April 16, 2026 —
Roche has announced the initiation of a new global Phase III clinical trial for its gene therapy Elevidys, marking a renewed effort to secure European approval following a prior rejection by the European Medicines Agency. The company is aiming to address regulatory concerns through a rigorous placebo-controlled study rather than relying on patient advocacy alone.
The EMA previously declined to support approval of Elevidys for ambulatory patients with Duchenne muscular dystrophy (DMD), citing insufficient statistical significance in functional outcomes. Specifically, data from earlier studies showed only a modest improvement in North Star Ambulatory Assessment (NSAA) scores compared to placebo, which did not meet regulatory thresholds.
Elevidys is currently approved in the United States and several other countries for ambulatory boys with confirmed DMD mutations, with commercialization led by Sarepta Therapeutics. However, its clinical profile has been subject to ongoing debate. The pivotal Phase III EMBARK study failed to meet its primary endpoint at one year, although longer-term follow-up data suggested potential functional benefits when compared to external control cohorts.
The newly announced Phase III trial will enroll approximately 100 ambulatory boys and evaluate Elevidys against placebo over a 72-week period. The primary endpoint will focus on change in “time to rise” from the floor, a clinically meaningful functional measure associated with disease progression. Patients initially assigned to placebo will have the opportunity to receive Elevidys after the primary study period, a design element intended to support recruitment and retention.
Safety remains a key consideration in Elevidys’ development. Reports of serious adverse events, including liver toxicity and patient deaths in non-ambulatory populations, prompted regulatory scrutiny and led to restrictions in certain markets. As a result, Roche is currently prioritizing development in the ambulatory population, where the benefit-risk profile is considered more favorable.
Despite earlier setbacks, Roche maintains confidence in Elevidys, citing long-term data and real-world treatment experience in over a thousand patients globally. The outcome of this new Phase III study will be critical in determining whether the therapy can meet European regulatory standards and expand access to patients with this progressive and life-limiting disease.
