Sarepta Therapeutics announced that its gene‑therapy product ELEVIDYS (delandistrogene moxeparvovec) is now commercially available in Japan for children with Duchenne muscular dystrophy (DMD) aged 3 to less than 8 years. The launch follows the drug’s conditional approval by Japan’s Ministry of Health, Labour and Welfare in May 2025 and its listing on the National Health Insurance price list. Sales and post‑marketing activities are being handled by Chugai Pharmaceutical, a Roche Group company, allowing Sarepta to leverage Roche’s extensive distribution network while retaining manufacturing and regulatory oversight.
The launch is accompanied by a $40 million milestone payment that will be triggered upon the first commercial sale of ELEVIDYS in Japan. The therapy is priced at approximately 305 million yen (about $1.99 million USD), the highest price point under Japan’s reimbursement system, underscoring the company’s pricing power in a new market. Eligibility requires a deletion of any portion or the entirety of exon 8 or exon 9 in the DMD gene and a negative test for anti‑AAVrh74 antibodies, and the therapy is approved only for ambulatory patients.
Safety concerns that led the U.S. FDA to restrict ELEVIDYS to ambulatory patients aged 4 and older in November 2025—due to reports of serious liver injury and acute liver failure in non‑ambulatory patients—are reflected in the Japanese approval, which limits use to ambulatory children. The company remains focused on patient safety and will conduct post‑marketing studies and surveillance to confirm long‑term efficacy and safety.
Sarepta’s recent financial performance has been mixed. Q4 2025 preliminary net product revenue was $369.6 million, a 42% decline from Q4 2024’s $638.2 million, and the company reported $1.86 billion in total net product revenue for 2025. The $40 million milestone payment from the Japan launch will help offset the revenue shortfall and support ongoing development of its siRNA pipeline and other rare‑disease programs. The company is also pursuing a restructuring plan that aims to achieve approximately $400 million in annual cost savings by 2026, including a workforce reduction of about 500 employees.
"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 & Development and Technical Operations. "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." CEO Doug Ingram noted that "Sarepta faced and overcame challenges in 2025, achieving meaningful pipeline progress and solid performance," and added that the company "in the face of obstacles, we achieved $1.86 billion in total net product revenue, and ELEVIDYS achieved $898.7 million. At $110.4 million, ELEVIDYS fourth quarter revenue was impacted by the severe year‑end flu season and the need in December to reschedule 6 patient infusions into 2026." Chugai President and CEO Dr. Osamu Okuda said, "We are very pleased to be able to deliver ELEVIDYS to patients diagnosed with DMD and their families who have been eagerly awaiting new treatment options. Putting patient safety as our highest priority, we will work to drive proper use of Elevidys. We will also conduct post‑marketing clinical studies and all‑case post‑marketing surveillance to confirm the long‑term efficacy and safety of ELEVIDYS."
The Japan launch represents a significant tailwind for Sarepta’s global gene‑therapy strategy, expanding its commercial footprint into a large, high‑value market and providing a new revenue stream that will help sustain its pipeline development. The milestone payment and the record‑high pricing in Japan signal strong confidence from Japanese regulators and payers in the therapy’s safety and efficacy profile, despite the safety concerns that have limited its use in non‑ambulatory patients elsewhere. The launch also positions Sarepta to capture early‑intervention opportunities in a population that could benefit most from the therapy, reinforcing the commercial viability of its gene‑therapy platform and supporting the company’s broader rare‑disease and siRNA initiatives.
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