The US Food and Drug Administration (FDA) has postponed by 90 days its Prescription Drug User Fee Act (PDUFA) action dates for AT-GAA, an investigational 2-component Pompe disease therapy, according to a news release from the therapies’ manufacturer, Amicus Therapeutics.
The revised action dates for the 2 components are now August 29, 2022, for the New Drug Application (NDA) for miglustat and October 29, 2022, for the Biologics License Application (BLA) for cipaglucosidase alfa. The FDA did not request any additional clinical data but said it needs more time to review the submitted information.
“We continue to work collaboratively with the FDA as it completes its review of the AT-GAA applications,” said John F. Crowley, chairman and chief executive officer at Amicus Therapeutics.
The additional time is also expected to allow for prelicense approval inspections of the WuXi Biologics manufacturing site in China.
Amicus still expects the FDA to approve the 2 applications together.
Read more about experimental Pompe disease therapies.
“We remain deeply committed to bringing AT-GAA to as many people living with Pompe disease as quickly as possible and delivering on our promise to become the potential new standard of care,” Crowley said.
AT-GAA is composed of a recombinant human acid alpha-glucosidase enzyme (cipaglucosidase alfa; ATB200) along with a miglustat (AT2221), a stabilizer for cipaglucosidase alfa. The FDA previously granted Breakthrough Therapy designation for AT-GAA for the treatment of late-onset Pompe disease. This designation was based on the results of a phase 1/2 clinical trial.
Preclinical trials also had encouraging results, with AT-GAA treatment being associated with increased levels of lysosomal forms of acid alpha-glucosidase as well as reductions in muscle glycogen levels and an improvement in muscle strength.
Enrollment has begun for an open-label, uncontrolled, multicenter study (NCT03911505) of AT-GAA in pediatric patients with late-onset Pompe disease. The study will evaluate the pharmacokinetics, pharmacodynamics, safety, and efficacy of the treatment.
Amicus Therapeutics receives notification of PDUFA date extensions for AT-GAA. News release. Amicus Therapeutics; May 10, 2022.