The U.S. Food and Drug Administration (FDA) has authorized Sarepta Therapeutics to start dosing nonambulatory participants in Cohort 8 of the ENDEAVOR study, according to a recently published press release.
The new cohort will examine whether adding sirolimus to the immunosuppression plan around the infusion of Elevidys (delandistrogene moxeparvovec-rokl) can reduce the risk of acute liver injury and acute liver failure, known concerns with adeno-associated virus gene therapies. For families, this move signals progress toward potentially broader access to a therapy designed to address the underlying genetic cause of DMD.
“We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk,” said Louise Rodino-Klapac, president of research & development and technical operations at Sarepta. “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.”
Sarepta expects roughly 25 nonambulatory participants with DMD in the United States to enroll. They will receive 14 days of peri-infusion sirolimus before administration of Elevidys, followed by 12 weeks of continued dosing. Researchers will track the incidence of acute liver injury and measure Elevidys-dystrophin expression at 12 weeks. If sirolimus proves effective at lowering liver complications, it could support safer use of the therapy in a group currently excluded from commercial treatment.
Read more about therapies for DMD
Elevidys, the only approved gene therapy for DMD, has already been given to more than 1,100 people worldwide in both clinical trials and real-world care. The treatment uses an adeno-associated virus to deliver a gene that helps the body produce micro-dystrophin, a needed substitute for the missing dystrophin protein. Elevidys is currently available only to ambulatory patients aged 4 and older under the updated label announced last month.
The company emphasized that decisions about resuming commercial access for nonambulant individuals will be made jointly with the FDA once Cohort 8 data become available. Primary endpoint data for Cohort 8 are expected in the second half of 2026, pending enrollment and follow-up.
ENDEAVOR, also known as Study 9001-103, spans multiple cohorts and includes 55 participants to date, ranging from younger ambulatory children to older ambulant and nonambulant individuals. Its main goal is to track changes in micro-dystrophin levels at 12 weeks. Secondary outcomes include the percentage of dystrophin positive fibers.
For patients, the updated safety approach reflects a serious effort to address liver risks while continuing to pursue treatments that could slow or alter the course of a progressive, life-limiting disease.
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