Elevidys FDA Approval History
Last updated by Judith Stewart, BPharm on June 24, 2024.
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 for the treatment of patients at least 4 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
- Elevidys is indicated in individuals at least 4 years of age:
- for the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene.
- for the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene.
The DMD indication in non-ambulatory patients 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 infusion-related reactions, acute serious liver injury, immune-mediated myositis, and myocarditis.
- Common adverse reactions include vomiting and nausea, liver injury, pyrexia, and thrombocytopenia.
Development timeline for Elevidys
Further information
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.