Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a leader in precision genetic medicine for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. This approval is based on the observed expression of ELEVIDYS micro-dystrophin in patients treated with the gene therapy.
ELEVIDYS addresses the underlying genetic cause of Duchenne muscular dystrophy by delivering a shortened form of the dystrophin gene, known as ELEVIDYS micro-dystrophin, to muscle cells. The therapy aims to compensate for the lack of dystrophin protein caused by mutations in the dystrophin gene.
It is important to note that ELEVIDYS is contraindicated in patients with specific deletions in exon 8 and/or exon 9 of the DMD gene. Adverse reactions reported in clinical studies include vomiting, nausea, increased liver function test results, fever, and thrombocytopenia. Serious liver injury, immune-mediated myositis, and myocarditis have also occurred in patients treated with ELEVIDYS.
Sarepta Therapeutics has committed to conducting a confirmatory trial called EMBARK, which is a global Phase 3 trial. This trial, a randomized, double-blind, placebo-controlled study, is fully enrolled, and top-line results are expected in late 2023.
The approval of ELEVIDYS is a significant milestone in the treatment of Duchenne muscular dystrophy, as it is the first gene therapy approved for this condition. Sarepta Therapeutics aims to provide a treatment that has the potential to alter the trajectory of this degenerative disease. ELEVIDYS is administered as a single-dose gene transfer therapy via intravenous infusion and has been evaluated in multiple clinical studies, with safety data collected from numerous treated patients.