Amicus Therapeutics Receives Japan Approval For Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Combination Therapy

Amicus Therapeutics has announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) in combination with Opfolda (miglustat) for the treatment of adults with late-onset Pompe disease (LOPD). This marks another key regulatory milestone for the therapy, which is now approved in multiple major markets including the United States, European Union, United Kingdom, Canada, Australia, Switzerland, and now Japan.

Pombiliti + Opfolda is a two-part therapy specifically designed to enhance treatment for LOPD, a rare and progressive neuromuscular disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Pombiliti is a recombinant human GAA enzyme that is engineered with high levels of bis-M6P (Mannose 6-Phosphate) to improve uptake into muscle cells, where it is most needed. Opfolda is an oral enzyme stabilizer that helps maintain the stability of the enzyme in the bloodstream, supporting its therapeutic activity.

Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics, Inc, stated, "We are delighted that we will now be able to offer a compelling new treatment option to patients living with late-onset Pompe disease in Japan. We are grateful to the MHLW and to Japan’s Pompe community, including the patients, families, and physicians who participated in our clinical studies, for their collaboration.”

The approval in Japan is based on results from the pivotal Phase 3 PROPEL study, which is the only trial in late-onset Pompe disease to include both treatment-naïve patients and those previously treated with enzyme replacement therapy (ERT), offering insights into a real-world patient population. The study provided evidence of the therapy’s safety and efficacy in improving clinical outcomes for adults living with LOPD. With this latest approval, Amicus continues to expand global access to Pombiliti + Opfolda, offering a new treatment option for patients with LOPD and strengthening its presence in the rare disease space.