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Elevidys is a prescription AAVrh74 vector-based gene therapy containing delandistrogene moxeparvovec-rokl. Current U.S. labeling limits treatment to ambulatory patients age 4 and older with Duchenne muscular dystrophy and a confirmed mutation in the DMD gene. It is administered once by intravenous infusion.
read more about elevidys ...Elevidys carries a transgene that encodes an engineered micro-dystrophin protein. The AAVrh74 vector delivers this genetic material to muscle cells, while the MHCK7 regulatory component directs micro-dystrophin expression mainly in skeletal and cardiac muscle. The resulting protein is a shortened form of dystrophin containing selected functional domains.
Elevidys is indicated for ambulatory patients age 4 and older with Duchenne muscular dystrophy and a confirmed DMD gene mutation. Treatment is contraindicated when the DMD mutation includes any deletion involving exon 8 or exon 9. Patients also undergo testing for anti-AAVrh74 antibodies before infusion.
Elevidys is given once by intravenous infusion. Patients weighing less than 70 kg receive 1.33 ? 10^14 vector genomes per kg, equal to 10 mL per kg. Patients weighing 70 kg or more receive a fixed total dose of 9.31 ? 10^15 vector genomes. Systemic corticosteroids are given before and after infusion.
Elevidys carries a boxed warning for acute serious liver injury and acute liver failure, including fatal cases. Other serious concerns include infections, myocarditis, infusion reactions, and immune-mediated myositis. Liver tests are checked weekly for at least three months, and troponin-I is checked weekly for the first month after treatment.
Elevidys treats Duchenne muscular dystrophy in ambulatory patients age 4 and older with a confirmed DMD gene mutation.
Elevidys is administered as a single intravenous infusion and is not re-administered.
Yes. The boxed warning covers acute serious liver injury and acute liver failure, including fatal outcomes.
Patients with DMD deletions involving exon 8 or exon 9 should not receive Elevidys under current labeling.
Delandistrogene Moxeparvovec. https://pubmed.ncbi.nlm.nih.gov/40997205/. Date Accessed September 29, 2026.
Delandistrogene Moxeparvovec-rokl Injection: MedlinePlus Drug Information. https://medlineplus.gov/druginfo/meds/a623058.html. Date Accessed September 29, 2026.
ELEVIDYS. https://www.fda.gov/vaccines-blood-biologics/tissue-tissue-products/elevidys. Date Accessed September 29, 2026.
ELEVIDYS Prescribing Information. https://www.fda.gov/media/184855/download?attachment=. Date Accessed September 29, 2026.
FDA Takes Action on New Boxed Warning for Acute Serious Liver Injury and Acute Liver Failure Following Treatment with Elevidys and Revised Indication that Is Limited to Ambulatory Duchenne Muscular Dystrophy Patients. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-takes-action-new-boxed-warning-acute-serious-liver-injury-and-acute-liver-failure-following. Date Accessed September 29, 2026.