FDA Clears Amicus Therapeutics’ Pompe Disease Treatment

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Pombiliti + Opfolda indicated for adults who are not improving on current enzyme therapy.

Amicus Therapeutics announced that it has officially received FDA approval for Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat). Indicated for the treatment of adults living with late-onset Pompe disease (LOPD) who aren’t improving on their current enzyme replacement therapy (ERT), the medication is designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen, with Opfolda working as an enzyme stabilizer designed to soothe the enzyme in the blood.


“Today’s FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine, and our passionate determination to improve the lives of people living with Pompe disease,” said John F. Crowley, executive chairman, Amicus Therapeutics, Inc. “This approval embodies our Amicus spirit, passion, and resilience and is a very meaningful step for the Pompe community. I am just so immensely proud of our team, and so very grateful to everyone who has worked to bring this medicine to this approval. Most especially to all of the people living with Pompe around the world.”

Reference: Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease. GlobeNewswire. September 28, 2023. Accessed September 28, 2023. https://www.globenewswire.com/news-release/2023/09/28/2751407/0/en/Amicus-Therapeutics-Announces-FDA-Approval-and-Launch-of-New-Treatment-for-Pompe-Disease.html