October 31, 2023
Roche's Elevidys Trial: Fails Primary Endpoint, Yet Demonstrates Functional Improvements in Duchenne Muscular Dystrophy
Roche presented the findings from the EMBARK trial, assessing Elevidys (delandistrogene moxeparvovec) for the treatment of boys with Duchenne muscular dystrophy (DMD). Despite Elevidys not meeting the primary endpoint of improvement in the North Star Ambulatory Assessment (NSAA), it demonstrated notable progress compared to the placebo after 52 weeks.
In individuals receiving Elevidys, the NSAA total score showed an improvement of 2.6 points, in contrast to the 1.9 points observed in those treated with the placebo over the 52 weeks. Additionally, the drug exhibited significant enhancements in secondary endpoints, such as time to rise, 10-metre walk, and time to ascend 4 steps.
Roche reported no new safety concerns regarding Elevidys. The company mentioned ongoing discussions with health authorities regarding the next steps for this treatment.