U.S. FDA Defers Action on Filing for AT-GAA in Late-onset Pompe Disease
October 28, 2022 at 16:01 PM EDT
FDA Issues Deferred Action Letter on AT-GAA Regulatory Filing Due to the Inability to Conduct Required Manufacturing Site Inspection Prior to the PDUFA Action Date
Company is Now Actively Engaged with the Agency to Develop Plans and Logistics for a Pre-Approval Inspection
PHILADELPHIA, Oct. 28, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (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.1
“We are now one step away from the necessary approvals for AT-GAA in the U.S. We continue to believe this is a question of ‘when’ not ‘if’ AT-GAA will be approved and we will continue to work with great urgency to support the FDA’s completion of the final plant inspection necessary for approval so that this important new treatment option is made available for people living with Pompe disease in the United States,” said Bradley Campbell, President and Chief Executive Officer at Amicus Therapeutics, Inc. “We are also very pleased with the progress of the regulatory review in the EU and look forward to a Committee for Medicinal Products for Human Use (“CHMP”) opinion by the end of the year. We remain committed to bringing AT-GAA to as many people living with Pompe disease around the world as quickly as possible.”
Previously, the FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease based on clinical efficacy results from the Phase 1/2 clinical study. In the European Union, where a pre-approval inspection is not required, the regulatory review is on track and the Committee for Medicinal Products for Human Use (CHMP) opinion is expected before year end.
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