Sarepta gets FDA clearance to test new immunosuppressive regimen with Elevidys

Sarepta Therapeutics says the FDA has approved dosing in Cohort 8 of ENDEAVOR, a study to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with gene therapy Elevidys for non-ambulant individuals with Duchenne muscular dystrophy.

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 enroll approximately 25 participants in the U.S. who are non-ambulatory. The immunosuppression regimen will include 14 days of peri-infusion sirolimus dosing prior to Elevidys administration and will continue for 12 weeks after Elevidys administration

Earlier this month, Sarepta announced an update to the prescribing information for Elevidys. Following the deaths of two non-ambulatory patients, the non-ambulatory indication was removed from the indication and usage section of the therapy’s prescribing information. A boxed warning indicating the risk of ALI and acute liver failure (ALF) was added to the therapy’s label.

Now, Sarepta says it plans to initiate Cohort 8 by the end of the year and pending enrollment, complete primary endpoint data collection in the second half of 2026.