Roche Launches Phase 3 Trial for Elevidys in Europe

Roche announced on Thursday that it is launching a Phase 3 clinical trial for Elevidys, its gene therapy developed in partnership with U.S.-based Sarepta Therapeutics, to address concerns raised by European regulators and pave the way for approval in the region.

The move follows feedback from the European Medicines Agency, which last year declined to back the therapy for Duchenne muscular dystrophy after scrutiny linked to two patient deaths. Elevidys, designed to treat the rare genetic disorder that primarily affects boys and leads to progressive muscle weakening, requires additional placebo-controlled data to support a resubmission, according to company officials. The trial will involve approximately 100 early-stage ambulatory boys with a confirmed DMD mutation and span more than 72 weeks. Researchers will assess the therapy's efficacy and safety against a placebo, with the primary measure focusing on changes in the time required for participants to rise from the floor, a key indicator of motor function.

Duchenne muscular dystrophy impacts muscle strength over time, often leaving patients reliant on wheelchairs by their teens and shortening life expectancy. Elevidys has...