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Roche has announced positive results from the Phase III EMBARK clinical trial of Elevidys (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD). Following two years of treatment with Elevidys, patients experienced a statistically significant and clinically meaningful improvement compared to an external control group across three key functional outcomes: North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR).1
Meanwhile, individuals treated with Elevidys in the first part of the EMBARK trial showed clinically meaningful and statistically significant improvements on the NSAA were sustained two years after treatment.
In a press release, Levi Garraway, MD, PhD, chief medical officer and head of global product development, Roche said, “After two years of treatment with Elevidys, we are seeing multiple sustained benefits in the day-to-day lives of these young boys, all of which are indicators of its disease modifying potential in Duchenne. These results, which include improvements in standing, walking and running, represent meaningful progress and we plan to share them with health authorities as quickly as possible.”
The Phase III EMBARK trial is a multinational, randomized, double-blind, two-part crossover, placebo-controlled study. In part one, participants were randomized according to age (4-5 or 6-7 years) or NSAA total score at screening (≤22 or >22) to receive either Elevidys or placebo, with a follow-up period for 52 weeks. In the second part, participants who were previously treated with placebo in part one received Elevidys and participants who were previously treated with Elevidys received placebo, with another follow-up period for 52 weeks. The primary endpoint was change from baseline in NSAA total score at week 52.
“As Duchenne progresses, children will lose the ability to walk, have difficulty breathing, and develop heart problems, all of which severely impact their health and ability to fully participate in life,” Professor Francesco Muntoni, director of Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, UK, said in the press release. “Encouraging results from year two of the EMBARK trial suggest that with innovative treatments like Elevidys, the period of mobility and independence can potentially be improved, reducing the physical and emotional challenge Duchenne poses for these young boys and their families.”
In June 2024, Elevidys received traditional FDA approval for ambulatory DMD patients and accelerated approval for non-ambulatory DMD patients. Under a collaboration agreement between Roche and Sarepta signed in 2019, Sarepta is responsible for the regulatory approval, commercialization, and manufacturing of Elevidys in the United States, while Roche is responsible for regulatory approvals and brining the therapy to patients across the rest of the globe.2
In a press release from the time, Doug Ingram, president and chief executive officer, Sarepta said, “Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science.”
References
1. Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD). News release. Roche. January 26, 2025. Accessed January 28, 2025. https://www.roche.com/media/releases/med-cor-2025-01-27
2. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. News release. Sarepta. June 20, 2024. Accessed January 28, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys