Sarepta Therapeutics Inc., a leader in precision genetic medicine for rare diseases, has announced that the Japanese Ministry of Health, Labour, and Welfare has granted approval for ELEVIDYS (delandistrogene moxeparvovec), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD). This approval falls under Japan's conditional and time-limited approval pathway, which allows for marketing authorization for up to seven years for innovative medicines aimed at treating serious conditions. ELEVIDYS is approved for children aged 3 to less than 8 years old who do not have deletions in exon 8 and/or exon 9 of the DMD gene. This approval is based on the efficacy and safety results from clinical studies, including longer-term functional outcomes from the EMBARK global phase 3 study. Sarepta is set to receive up to $103.5 million in near-term regulatory and commercial milestone payments. Commercialization in Japan will be handled by Chugai Pharmaceuticals through its alliance with Roche.
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