Sarepta Therapeutics’ Delandistrogene Moxeparvovec Receives Conditional Approval in Japan for Younger Duchenne Patients

CAMBRIDGE, Mass. – May 13, 2025 – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leading precision genetic medicine company focused on rare diseases, today announced that the Japanese Ministry of Health, Labour, and Welfare (MHLW) has granted conditional and time-limited approval for delandistrogene moxeparvovec (marketed as ELEVIDYS) for the treatment of Duchenne muscular dystrophy (DMD) in Japan.

The approval applies to individuals aged 3 to less than 8 years old who do not have deletions in exon 8 and/or exon 9 of the DMD gene and who test negative for anti-AAVrh74 antibodies. Notably, this marks the first global approval for ELEVIDYS to include children younger than 4 years of age.

The MHLW’s decision is based on efficacy and safety data from ELEVIDYS clinical programs, including two-year functional outcome data from the global Phase 3 EMBARK clinical trial (Study SRP-9001-301). These longer-term results demonstrated that ELEVIDYS-treated individuals experienced significantly better outcomes across multiple motor function measures compared to a well-matched external control group. No new safety signals were observed in the EMBARK study over the two-year period. These findings were recently presented at the 2025 Muscular Dystrophy Association meeting. Additionally, one-year data and quantitative muscle MRI outcomes from Part 1 of the EMBARK study were published in Nature Medicine (October 2024) and JAMA Neurology (May 2025), respectively.

Louise Rodino-Klapac, Sarepta’s head of research and development and chief scientific officer, emphasized Sarepta’s decade-long commitment to advancing DMD treatment and highlighted that this approval provides an additional treatment option for young children in Japan living with this severe and progressive muscle-wasting disease. She also noted Sarepta’s commitment to supporting their partners to bring this potential disease-modifying therapy to more patients worldwide.

The conditional and time-limited approval pathway in Japan allows for marketing authorization for up to seven years for innovative medicines treating serious conditions, provided certain criteria, including promising early clinical trial efficacy and safety data, are met.

Sarepta is collaborating with Roche on the global development and commercialization of ELEVIDYS. Sarepta is responsible for regulatory approval and commercialization in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and distribution in the rest of the world, with commercialization in Japan being handled by Chugai Pharmaceuticals through their alliance with Roche.