Elevidys FDA Approval History
Last updated by Judith Stewart, BPharm on June 27, 2023.
FDA Approved: Yes (First approved June 22, 2023)
Brand name: Elevidys
Generic name: delandistrogene moxeparvovec-rokl
Dosage form: Suspension for Intravenous Infusion
Company: Sarepta Therapeutics, Inc.
Treatment for: Duchenne Muscular Dystrophy
Elevidys (delandistrogene moxeparvovec-rokl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
- This indication is approved under accelerated approval based on expression of Elevidys microdystrophin in skeletal muscle observed in patients treated with Elevidys. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
- Duchenne muscular dystrophy is a disease characterized by progressive muscle degeneration and weakness. DMD is caused by a mutation in the DMD gene that encodes instructions for a protein called dystrophin, which works to help keep muscle cells intact.
- Elevidys is a one-time, single-dose gene transfer therapy designed to treat the underlying cause of Duchenne muscular dystrophy by delivering a functional shortened dystrophin into the muscle tissue.
- Elevidys is administered as an intravenous infusion over 1-2 hours.
- Warnings and precautions associated with Elevidys include acute serious liver injury, immune-mediated myositis, and myocarditis.
- Common adverse reactions include vomiting and nausea, elevated liver enzymes or total bilirubin, pyrexia, and thrombocytopenia.
Development timeline for Elevidys
Further information
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