FDA Defers Action on Pompe Disease Candidate

The FDA has issued a Deferred Action letter for Amicus Therapeutics’ biologic license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA, its investigational dual therapy for muscle-wasting Pompe disease.

The agency said COVID-related travel restrictions prevented inspectors from visiting WuXi Biologics, the Chinese manufacturing plant that produces the enzyme, a recombinant form of the naturally occurring alpha-glucosidase (rhGAA) enzyme.

AT-GAA consists of a novel rhGAA for improved uptake and miglustat, an enzyme stabilizer.

The indefinite delay will be lifted only when FDA inspectors are able to visit the plant, the company said. But regulatory action on miglustat, the second component of the dual candidate is proceeding, even in the absence of a confirmed timeline.