Outrage of the Month: Patient Deaths From a Duchenne Muscular Dystrophy Gene Therapy
Health Letter, August 2025
By Robert Steinbrook, M.D.
Director, Public Citizen's Health Research Group
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On July 18, 2025, days after initially refusing a Food and Drug Administration (FDA) request to pull from the market an approved gene therapy for Duchenne muscular dystrophy linked to three patient deaths, Sarepta Therapeutics announced a pause of shipments of delandistrogene moxeparvovec-rokl (Elevidys).
The pause in the distribution of delandistrogene moxeparvovec-rokl was urgently needed, protected patient safety, and allowed the FDA to decide whether the gene therapy should remain on the market.
However, 10 days later, in a dumbfounding turn of events, the FDA caved. Following public pressure from the Wall Street Journal editorial page, right-wing influencer Laura Loomer and others, the FDA allowed shipments of the gene therapy to restart for young boys able to walk. On July 29, the recently hired official in charge of the pause resigned, “to return to California and spend more time with his family.”
Public Citizen called for the pause in use of delandistrogene moxeparvovec-rokl in June 2025, after the second of the deaths and a month before the FDA and the company acted. In 2023 and 2024 we urged the FDA not to approve the drug, because of concerns about safety, effectiveness, and inadequate clinical trials.
After two teenage boys died from acute liver failure linked to the gene therapy earlier in 2025, the company reported a third death of a 51-year-old man with limb-girdle muscular dystrophy who died from liver failure in a related clinical trial. The liver toxicities may be related to a massive immune response to the viral vector used to deliver the gene therapy for both types of muscular dystrophy.
Subsequently, the FDA announced that it was investigating a fourth death, of an 8-year-old-boy who received the gene therapy and who died on June 7, 2025, but concluded days later that the death was unrelated to the gene therapy product itself.
On July 28, for reasons that were inadequately explained, the FDA recommended “the removal of the voluntary hold for ambulatory patients who may now receive Elevidys.” The company said that it will resume shipments of the product “imminently.”
No one disputes the fact that better therapies are needed for Duchenne muscular dystrophy, a rare but devastating genetic condition that mainly affects males (incidence of about 1 in 5,000 live male births). Affected children typically have gait disturbances by age of five years and become wheelchair dependent in their early teenage years.
Delandistrogene moxeparvovec-rokl is not that better therapy, however. The therapy is administered as a single intravenous infusion and costs more than $3 million per treatment, making it one of the most expensive drugs in the world. More than 900 patients four years of age and older have received the therapy.
By identifying and reviewing all cases of liver failure requiring hospitalization following the gene therapy, the FDA should not only establish the risk of the treatment but also make the essential information public so that patients, families and clinicians are fully informed.
Going forward, the FDA must also review its internal standards and procedures for approving potentially risky gene therapies. During the Biden administration, a senior FDA official twice overruled the objections of other agency officials and review teams, authorizing both an initial approval of delandistrogene moxeparvovec-rokl in 2023 and subsequent expansion of the approval in 2024 to cover most Duchenne patients.
Instead of reversing course, the FDA should have required the manufacturer to demonstrate not only that enhanced safety protocols can end the risk of serious liver injury, but also to provide additional data establishing that the therapy has meaningful clinical benefit. As of the end of July, neither the FDA nor the company had made public sufficient updated information.
The FDA’s flip-flopping about the gene therapy and whether it should continue to be marketed makes little sense and has severely damaged its credibility as a public health agency. A gene therapy cannot be unsafe on Friday and safe on Monday.