Roche to Rechallenge Europe With Elevidys in Phase 3 Trial After Earlier Rejection

Roche is launching a new Phase 3 trial as it seeks to renew its bid for European approval of Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD).

Roche made the decision after receiving a negative review of Elevidys from European regulators last year, according to Stat News, a biotech news outlet, on the 16th local time.

European regulators previously rejected approval, saying Elevidys had failed to demonstrate a long-term benefit for patients with the degenerative muscle disease.

The newly launched Phase 3 trial will enroll approximately 100 boys with early-stage Duchenne muscular dystrophy and run for 72 weeks. The goal is to evaluate the safety and efficacy of Elevidys compared with placebo.

Based on evidence generated in the trial, Roche plans to reapply to the European Medicines Agency (EMA) for marketing approval and pursue applications with regulatory agencies in other countries. Roche holds the rights to Elevidys outside the United States, while Sarepta Therapeutics, its developer, markets the therapy in the United States.