Sarepta Therapeutics and global partner, Roche, shared positive topline results from year two of the EMBARK trial, a phase 3 study of the approved gene therapy, Elevidys, for the treatment of individuals with Duchenne muscular dystrophy.
According to Sarepta, two years after treatment with Elevidys, statistically significant and clinically meaningful improvements were observed across three key motor function measures — North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR) — when compared to an untreated external control group.
Individuals who received a placebo in part one of the EMBARK study (patients ages 4-7 years) crossed over at 52 weeks and were treated with Elevidys in part two (patients ages 5-9 years). Despite being one year older than those treated in part one, the crossover treated group experienced similar changes 52 weeks after treatment, favoring Elevidys compared to the external control.
Importantly, no new safety signals were observed, reinforcing the consistent and manageable safety profile of Elevidys to date.
Elevidys, an in vivo AAV-based gene therapy, was first approved under the accelerated approval pathway in June 2023 for a segment of pediatric patients with DMD — specifically, ambulatory pediatric patients aged 4-5 years who have a confirmed mutation in the DMD gene. A year later, the FDA expanded the approval to include the treatment of Duchenne for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. At the same time, the agency granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients.
As part of a collaboration agreement signed in 2019, Sarepta is responsible for regulatory approval, commercialization and manufacturing of Elevidys in the U.S. Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.
Detailed results from year two of the EMBARK study will be shared at an upcoming medical meeting and discussed with health authorities.
Subscribe to our e-Newsletters
Stay up to date with news, articles and insights relevant to cell and gene therapy development and manufacturing. Plus, get special offers from Cell & Gene Therapy Review delivered right to your inbox!
Sign up now!