With U.S. Food and Drug Administration (FDA) approval, Sarepta Therapeutics plans to start a new study group as part of the ENDEAVOR trial by the end of this year.  This additional study plans to enroll 25 non-walking patients with Duchenne muscular dystrophy.

Patients will be treated with the gene therapy ELEVIDYS, which contains sirolimus, a medicine that can quiet the immune system. The goal of this extra medicine is to try to lower the risk of liver injury after treatment.  Patients will receive sirolimus for 2 weeks before ELEVIDYS treatment. They will then be treated with ELEVIDYS and sirolimus simultaneously for 12 weeks.

Sarepta states, “We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk.”

For reference, ELEVIDYS is a single-dose gene therapy for Duchenne. It works by delivering a short version of the dystrophin protein to treat the condition. ELEVIDYS is recommended for treating walking patients with Duchenne who are 4 years of age or older. The U.S. FDA recently added a boxed safety warning for ELEVIDYS because of the risk of serious liver injury and liver failure.

Defeat Duchenne Canada remains committed to bringing you the most up-to-date information.

Update written by Defeat Duchenne Canada Research Volunteer Omar Sheikh.