Clinical data | |
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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 | |
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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 and is manufactured by Catalent.[5]