Also known as: Elevidys, ELEVIDYS
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.
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Mesoblast received FDA clearance for a registrational DMD trial of remestemcel-L-rknd, while Satellos began dosing boys in the Phase 2 BASECAMP study of SAT-3247. The studies are enrolling children ages 5-9 and 7-9, respectively.
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07542314 |
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting |
NOT_YET_RECRUITING | PHASE4 |
