CAMBRIDGE, Mass. — Sarepta Therapeutics has received regulatory approval in Japan for ELEVIDYS, its gene therapy for Duchenne muscular dystrophy (DMD), marking a significant milestone in the global fight against the rare and progressive neuromuscular disorder.
Japan’s Ministry of Health, Labour and Welfare (MHLW) approved ELEVIDYS (delandistrogene moxeparvovec) under its conditional and time-limited approval pathway. The treatment is now authorized for boys aged 3 to under 8 years who do not have deletions in exon 8 or 9 of the DMD gene and who are negative for anti-AAVrh74 antibodies. Notably, this marks the first global approval of the therapy for children under age 4.
The decision was based on safety and efficacy data from Sarepta’s clinical program, including two-year outcomes from the global Phase 3 EMBARK trial. Results showed that children treated with ELEVIDYS demonstrated significant improvements in muscle function compared to a well-matched external control group. No new safety concerns were reported during the study period. Key findings from the trial were presented at the 2025 Muscular Dystrophy Association meeting, with additional data published in Nature Medicine and JAMA Neurology.
“For nearly a decade, Sarepta has led the effort to transform the treatment landscape for Duchenne muscular dystrophy,” said Dr. Louise Rodino-Klapac, Sarepta’s chief scientific officer and head of R&D. “This approval gives new hope to young children and their families in Japan living with this devastating disease.”
The conditional approval allows marketing of ELEVIDYS in Japan for up to seven years, during which time further data collection will continue. Japan’s pathway is designed for innovative therapies targeting serious diseases with demonstrated early clinical benefit.
As part of a 2019 collaboration agreement, Roche is responsible for global regulatory approval and commercialization of ELEVIDYS outside the U.S. In Japan, commercialization will be managed by Chugai Pharmaceuticals, a member of the Roche Group. Sarepta retains responsibility for U.S. regulatory oversight and manufacturing.
The company is eligible to receive up to $103.5 million in near-term regulatory and commercial milestone payments under its agreements.
Duchenne muscular dystrophy affects approximately one in every 3,500 to 5,000 male births worldwide. The disease leads to progressive muscle wasting and typically results in loss of mobility and premature death. ELEVIDYS, a gene therapy designed to address the underlying genetic cause of DMD, offers a potential new avenue for extending and improving quality of life.
