FDA Delays PDUFA Date for Amicus’ AT-GAA Therapy

The FDA has extended by 90 days its Prescription Drug User Fee Act (PDUFA) decision dates for Amicus Therapeutics’ AT-GAA dual therapy, which includes a Biologics License Application (BLA) for cipaglucosidase alfa and a New Drug Application (NDA) for miglustat for the treatment of patients with the rare genetic disorder Pompe disease.

The PDUFA decision date for the NDA is Aug. 29 and for the BLA is Oct. 29. The company said the agency needs more time to review the information submitted.

Amicus said that the FDA did not request any additional data, but that the delay would allow for prelicense approval inspections at its manufacturing site in China.

The dual therapy, which is meant to stimulate and stabilize enzyme activity, previously received the FDA’s Breakthrough Therapy designation for treatment of late-onset Pompe disease.