Roche Restarts Phase III Trial for Elevidys to Secure EMA Approval
New Delhi: Roche on Thursday said it is initiating a late stage study for its gene therapy Elevidys following feedback from the European drug regulator. The therapy for duchenne muscular dystrophy (DMD), for which Roche partners with American Sarepta Therapeutics had failed to win backing from the European Medicines Agency (EMA) last year after coming under scrutiny following two patient deaths tied to the treatment.
- The phase III study aims to generate additional placebo-controlled data required for a regulatory re-submission with EMA.
- Elevidys is currently approved in nine countries for ambulatory boys with a confirmed DMD mutation, including in the United States, which had temporarily ordered a pause in shipments last July.
- DMD is a rare, inherited disease that mostly affects boys and causes muscles to weaken steadily over time.
- The study will span more than 72 weeks and evaluate the efficacy and safety of Elevidys compared to placebo in around 100 early ambulatory boys with DMD. The primary endpoint is the change in the so-called "time to rise" from the floor velocity.
- "Our confidence is rooted in robust long-term data showing the durable efficacy and safety of Elevidys, alongside the experience of treating more than a thousand ambulatory boys worldwide," Roche's Chief Medical Officer and Head of Global Product Development Levi Garraway said.
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