Sarepta announces FDA approval of Elevidys gene therapy for DMD

Sarepta Therapeutics (SRPT) announced U.S. Food and Drug Administration accelerated approval of Elevidys, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy, or 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. “Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial. EMBARK, the global, randomized, double-blind, placebo-controlled Phase 3 trial for Elevidys, will serve as the post-marketing confirmatory trial and is fully enrolled with top-line results expected in late 2023,” the company stated. “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,” said Doug Ingram, president and CEO of Sarepta. Sarepta is responsible for global development and manufacturing for Elevidys, and distribution within the U.S. will commence immediately. In December 2019, Sarepta partnered with Roche (RHHBY) to accelerate access to Elevidys for patients outside the United States.