Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years

Sarepta Therapeutics, a leader in precision genetic therapies for rare diseases, announced the commercial launch of ELEVIDYS (delandistrogene moxeparvovec) in Japan through Chugai Pharmaceutical following its listing on Japan’s National Health Insurance price schedule. ELEVIDYS is the first gene therapy approved in Japan for Duchenne muscular dystrophy and is available for ambulatory patients aged 3 to under 8 with a deletion in exon 8 or 9 of the DMD gene and negative for anti-AAVrh74 antibodies.

Louise Rodino-Klapac, Ph.D., President of Research & Development and Technical Operations at Sarepta, said the launch represents a major step in expanding access to early intervention for Duchenne, which may help preserve muscle function. She emphasized that the company remains committed to advancing scientific research and generating long-term clinical and real-world evidence to guide care for the global Duchenne community.

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Chugai Pharmaceutical will handle postmarketing studies and surveillance as part of its collaboration with Roche, following the conditional approval granted by Japan’s Ministry of Health, Labour and Welfare in May 2025. The approval was based on data from the ELEVIDYS clinical program, including results from the global Phase 3 EMBARK study, which demonstrated meaningful improvements in motor function among ambulatory boys with Duchenne. Longer-term follow-up shows sustained efficacy and slower disease progression compared to external controls, with no new safety concerns observed. To date, ELEVIDYS has been administered to over 1,200 patients worldwide.

Under a collaboration agreement signed in 2019, Sarepta manages regulatory approvals and commercialization in the U.S. while Roche oversees territories outside the U.S., including Japan. The first commercial sale in Japan will trigger a milestone payment of $40 million to Sarepta. ELEVIDYS is a single-dose, adeno-associated virus-based gene therapy designed to address the underlying genetic cause of Duchenne by delivering a transgene that produces micro-dystrophin in skeletal muscles. In the United States, it is indicated for ambulatory patients aged 4 and older with confirmed DMD gene mutations.

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