Amicus Therapeutics, Inc. (FOLD), Wednesday announced that Japan's Ministry of Health, Labour and Welfare or MHLW has approved the combination therapy of Pombiliti and Opfolda for the treatment of adult patients with late-onset Pompe disease, an inherited lysosomal disorder.
The biotechnology company explained that Pombiliti is a recombinant human GAA enzyme naturally expressed with high levels of bis-M6P, designed for increased uptake into muscle cells, whereas, Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.
The recent decision was based on clinical data from the Phase 3 pivotal study, which explored the real-world population of both ERT-naive and ERT-experienced participants in a controlled setting.
With this approval, the combination therapy is now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland and Japan.
In the pre-market hours, FOLD is trading at $5.77, up 1.05 percent on the Nasdaq.
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