets European Approval For Use...

The FDA has approved Sanofi's SA (NASDAQ: SNY) Nexviazyme (avalglucosidase alfa-ngpt) to treat patients one year of age and older with late-onset Pompe disease. Pompe disease is a progressive and debilitating muscle disorder that impairs a person's ability to move and...

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Sanofi SA's (NASDAQ: SNY) avalglucosidase alfa for Pompe disease. The positive opinion is based on Phase 3 COMET study data, which showed...

Amicus Therapeutics Inc (NASDAQ: 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...