CAMBRIDGE, Mass. — Sarepta Therapeutics said ELEVIDYS, its gene therapy for Duchenne muscular dystrophy, has been commercially launched in Japan through partner Chugai Pharmaceutical following reimbursement approval under Japan’s National Health Insurance program.
The launch makes ELEVIDYS available in Japan to ambulatory children with Duchenne muscular dystrophy between the ages of 3 and less than 8, meeting specific genetic and antibody criteria. It is the first gene therapy approved and launched in Japan for Duchenne muscular dystrophy.
“The commercial launch of ELEVIDYS in Japan marks an important step in expanding access to this therapy, where early intervention may offer the greatest opportunity to preserve muscle function,” said Louise Rodino-Klapac, Ph.D., president of research and development and technical operations at Sarepta. “We are delighted that ELEVIDYS is now available to eligible patients in Japan and remain focused on advancing rigorous science and generating long-term clinical and real-world evidence to help inform care for the Duchenne community worldwide.”
Chugai announced the launch following the therapy’s reimbursement listing, which enables access under a conditional and time-limited approval granted by Japan’s Ministry of Health, Labour and Welfare in May 2025. As part of the approval, Chugai will oversee postmarketing clinical studies and conduct all required postmarketing surveillance in Japan under the broader Roche Group collaboration.
Japan’s regulatory decision was based on data from the ELEVIDYS clinical development program, including results from the global Phase 3 EMBARK study. The study evaluated the therapy in ambulatory boys with Duchenne muscular dystrophy and showed clinically meaningful improvements in key motor function measures. Longer-term follow-up data indicated sustained functional benefits and slower disease progression compared with external controls, with no new treatment-related safety signals observed in ambulatory patients. Two-year EMBARK results were published in Neurology & Therapy in January, and three-year data are expected to be presented at an upcoming medical meeting. To date, more than 1,200 patients worldwide have received ELEVIDYS across clinical trials and real-world use.
Sarepta is collaborating with Roche under an agreement signed in 2019 aimed at expanding treatment options for the Duchenne community. Under the collaboration, Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the United States, as well as manufacturing, while Roche handles regulatory approvals and commercialization outside the U.S. In Japan, commercialization is being carried out by Chugai Pharmaceutical, a member of the Roche Group.
Under the terms of the collaboration, the first commercial sale of ELEVIDYS in Japan will trigger a $40 million milestone payment to Sarepta.
ELEVIDYS is a single-dose, adeno-associated virus-based gene transfer therapy administered by intravenous infusion. It is designed to address the underlying genetic cause of Duchenne muscular dystrophy by delivering a transgene that enables targeted production of micro-dystrophin in skeletal muscle. In the United States, ELEVIDYS is approved for the treatment of ambulatory patients aged 4 and older with Duchenne muscular dystrophy who have a confirmed mutation in the DMD gene.
