| Clinical data | |
|---|---|
| Trade names | Elevidys |
| Other names | SRP-9001, delandistrogene moxeparvovec-rokl |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a623058 |
| License data | |
| Routes of administration | Intravenous infusion |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy.[3] It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells.[3] It is an adeno-associated virus vector-based gene therapy that is given by intravenous infusion (slow injection into a vein).[1]
The most commonly reported side effects include vomiting, nausea, acute liver injury, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood).[3]
Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023.[3][4] It was developed by Sarepta Therapeutics, together with Roche, and is manufactured by Catalent.[5]
- ^ a b "Elevidys- delandistrogene moxeparvovec-rokl kit". DailyMed. 22 June 2023. Archived from the original on 29 August 2023. Retrieved 29 August 2023.
- ^ "Elevidys". U.S. Food and Drug Administration (FDA). 22 June 2023. Archived from the original on 5 July 2023. Retrieved 22 June 2023.
- ^ a b c d "FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy" (Press release). U.S. Food and Drug Administration (FDA). 22 June 2023. Archived from the original on 29 November 2023. Retrieved 22 June 2023.
This article incorporates text from this source, which is in the public domain.
- ^ "Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy" (Press release). Sarepta Therapeutics. 22 June 2023. Archived from the original on 23 June 2023. Retrieved 22 June 2023 – via Business Wire.
- ^ "Catalent inks deal to manufacture Sarepta's DMD gene therapy". Archived from the original on 17 February 2023. Retrieved 23 June 2023.
