Sarepta Therapeutics developed a novel gene therapy called ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory paediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
ELEVIDYS, also known as delandistrogene moxeparvovec-rokl, is an innovative gene transfer therapy administered through a single intravenous infusion. It aims to target and address the root cause of Duchenne muscular dystrophy by facilitating the production of ELEVIDYS micro-dystrophin specifically within skeletal muscle tissue.
To assess its efficacy and safety, ELEVIDYS is currently undergoing evaluation in three ongoing clinical studies known as SRP-9001-101, SRP-9001-102, and SRP-9001-103.
In clinical studies, the most frequently reported adverse reactions were vomiting, nausea, elevated liver function test results, pyrexia (fever), and thrombocytopenia (low platelet count).
U.S. Food and Drug Administration (FDA) grants approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus based gene therapy for the treatment of Duchenne Muscular Dystrophy.