Chugai Pharmaceutical has launched Elevidys Intravenous Infusion (delandistrogene moxeparvovec) in Japan following its conditional and time-limited approval on May 13, 2025, and inclusion in the National Health Insurance (NHI) reimbursement price list. The therapy has been approved as a regenerative medical product for the treatment of Duchenne Muscular Dystrophy (DMD).

Elevidys is indicated for ambulatory patients aged three to less than eight years who do not have deletions in exon 8 and/or exon 9 of the DMD gene and who test negative for anti-AAVrh74 antibodies.

DMD is a rare, progressive genetic disorder that begins in early childhood and leads to worsening muscle weakness, loss of ambulation and eventual respiratory and cardiac complications. The disease primarily affects boys, with approximately one in 5,000 male births worldwide diagnosed with DMD.

Chugai’s President and CEO, Dr. Osamu Okuda, said the company is committed to ensuring patient safety while delivering a new treatment option to families affected by DMD. He added that post-marketing clinical studies and all-case surveillance will be conducted to monitor long-term efficacy and safety.

The approval is supported by data from multiple clinical studies, including the global Phase III EMBARK trial. The randomised, double-blind, placebo-controlled study evaluated Elevidys in ambulatory boys aged four to seven years with confirmed DMD mutations. While the primary endpoint—change in motor function measured by the North Star Ambulatory Assessment (NSAA) at 52 weeks—did not reach statistical significance compared to placebo, clinically meaningful improvements were observed in several key secondary endpoints. These included time to rise from the floor, 10-metre walk time, stride velocity (SV95C), and time to ascend four steps. The trial remains ongoing, with three-year follow-up data available for Part 1 participants.

Elevidys is designed to address the underlying cause of DMD by delivering a gene therapy that enables expression of a shortened dystrophin protein in skeletal, respiratory and cardiac muscle. The therapy received orphan regenerative medical product designation in Japan.

Following reports of two fatal cases of acute liver failure in non-ambulatory DMD patients treated overseas, Chugai has strengthened safety measures. These include revising prescribing information, publishing updated educational materials, establishing a specialist consultation framework in collaboration with the Japanese Society of Child Neurology and implementing an industry-government-academia framework to promote appropriate use.

To determine eligibility prior to treatment, patients must test negative for anti-AAVrh74 antibodies using the Elecsys anti-AAVrh74 assay, launched in Japan by Roche Diagnostics K.K. as a companion diagnostic and listed on the NHI reimbursement schedule in February 2026.

Elevidys was originally developed by Sarepta Therapeutics and co-developed with Roche. Chugai has in-licensed the therapy from Roche and holds exclusive marketing rights in Japan.

In the United States, Elevidys received approval in June 2023 as the first gene therapy for DMD, though its indication for non-ambulatory patients was later removed following safety concerns. In Europe, the European Commission declined conditional marketing approval in September 2025. The therapy is currently approved for ambulatory DMD patients in nine countries worldwide.