Roche announced on Thursday that it will commence a late-stage clinical trial for its gene therapy Elevidys after receiving feedback from European drug regulators. The therapy, developed in collaboration with U.S.-based Sarepta Therapeutics, is intended for the treatment of Duchenne muscular dystrophy (DMD). The drug failed to gain approval from the European Medicines Agency last year, following scrutiny linked to the deaths of two patients.

The Phase III trial is designed to supplement placebo-controlled data requested by the European Medicines Agency to support a resubmission of the marketing application. Elevidys is currently approved in nine countries, including the United States, for ambulatory boys confirmed to have a DMD gene mutation. The U.S. had temporarily paused shipments of the therapy in July of last year.

Duchenne muscular dystrophy is a rare genetic disorder that primarily affects young males and leads to progressive muscle weakening over time. The study will last more than 72 weeks and will evaluate the efficacy and safety of Elevidys compared to a placebo in approximately 100 ambulatory boys with early-stage DMD. The primary endpoint will be the change in the time taken for subjects to stand up from the floor.

Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development, stated, "Our confidence is supported by substantial long-term data confirming the durable efficacy and safety of Elevidys, as well as our experience in treating over a thousand ambulatory boys globally."