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Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study, which is expected to begin before the end of the year
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The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy
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Decisions regarding resuming commercial dosing for this population will be made in collaboration with FDA after reviewing study data
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 25, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl) 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. Primary endpoints include incidence of ALI and ELEVIDYS-dystrophin expression at 12 weeks. The approach is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health.
“We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk. Guided by real-world experience, external clinician expertise, and FDA input, Cohort 8 of the ENDEAVOR study will evaluate integrating sirolimus into our immunosuppression approach, with the goals of mitigating the risk of acute liver injury and restoring access for non-ambulant individuals living with Duchenne,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations. “We plan to initiate Cohort 8 by the end of this year and pending enrollment, complete primary endpoint data collection in the second half of 2026.”
ELEVIDYS is the only approved gene therapy for Duchenne. To date, ELEVIDYS has been administered to over 1,100 patients globally in clinical and real-world settings. Sarepta continues to work closely with the FDA to ensure that all regulatory decisions are grounded in science and the best interests of patients who face a rare, irreversibly progressive and ultimately fatal disease. ELEVIDYS remains available as appropriate to ambulatory individuals ages 4 and over per an updated label announced earlier this month.
About ENDEAVOR (Study 9001-103)
Study SRP-9001-103, also known as ENDEAVOR, is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS (delandistrogene moxeparvovec) in multiple cohorts of male patients with Duchenne. The study has enrolled 55 participants across 7 cohorts, and has dosed younger ambulatory individuals aged 2-7 at time of treatment, and older ambulant individuals and non-ambulant individuals.
The primary endpoint in ENDEAVOR is the change from baseline in the quantity of ELEVIDYS micro-dystrophin protein expression measured by western blot at 12 weeks. Secondary outcome measures include change from baseline in micro-dystrophin expression measured by percent dystrophin positive fibers at 12 weeks.
About ELEVIDYS (delandistrogene moxeparvovec-rokl)
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
ELEVIDYS is indicated for the treatment of ambulatory patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases.