Amicus Therapeutics Announces Approval of Pombiliti® (Cipaglucosidase Alfa) + Opfolda® in Japan

Published: 2025-06-25 08:05:46 ET FOLD

Published on 06/25/2025 at 08:05

Amicus Therapeutics announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD). Pombiliti + Opfolda is a 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.

Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. The MHLW approval for Pombiliti + Op folda was based on clinical data from the Phase 3 pivotal study (PROPEL). PROPEL is the only trial in LOPD to study the real-world population of both ERT-naive and ERT-experienced participants in a controlled setting.

With this approval, Pombiliti + Op foldinga is now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland and Japan. Pombiliti® + Opfolda®, is a two-component therapy that consists of cipaglucosidases alfa-atga, a bis-M6P-enriched rgAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONSANDUSAGE POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients With late-onset Pompe disease ("lysosomal acid alpha-glucosidase [GAA] deficiency) weighing 40 kg and who are not improving on their current enzyme replacement therapy (ERT).

SFETY INFORMATION including ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions < 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.