Amicus Therapeutics (NASDAQ: FOLD), headquartered in the United States, has announced that it has received marketing approval from the U.S. Food and Drug Administration (FDA) for its combination therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules. This innovative two-component therapy is specifically indicated for adults diagnosed with late-onset Pompe disease (LOPD) who weigh 40 kg or more and are not experiencing improvements with their current enzyme replacement therapy (ERT).
Background on Pombiliti and Its Development
Pombiliti is a recombinant human GAA enzyme (rhGAA) that is naturally expressed with elevated levels of bis-M6P (Mannose 6-Phosphate). The development of this treatment originated from concepts generated by Wuxi Biologics’s platform in 2012. Amicus Therapeutics collaborates with Wuxi Biologics, a leading Chinese Contract Research, Development, and Manufacturing Organization (CRDMO), which supports Amicus in commercial manufacturing across five production facilities.
This FDA approval marks a significant advancement in treatment options for patients suffering from LOPD, a rare genetic disorder characterized by progressive muscle weakness and respiratory issues.-Fineline Info & Tech
