Sarepta Therapeutics has received approval from Japan’s Ministry of Health, Labour, and Welfare (MHLW) for its gene therapy, Elevidys (delandistrogene moxeparvovec), targeting Duchenne muscular dystrophy (DMD).
The approval, granted under a conditional and time-limited framework, applies to children aged three to under eight years, excluding those with deletions in exon 8 and/or exon 9 of the DMD gene and those with anti-AAVrh74 antibodies. This decision marks the first instance globally where approval includes patients younger than four years, said Sarepta.
The decision relied on data from Elevidys clinical studies, including results from the global Phase 3 EMBARK trial (Study SRP-9001-301). Over a two-year period, patients treated with Elevidys demonstrated improved motor function compared to a matched external control group, with no new safety issues reported.
Findings from Part 2 of the EMBARK study were shared at the 2025 Muscular Dystrophy Association meeting. Part 1 results were published in Nature Medicine in October 2024, and quantitative muscle MR outcomes appeared in JAMA Neurology in May 2025.
Sarepta R&D head and chief scientific officer Louise Rodino-Klapac said: “For nearly a decade, Sarepta has been a leader in advancing the treatment of Duchenne muscular dystrophy through innovative treatment options.
“With the approval of Elevidys in Japan, children who are living with this rare, fatal disease, one marked by progressive muscle deterioration and weakness, now have an additional treatment option.
“Sarepta is committed to supporting our partners so we can bring this treatment to additional people with Duchenne around the world who urgently need a therapy that potentially addresses the root cause of disease.”
Japan’s conditional and time-limited approval pathway allows for the marketing of innovative treatments for serious diseases for up to seven years, contingent on early clinical trial results showing significant efficacy and safety.
Sarepta and Roche entered a collaboration in 2019 to enhance treatment options for the Duchenne community. Sarepta manages regulatory approval and commercialisation in the US and oversees manufacturing, while Roche is responsible for regulatory processes and distribution internationally.
Chugai Pharmaceuticals, in partnership with Roche, will handle the commercialisation of Elevidys in Japan.
