A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 in Gene Therapy suggests the body’s natural immune system might be impairing the therapy’s efficacy.
The FDA approved Elevidys in June 2024 for young patients with Duchenne muscular dystrophy, a rare genetic condition that weakens the body’s muscles. In October, a phase 3 clinical trial found no association between drug and improved functional motor abilities — the study’s primary endpoint. Sarepta has priced Elevidys at $3.2 million per treatment.
More recently, Cambridge, Mass.-based Sarepta reported the death of a young man who experienced acute liver failure after being treated with Elevidys. Acute liver failure is a known side effect of the therapy.
In the Gene Therapy study, researchers from the University of Portsmouth in the U.K. speculated that the body’s natural immune system might be impairing the efficacy of Elevidys (delandistrogene moxeparvovec).
Duchenne muscular dystrophy is caused by the absence of the protein dystrophin. Therapies for DMD, including Elevidys, work to reintroduce dystrophin — but the immune system might perceive dystrophin as a foreign antigen and trigger a response to block it, eroding the drug’s efficacy.
The researchers concluded that DMD drug research and development should consider evaluating each patient’s immune status and testing therapeutics that “prevent or mitigate anti-dystrophin immunity.”
