The US Food and Drug Administration (FDA) deferred action on the Biologics License Application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA, a next-generation enzyme replacement therapy for late-onset Pompe disease, according to a press release by Amicus Therapeutics, the developers of the treatment.

The delay is due to the agency’s inability to inspect the manufacturing site of cipaglucosidase alfa in China during the review cycle due to COVID-19-related travel restrictions.

“We are now one step away from the necessary approvals for AT-GAA in the US,” Bradley Campbell, the president and chief executive officer of Amicus Therapeutics, said in the press release.

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“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.”

AT-GAA consists of 2 components: cipaglucosidase alfa, a recombinant human acid α-glucosidase enzyme, and miglustat, a stabilizer. The company is still hoping the agency will approve the 2 components of AT-GAA.

The FDA had previously accepted a BLA and a New Drug Application for AT-GAA for review. The potential new treatment is also under regulatory review in the European Union, where a preapproval inspection is not needed.

“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,” Campbell said. “We remain committed to bringing AT-GAA to as many people living with Pompe disease around the world as quickly as possible.”

Pompe disease is a rare genetic disease characterized by a deficiency of the GAA enzyme, causing glycogen build up, and leading to muscle weakness and poor muscle tone, among other symptoms. It is thought that the disease affects around 1 in 40,000 people in the US.

The FDA has not set anticipated review dates.


U.S. FDA defers action on filing for AT-GAA in late-onset Pompe disease. News release. Amicus Therapeutics; October 28, 2022