BRIEF—FDA defers action on Amicus' Pompe disease candidate BLA

The US Food and Drug Administration (FDA) has deferred action on the Biologics License Application (BLA) for the investigation Pompe disease drug cipaglucosidase alfa, the biologic component of AT-GAA, US biotech Amicus Therapeutics announced on Friday, leading to a 7.4% drop in its share price in after hours trading.

Due to restrictions on travel related to COVID-19, the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle.

As a result, the FDA is deferring action on the application until the manufacturing site inspection is complete.

Amicus says it continues to expect the FDA to approve the two components of AT-GAA, including the BLA and New Drug Application (NDA) for miglustat, together.

While both applications remain under review, the FDA has not provided anticipated action date(s) as the agency continues to monitor the public health situation and travel restrictions in China.