Cipaglucosidase alfa and AT-GAA for the treatment of Pompe disease have had a Biologics License Application (BLA) and New Drug Application (NDA) accepted for review by the US Food and Drug Administration (FDA), respectively.

“The FDA’s acceptance of these filings is an immensely important step forward for people living with Pompe disease and their families in the United States,” John F. Crowley, chairman and chief executive officer of Amicus Therapeutics Inc., the developer of the treatments, said in a press release. “Patients need new medicines as soon as possible. We will work with great urgency with the FDA as they review the applications over the course of the coming months.” 

Crowley also said that the company is working toward additional regulatory submissions outside of the United States. “With today’s announcement, we remain confident in the potential of this medicine to become the next standard of care in Pompe disease,” he added.

Continue Reading

Cipaglucosidase alfa is a recombinant human acid α-glucosidase (rhGAA) enzyme, a next-generation enzyme replacement therapy (ERT) for Pompe disease.

Read more about ERT on our Pompe disease treatment page

AT-GAA is a 2-component therapy that consists of cipaglucosidase alfa and a stabilizer called miglustat. 

A BLA is a request for permission to introduce a biologic product into interstate commerce. An NDA is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the United States.

Prescription Drug User Fee Act (PDUFA) action dates of May 29, 2022, for the NDA and July 29, 2022, for the BLA were set by the FDA. 

Pompe disease is a rare genetic disease characterized by lysosomal acid alpha-glucosidase (GAA) enzyme deficiency. It is caused by mutations in the GAA gene and leads to muscle weakness, among other symptoms.


U.S. FDA accepts filings for Amicus’ AT-GAA for the treatment of Pompe disease. News release. Amicus Therapeutics; September 29, 2021.