Sarepta Therapeutics, Inc. has shared that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR, an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts of individuals living with Duchenne. The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS for non-ambulatory individuals with Duchenne. According to Sarepta, data from Cohort 8 will be used to determine whether administering sirolimus prior to and after ELEVIDYS infusion can help reduce acute liver injury (ALI), a known risk associated with AAV gene therapy.
The cohort will include approximately 25 non-ambulatory participants in the U.S. and will use an immunosuppression regimen that begins with 14 days of sirolimus before ELEVIDYS administration, continuing for 12 weeks afterward. Primary endpoints include both safety, the incidence of ALI, and efficacy, ELEVIDYS-dystrophin expression at 12 weeks, of ELEVIDYS with this immunosuppression regimen.
Sarepta anticipates initiating Cohort 8 by the end of 2026 and, pending enrollment, completing primary endpoint data collection in the second half of 2026. The company reports that decisions regarding resuming commercial dosing for this population will be made in collaboration with FDA after reviewing study data.
Earlier this month, the FDA approved a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients treated with the product.
PPMD is encouraged to see continued collaboration between Sarepta and the FDA as they work to advance therapeutic options and improve safety for our community. We look forward to future updates as Cohort 8 progresses and will share additional information with families as soon as it becomes available.
Read Sarepta’s press release here.
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