FDA Should Immediately Pause Use of Duchenne Muscular Dystrophy Gene Therapy
WASHINGTON, D.C. — Sarepta Therapeutics said a second teenage boy died from acute liver failure linked to its FDA-approved gene therapy for Duchenne muscular dystrophy. The rare but deadly muscle-wasting disease with limited treatment options typically affects boys, who lose the ability to walk by their teenage years.
In 2023 and 2024, Public Citizen repeatedly urged the Food and Drug Administration (FDA) not to approve the gene therapy, known as delandistrogene moxeparvovec-roki (Elevidys) because of concerns about safety, effectiveness and inadequate testing. More than 900 patients four years of age and older have received the therapy, which is administered in a single intravenous infusion and costs more than $3 million per treatment. The first patient died after being treated in March.
Public Citizen Health Research Group Director Dr. Robert Steinbrook issued the following statement:
“The FDA should immediately pause use of delandistrogene moxeparvovec-roki (Elevidys) and conduct a full safety review. It is no secret that the gene therapy can lead to acute serious liver injury and that many patients develop elevated liver enzymes, which can precede severe liver injury.
“All cases of acute liver injury, including cases of liver failure requiring hospitalization, should be urgently identified and reviewed so that the agency can act to protect patient safety. The number of cases and the ages of the patients should also be made public, so that patients, families, and clinicians can be fully informed about the risks. Although the company has temporarily suspended shipments of the gene therapy for patients who are unable to walk, a complete pause is needed.”