FDA Approves Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for the Treatment of Late-Onset Pompe Disease
Amicus Therapeutics announced that the U.S. Food and Drug Administration 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) who are not improving on their current enzyme replacement therapy (ERT).
Pombiliti + Opfolda is a unique two-component therapy. 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. Amicus Therapeutics will launch Pombiliti with Opfolda immediately in the U.S.
