Sarepta's ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Approved in Japan

Sarepta Therapeutics, Inc., a leader in precision genetic medicine, has announced a significant development in the treatment of Duchenne muscular dystrophy (DMD). The Japanese Ministry of Health, Labour, and Welfare (MHLW) has granted approval for ELEVIDYS (delandistrogene moxeparvovec) under a conditional and time-limited approval pathway. This decision marks the first global approval to encompass children as young as three, extending up to those nearly eight years old. The treatment is specifically aimed at patients who do not have deletions in exon 8 and/or exon 9 of the DMD gene and are negative for anti-AAVrh74 antibodies.

ELEVIDYS has been approved based on its demonstrated efficacy and safety, supported by data from extensive clinical studies. Notably, the approval is underpinned by results from the EMBARK global Phase 3 study (Study SRP-9001-301), which involved two years of monitoring. The study revealed substantial improvements in motor function among participants treated with ELEVIDYS compared to a well-matched control group. Importantly, no new safety concerns emerged during the study period. These promising outcomes were showcased at the 2025 Muscular Dystrophy Association meeting, further emphasizing the potential of ELEVIDYS as a viable treatment option.

Louise Rodino-Klapac, Sarepta's head of research and development, expressed optimism over the approval. She highlighted Sarepta's dedication to developing advanced treatments for DMD, a condition characterized by progressive muscle weakening. According to Rodino-Klapac, the approval of ELEVIDYS in Japan offers new hope for affected children and reaffirms Sarepta's commitment to expanding access to this treatment globally.

Japan's conditional and time-limited approval pathway provides market authorization for innovative therapies addressing severe medical conditions for up to seven years. Eligibility for this pathway requires early clinical trial outcomes demonstrating significant efficacy and safety.

Sarepta's collaboration with Roche, initiated in 2019, aims to transform the outlook for the DMD community by preserving muscle function in those affected by the disease. Under this partnership, Sarepta oversees the regulatory approval and commercialization of ELEVIDYS in the United States, while Roche is responsible for the rest of the world. In Japan, ELEVIDYS will be commercialized through Chugai Pharmaceuticals, aligning with Roche's global strategy.

ELEVIDYS is an adeno-associated virus (AAV)-based gene therapy delivered via a single intravenous infusion. It targets the genetic root of DMD by introducing a transgene that promotes the production of micro-dystrophin in skeletal muscle. In the U.S., ELEVIDYS is approved for treating DMD in individuals aged four and older, including both ambulatory and non-ambulatory patients with confirmed mutations in the DMD gene. The approval for non-ambulatory patients is granted under accelerated approval, contingent on confirming clinical benefits in further trials.

The safety profile of ELEVIDYS has been thoroughly evaluated. It is contraindicated for patients with deletions in exon 8 and/or exon 9 of the DMD gene. Various precautions have been outlined, including monitoring for infusion-related reactions, liver injury, immune-mediated myositis, myocarditis, and pre-existing immunity against AAVrh74. Common adverse reactions reported include vomiting, nausea, liver injury, fever, and thrombocytopenia.

Sarepta Therapeutics continues to lead efforts in genetic medicine for rare diseases, focusing on Duchenne muscular dystrophy and limb-girdle muscular dystrophies. The company is rapidly advancing its portfolio across muscle, central nervous system, and cardiac diseases, striving to change the trajectory of these conditions through innovative therapies.