FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy.- Sarepta Therapeutics.
Sarepta Therapeutics, Inc. the leader in precision genetic medicine for rare diseases, announced FDA accelerated approval of Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus based gene therapy 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 micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. This accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle. Elevidys is supported by biologic and empirical evidence, in addition to efficacy data from two clinical studies: SRP-9001-102 and SRP-9001-103 and safety data from SRP-9001-101, SRP-9001-102 and SRP-9001-103. Acute serious liver injury, immune-mediated myositis and myocarditis have occurred in patients treated with Elevidys.
The most common adverse reactions in clinical studies were vomiting, nausea, liver function test increased, pyrexia and thrombocytopenia.
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