Pause in Use of Duchenne Muscular Dystrophy Gene Therapy Protects Patient Safety
WASHINGTON, D.C. — After initially refusing a Food and Drug Administration (FDA) request to pull an approved gene therapy for Duchenne muscular dystrophy linked to patient deaths, Sarepta Therapeutics announced a pause on shipments of delandistrogene moxeparvovec-rokl (Elevidys) effective at the close of business today.
Two teenage boys have died from acute liver failure and a 51-year-old man with limb girdle muscular dystrophy recently died from liver failure in a related clinical trial. The therapy is administered as a single intravenous infusion and costs more than $3 million per treatment. More than 900 patients four years of age and older have received the therapy.
Public Citizen Health Research Group Director Dr. Robert Steinbrook issued the following statement:
“The complete pause in the use of delandistrogene moxeparvovec-rokl (Elevidys) was urgently needed and protects patient safety. Public Citizen called for the pause a month ago, after the second patient death. In 2023 and 2024, we repeatedly urged the FDA not to approve the gene therapy because of concerns about safety, effectiveness and inadequate testing.
“The FDA should immediately identify and review all cases of liver failure requiring hospitalization, make the essential information about the cases public so that patients, families and clinicians can be fully informed and decide whether delandistrogene moxeparvovec-rokl should remain on the market.”