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# FDA Defers Action on Amicus BLA
- URL: https://www.fdaweb.com/fda-defers-action-on-amicus-bla/
- Published: 2022-11-01T12:00:00.000Z
- Updated: 2026-09-14T18:04:47.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5153100

FDA has deferred action on an Amicus Therapeutics BLA for cipaglucosidase alfa, the biological component of AT-GAA in late-onset Pompe disease, due to the agency’s inability to conduct a required manufacturing site inspection in China before the PDUFA action date. A company [announcement](https://www.globenewswire.com/news-release/2022/10/28/2544135/15991/en/U-S-FDA-Defers-Action-on-Filing-for-AT-GAA-in-Late-onset-Pompe-Disease.html?ref=fdaweb.com) says the inability to conduct the inspection was the only reason cited by FDA for the deferred action. No anticipated new action date was given, Amicus says.

AT-GAA is described as an investigational two-component therapy consisting of cipaglucosidase alfa, a unique recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa. FDA granted it breakthrough therapy designation to treat late-onset Pompe disease based on clinical efficacy results from a Phase 1/2 clinical study.