Sarepta Therapeutics launched an additional Phase III trial intended to address regulatory concerns from the European Medicines Agency for Elevidys in Duchenne muscular dystrophy. The EMA denied marketing authorization after Elevidys failed to meet endpoints in the EMBARK-based evidence package used for a conditional marketing request. Roche, Sarepta’s licensing partner, will oversee the registration-enabling study, which is designed to generate additional placebo-controlled efficacy and safety data over 72 weeks. The effort targets the specific discrepancy the EMA cited: no significant movement benefit after 12 months under the initial evidence. The program highlights a two-track reality for gene therapies—FDA pathways can open first, while EMA evidence thresholds can still reset timelines and trial design requirements. Sarepta and Roche also noted prior safety interruptions following reported patient deaths and stated the new trial is meant to establish an EMA-compliant pathway to access.