FDA defers PDUFA action on filing for AT-GAA in late-onset Pompe disease.-Amicus Therapeutics.
Amicus Therapeutics announced that the FDA has deferred action on the 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. As a result, the FDA is deferring action on the application until the manufacturing site inspection is complete.
The Company continues to expect the FDA to approve the two components of AT-GAA, including the BLA and New Drug Application (NDA) for miglustat, together.
The sole reason cited in the FDA-issued letter for the deferred action was the Agency’s inability to complete the manufacturing facility inspection. While both applications remain under review, the FDA has not provided anticipated action date(s) as they continue to monitor the public health situation and travel restrictions in China. However, the Company is now actively engaged with the FDA on developing plans and logistics for a pre-approval inspection plan.
Under FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, the Agency may defer action on a pending application when a facility inspection is necessary but cannot be completed by the PDUFA goal date due to travel restrictions, provided that no deficiencies have been identified and the application otherwise satisfies the requirements for approval.
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