Amicus Therapeutics received approval from the U.S. FDA for Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) 65mg capsules. This two-component therapy is indicated for adults with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).
 
Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage. Disease severity ranges across a spectrum, with predominant manifestations such as skeletal muscle weakness and progressive respiratory involvement.
 
Pombiliti is a recombinant human GAA enzyme (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 glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.
 
The FDA approval was based on clinical data observed from the Phase 3 study (PROPEL), the only trial in LOPD to study ERT-experienced participants in a controlled setting.
 
“The 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. 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.

Amicus Therapeutics also entered into a definitive agreement for a $430 million financing collaboration with funds managed by Blackstone.