UPDATE (3 November 2023):
A webinar regarding the EMBARK study results for families took place online via Zoom on 2 November. It was organised by the Northstar Network and support by us, Action Duchenne and Duchenne UK.
On Monday 30 October 2023, Sarepta Therapeutics announced the one-year results of part 1 of the EMBARK Phase 3 clinical trial. This multinational, randomised, controlled, and double-blind study aims to assess the safety and efficacy of delandistrogene moxeparvovec, a gene therapy now known as Elevidys (previously called SRP-9001) in boys aged four-seven with Duchenne muscular dystrophy (DMD).
The results show that the study failed to achieve its primary objective (also known as endpoint), which was to demonstrate a significant difference in the change of the North Star Ambulatory Assessment (NSAA) total score following Elevidys treatment. The NSAA is a recognised way of evaluating motor function (the ability to move) in people living with DMD. Although some improvement in motor function was observed in children treated with Elevidys compared to the placebo group, these differences were not statistically significant.
The study did successfully meet two of its secondary endpoints -the time required to rise from the floor and the 10-metre walk test. These secondary measures showed significant improvements in children who received Elevidys over the course of one year.
Additionally, there were no new safety concerns raised during this part of the study.
The study involved 125 boys with DMD who can walk unassisted, and who were randomly assigned to either the treatment group or the placebo group. Participants will continue to be closely monitored for the next five years for both safety and clinical outcomes.
Part 2 of the EMBARK Phase 3 study is currently ongoing and should finish late 2024, when it will provide further insights into the safety and efficacy of Elevidys in boys with DMD.
There will be a webinar for families in the coming days with members of the UK paediatric NorthStar network and other patient organisations – Action Duchenne and Duchenne UK. We will share more about the webinar when the details are known. We also remain in communication with Roche, Sarepta's global distribution partner, and will provide ongoing updates. In the meantime, you can read a community letter from Roche about the study and what it means for people in the UK.
Notably, Elevidys received what is known as accelerate approval for boys aged four-five in the United States earlier this year. The health authority in the UK responsible for issuing marketing authorisations (the licence) for medicines is the Medicines & Healthcare Products Regulatory Agency (MHRA), not the FDA. Roche is committed to working with the relevant agencies to determine the best possible route to achieving a potential licence for Elevidys.