Sarepta agrees to include black box warning in Elevidys label

Following previously communicated steps being taken to strengthen the safety profile of ELEVIDYS, Sarepta (SRPT) is providing an update on on-going engagement with the U.S. Food and Drug Administration regarding the ELEVIDYS label. Consistent with other AAV-delivered gene therapies, the FDA has requested that the label include a black box warning for acute liver injury and acute liver failure. Sarepta agrees with this change, which appears to resolve any material issues with the ambulant portion of the ELEVIDYS label. Sarepta recently announced that it was pausing shipments of ELEVIDYS for non-ambulant patients while it explored the adoption of additional prophylactic immunosuppression. To that end, Sarepta convened an Expert Committee of neuromuscular specialists, hepatologists, hematologists, and immunologists to review cases of ALF and explore additional immunosuppression regimens. The Committee aligned on an enhanced immunosuppressive regimen with sirolimus for ELEVIDYS in non-ambulant patients. Sarepta will submit the finding of the expert panel and proposed protocol to the FDA imminently and will discuss a proposal to gather data on the regimen in a new cohort of the ENDEAVOR study as a pathway to re-establish dosing in the non-ambulant setting. Additionally, Sarepta is assessing real-world data generation opportunities for ambulant patients through investigator-initiated trials.