Source – Sarepta Therapeutics
On June 22, 2023, Sarepta Therapeutics made an announcement regarding the accelerated approval granted by the US Food and Drug Administration (FDA) for Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy based on adeno-associated virus, to treat ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. This approval was granted based on the expression of Elevidys micro-dystrophin observed in patients who received the treatment. However, further verification and demonstration of clinical benefit in confirmatory trials are required to maintain the approval for this indication. It is important to note that Elevidys should not be administered to patients with deletions in exon 8 and/or exon 9 of the DMD gene.
Elevidys aims to address the root cause of Duchenne muscular dystrophy, which is mutations in the dystrophin gene resulting in the absence of dystrophin protein. The therapy delivers a gene that encodes a shortened version of dystrophin, known as Elevidys micro-dystrophin, to muscle cells. The accelerated approval was based on increased expression of Elevidys micro-dystrophin protein in skeletal muscles. The approval was supported by biological and empirical evidence, as well as efficacy data from two clinical studies (SRP-9001-102 and SRP-9001-103) and safety data from three studies (SRP-9001-101, SRP-9001-102, and SRP-9001-103). Adverse reactions observed in clinical studies included vomiting, nausea, increased liver function tests, fever, and decreased platelet count.
Related: Sarepta’s Breakthrough DMD Gene Treatment, Elevidys, Triumphs FDA Hurdles at $3.2M
As part of the accelerated approval pathway, Sarepta Therapeutics has committed to conducting a confirmatory trial called EMBARK. This global Phase III trial is randomized, double-blind, and placebo-controlled, and it is fully enrolled. The top-line results from this trial are expected in late 2023.
“Duchenne is a relentlessly progressive, degenerative disease, robbing children of muscle function. The increases in Elevidys dystrophin expression and the functional results that we see can make a difference in the lives of our patients.”
– Jerry Mendell, M.D., pediatric neurologist and principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital
“The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevidys is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease. As we prepare to launch Elevidys, we should acknowledge and celebrate the decades of dedication and work from the patient community, families, clinicians, and our Sarepta colleagues that resulted in today’s approval. Our confirmatory trial, EMBARK, should read out in the fourth quarter of this year. If EMBARK confirms the benefits seen in our prior trials, Sarepta will move rapidly to submit a BLA supplement to expand the approved label as broadly as good science permits.”
– Doug Ingram, president and chief executive officer, Sarepta
