Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan

Core Insights - Amicus Therapeutics has received approval from Japan's Ministry of Health, Labour and Welfare for Pombiliti (cipaglucosidase alfa) in combination with Opfolda (miglustat) to treat adult patients with late-onset Pompe disease [1][4]. Company Overview - Amicus Therapeutics is a biotechnology company focused on developing high-quality medicines for rare diseases, emphasizing patient dedication and innovative treatments [9][10]. Product Details - Pombiliti + Opfolda is a two-component therapy designed for increased uptake into muscle cells, with Pombiliti being a recombinant human GAA enzyme and Opfolda serving as an enzyme stabilizer [3][5]. - The therapy is indicated for adult patients with late-onset Pompe disease who weigh 40 kg or more and are not improving on current enzyme replacement therapy [6]. Clinical Study - The approval was based on clinical data from the Phase 3 pivotal study (PROPEL), which included both ERT-naïve and ERT-experienced participants [4]. Market Approval - With this approval, Pombiliti + Opfolda is now authorized in multiple regions including the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan [4]. Disease Background - Late-onset Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase, leading to glycogen accumulation and progressive muscle weakness [8].