FDA Again Extends Review for Pompe Disease Therapy

The FDA cited COVID-19-related travel restrictions that have impacted the agency’s inspection of a manufacturing site in China.

The FDA has deferred action on Amicus Therapeutics’ biologics license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA. 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.

AT-GAA is a therapeutic to treat patients with Pompe Disease, a genetic lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA) that leads to severe muscle weakness that worsens over time. AT-GAA contains two components: cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme, and miglustat, a stabilizer of cipaglucosidase alfa.

Amicus officials expect the FDA to approve the two components of AT-GAA, including the BLA for cipaglucosidase alfa and a new drug application for miglustat, together.

Related: FDA Extends Review Time for Pompe Disease Therapy

This is second time action this therapeutic has been delay. In May 2022, the FDA had extended the review of this BLA by 90 days to allow additional time to review information submitted by the company as part of its ongoing reviews.