Elevidys (delandistrogene moxeparvovec-rokl) is an AAVrh74 vector-based gene therapy for Duchenne muscular dystrophy (DMD) with confirmed DMD mutations in eligible patients. FDA labeling was revised in November 2025 to limit indication to ambulatory patients aged 4 years and older and to add a boxed warning for serious liver injury and acute liver failure. The therapy uses a micro-dystrophin transgene approach.
Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
