Sarepta gives data for DMD gene therapy

Wednesday 1 November 2023

United States

Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.