PHILADELPHIA, Sept. 28, 2023 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly. weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)A treatment that replaces the missing enzyme through IV infusion..
PombilitiAn investigational enzyme replacement therapy designed to improve enzyme activity in muscles. + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzymeA protein that helps the body carry out chemical reactions. (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), 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 glycogenA stored form of sugar used for energy.. Opfolda is an enzyme stabilizer designed to stabilize 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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart.. 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,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc.