Sarepta Therapeutics has announced that the FDA has approved Cohort 8 of the ENDEAVOR study, to evaluate the use of immunosuppression with sirolimus as part of treatment with Elevidys in non-ambulatory individuals with Duchenne. Data from this cohort will determine whether administering sirolimus before and after Elevidys infusion can help reduce acute liver injury observed in some individuals.
Sarepta voluntarily paused commercial shipments of Elevidys for non-ambulatory individuals with Duchenne several months ago, after cases of acute liver failure resulting in death occurred. It also paused the ENVISION study, a placebo-controlled trial evaluating Elevidys in older ambulatory and non-ambulatory individuals.
Cohort 8 of the ENDEAVOR study will enroll approximately 25 non-ambulant participants in the US. Sarepta aims to begin enrolling by the end of this year, and complete data collection in the second half of 2026.
Link to press release: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-approval-begin-endeavor-cohort-8-evaluate
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