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Nov 6, 2026

Sarepta halts enrollment in confirmatory Phase 3 trial of Elevidys

Sarepta Therapeutics announced it will not enroll additional patients in the confirmatory Phase 3 trial of Elevidys, its Duchenne muscular dystrophy gene therapy, a study required to maintain the drug's accelerated approval.

Nov 6, 2026·2 sources

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Top claims

▪Following the ENVISION trial closure, non-ambulatory Duchenne patients' potential pathway to Elevidys access rests on ENDEAVOR Cohort 8, an open-label Phase 1b safety study enrolling approximately 25 U.S. patients
▪The U.S. FDA granted accelerated approval to Elevidys in June 2023 based on micro-dystrophin expression in skeletal muscle as a surrogate endpoint
▪In July 2025, a third non-ambulatory patient death led the U.S. FDA to request a complete halt of Elevidys distribution and place clinical trials on hold

Topics

Clinical trial design Drug Policy, Pricing & Approval Gene therapy Pharmaceutical earnings Drug safety Clinical trials Genetic diseases Accelerated approval pathways