FDA Approves Sarepta to Begin New ELEVIDYS Gene Therapy Study for Non-Ambulant Duchenne Patients

Reuters11-25
FDA Approves Sarepta to Begin New ELEVIDYS Gene Therapy Study for Non-Ambulant Duchenne Patients

Sarepta Therapeutics Inc. has received approval from the U.S. Food and Drug Administration (FDA) to begin dosing in Cohort 8 of the ENDEAVOR study (Study 9001-103). This cohort will evaluate an enhanced immunosuppressive regimen, including sirolimus, as part of ELEVIDYS gene therapy for non-ambulant individuals with Duchenne muscular dystrophy. The study aims to mitigate the risk of acute liver injury and failure associated with AAV gene therapy. Sarepta will collaborate with the FDA to determine the resumption of commercial dosing for this patient population after reviewing study data.

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