A 16-year-old patient taking Sarepta Therapeutics’s Elevydis gene therapy for Duchenne muscular dystrophy has died, the biopharmaceutical company shared in a Tuesday statement.
The teen died of acute liver failure, a known potential side effect of Elevidys and similar therapies, but not one that had previously been linked to death for Elevidys, the company said in the release. Sarepta added that the patient had also recently had a cytomegalovirus infection, a common virus that can lead to liver problems and could have contributed to his death.
Sarepta shares fell from $101.35 at market close on Monday to $78.61 at market open on Tuesday after the news broke.
The patient’s death comes amid debate surrounding Elevydis and the process that led to its initial release: accelerated approval.
“The tragic death of a young patient from Elevydis, unfortunately, was a risk that should not have been taken,” Rita Redberg, a cardiologist and professor of medicine at the University of California, San Francisco, told Healthcare Brew via email.
Green lights, red flags. The FDA’s accelerated approval pathway began in 1992 to help speed up access to drugs for AIDS/HIV. It allows drugs for serious conditions to be approved with less evidence than normally required, with approval instead based on a surrogate endpoint—a measurement likely, but not guaranteed, to predict clinical benefit—and a promised confirmatory clinical trial.
However, a Jan. 14 report from the Department of Health and Human Service’s Office of the Inspector General (OIG) found that through December 2021, this process led three of 20 sampled drugs to be approved despite FDA reviewers or advisory committee members having expressed concerns about the evidence behind the drugs, Healthcare Brew previously reported. This included Biogen’s Alzheimer treatment Aduhelm, Covis Pharma’s preterm birth prevention treatment Makena, and another Duchenne muscular dystrophy therapy by Sarepta Therapeutics, Exondys 51.
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The same thing also happened with Elevidys.
Peter Marks, the FDA’s director of the Center for Biologics Evaluation and Research, overruled his staff in June 2023 to give Elevydis accelerated approval for certain four- and five-year-olds with a specific gene mutation.
Marks then granted the drug full approval for almost all patients in June 2024, overruling three review teams and two top lieutenants who said Sarepta “provided no satisfactory data to support effectiveness claims for all ages and for nonambulatory patients,” Stat reported at the time.
Redberg said that, in her opinion, Elevydis was approved without enough evidence that its benefits outweighed its risks, which most often include vomiting and nausea, liver injury, fever, and low blood platelet count, according to the drug’s prescribing information.
“This tragedy underscores the need for urgent reform to the FDA’s flawed approval process and its pro-industry culture. All treatments must be assessed for both risks and benefits, and the consequences of approving a treatment with no or uncertain benefits are predictable,” Redberg said.
Sarepta noted in the release that, to date, Elevydis has treated more than 800 patients in clinical trials or as a prescribed therapy, and the company is still confident that the drug’s benefits outweigh any risks.
Sarepta said it plans to update prescribing information and has informed health authorities, clinical study investigators, and prescribers.