Sarepta Therapeutics Announces FDA Accelerated Approval of Elevidys to Treat Children with Duchenne Muscular Dystrophy

Through its Accelerated Approval Pathway, the Food and Drug Administration (FDA) has approved Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA) to treat pediatric patients with Duchenne Muscular Dystrophy (DMD) aged 4 to 5 years who are ambulatory and have a confirmed mutation in the DMD gene. The approval is based on data from a phase 1/2 clinical trial of 4 participants (NCT03375164), a phase 1 clinical trial of 46 participants called ENDEAVOR (NCT04626674), and a phase 2 clinical trial of 41 participants (NCT03769116).

Elevidys is a recombinant gene therapy injected as a single intravenous dose causing cells to produce a shortened protein called Elevidys micro-dystrophin which contains domains of the dystrophin protein found in healthy muscle cells. In DMD, a mutation results in absence of the dystrophin protein. Clinical trials demonstrated that Elevidys met the primary endpoint of increased skeletal muscular expression of micro-dystrophin, a reasonable predictor for clinical benefit. Common adverse events included vomiting, nausea, acute liver injury, fever, and thrombocytopenia. A global, randomized, double-blind, placebo-controlled phase 3 post-marketing confirmatory clinical trial of 126 participants, EMBARK (NCT05096221), is ongoing to examine the benefit of Elevidys for patients’ ambulation and functional skills.

“The approval of ELEVIDYS is a watershed moment for the treatment of Duchenne,” said Doug Ingram, President and CEO of Sarepta Therapeutics. “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.”

DMD is a progressive neuromuscular disorder associated with ambulatory difficulties, learning disabilities, fatigue, breathing problems, and issues with heart function. Symptom onset for DMD typically begins in childhood, with an average life expectancy of 26 years.