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Roche Announces Positive Year-Two Results for Elevidys™ in Treating Duchenne Muscular Dystrophy

Phase III EMBARK trial demonstrates sustained motor function improvements with Elevidys gene therapy.

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  • Jan 27, 2025

  • Mrudula Kulkarni

Roche Announces Positive Year-Two Results for Elevidys™ in Treating Duchenne Muscular Dystrophy

January 27, 2025 Roche has reported positive topline results from year two of the EMBARK trial, a global Phase III study of Elevidys™ (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular dystrophy (DMD). The study revealed sustained and statistically significant improvements in motor function across three key measures—NSAA (North Star Ambulatory Assessment), TTR (Time to Rise), and 10MWR (10-Meter Walk/Run)—compared to a propensity-weighted untreated external control group.

Functional benefits for patients treated with Elevidys (n = 63) increased between the first and second years post-dosing, highlighting its potential to modify the disease course.

“These results underscore the transformative potential of Elevidys for young boys with Duchenne muscular dystrophy,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “The sustained improvements in standing, walking, and running represent meaningful progress in preserving mobility and independence.”

Clinical Insights and Next Steps
Duchenne muscular dystrophy is a progressive genetic disorder that leads to muscle degeneration and loss of function, significantly impacting quality of life. According to Professor Francesco Muntoni of the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital, UK, “The EMBARK trial’s year-two results suggest Elevidys may extend the period of mobility and independence for these children, reducing the physical and emotional burden on families.”

Detailed findings from the EMBARK study will be presented at an upcoming medical conference and submitted to regulatory authorities for further review. The study’s first-year data were published in Nature Medicine in October 2024.With Elevidys showing consistent, sustained benefits, Roche is advancing its commitment to innovative treatments that address significant unmet needs in neuromuscular disorders.

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