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Roche: Topline Results from the Phase 3 Study of Elvidys Gene Therapy

October 31, 2023

Roche has announced topline results from the global, randomized, double-blind Phase 3 EMBARK study of Elevidys, a micro-dystrophin gene therapy, in ambulatory boys with Duchenne muscular dystrophy aged 4-7 years.

While the EMBARK trial did not meet its primary outcome of change from baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52, all key secondary outcomes did show statistical significance in favour of treatment, with no new safety signals.

  • Elevidys-treated boys aged 4-7 years with Duchenne showed an increase on the NSAA, a measure of motor function, compared to the placebo at 52 weeks, but the primary endpoint was not met.
  • For all key pre-specified secondary functional endpoints, time to rise and 10-metre walk test across age groups, clinically meaningful and statistically significant treatment benefits were observed.
  • No new safety signals were observed, reinforcing the favourable and manageable safety profile observed with Elevidys to date.
  • Further data evaluation is ongoing, and Roche will discuss the path forward with health authorities.

“High unmet need remains in Duchenne, and we are encouraged by the consistent and meaningful results seen in all key secondary functional endpoints for Elevidys, an innovative gene therapy. We will work to further analyse the EMBARK results and consult with health authorities as quickly as possible. We sincerely thank all the boys, their families and the wider Duchenne community involved in this important research effort.”

Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche

As part of a collaboration agreement, Roche is working with Sarepta Therapeutics to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function, keeping them stronger for longer. Sarepta is responsible for managing regulatory approval and the commercialization of Elevidys in the US. Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world, including Canada. Sarepta is responsible for the manufacturing of Elevidys, and together, the companies are working on a comprehensive joint clinical development plan to maximize the chances of broad approval and access.

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