for those following srpt:
Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy
BUSINESS WIRE
May-13-2025 9:25 a.m. ET
- Approval based on efficacy and safety results from clinical studies, including longer-term functional outcomes from EMBARK global phase 3 study
- Company is eligible to receive up to $103.5M in near-term regulatory and commercial milestone payments
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (SRPT) , the leader in precision genetic medicine for rare diseases, today announced that the Japanese Ministry of Health, Labour, and Welfare (MHLW) has approved ELEVIDYS (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway in Japan. ELEVIDYS is approved for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon 8 and/or exon 9 in the DMD gene, and who are negative for anti-AAVrh74 antibodies. This is the first global approval to include individuals younger than 4 years of age.
The approval is based on the efficacy and safety data for ELEVIDYS, which includes muscle health and longer-term functional results from the ELEVIDYS clinical programs, including the two-year data from the global Phase 3 EMBARK clinical trial (Study SRP-9001-301). These longer-term functional outcomes showed that individuals treated with ELEVIDYS had significantly better outcomes in 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 duration. These findings from Part 2 of EMBARK were presented at the 2025 Muscular Dystrophy Association meeting. In addition, one-year data from part 1 of the EMBARK study were published in Nature Medicine in October 2024 and quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025.
“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,” said Louise Rodino-Klapac, Sarepta’s head of research and development and chief scientific officer. “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.”
The conditional and time-limited approval pathway in Japan provides for marketing authorization in Japan for up to seven years for innovative medicines to treat serious conditions. To be eligible for the pathway, certain additional criteria must also be met, including early clinical trial results that have demonstrated significant efficacy and safety.
As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche. |
