June 16, 2025 at 5:20 p.m. EDT
Biotech firm Sarepta Therapeutics said that a second patient died after receiving its gene therapy to treat Duchenne muscular dystrophy, the latest drug to draw scrutiny after winning fast regulatory approval.
The Cambridge, Massachusetts-based firm’s stock lost more than 40 percent of its value Monday, a day after the company disclosed the death and announced that it would temporarily stop shipping the treatments for certain patients, and pause a clinical trial, while it evaluates its protocols.
Sarepta said the patient died of acute liver failure linked to its therapy, Elevidys. The first patient died after receiving the therapy in March. The treatment, which costs $3.2 million per patient, is administered as a one-time infusion and is designed to address the underlying cause of Duchenne, a fatal disease that causes the muscles of young boys to waste away. Both deaths occurred in patients who were unable to walk, according to a company release.
The deaths have put Duchenne patients and their families in an even more difficult position, weighing the risks of treatment against a progressive, deadly disease.Following
“With these two deaths, just months apart, that risk-benefit calculation has changed markedly,” said Michael Kelly, chief scientific officer for CureDuchenne, a patient advocacy group. Debra Miller, the group’s founder and CEO, said: “It’s almost like the diagnosis of a deadly disease all over again. You have hope, then your hope is crushed.”
More than 900 patients have received Elevidys, according to Sarepta, which reported about $821 million in revenue from the therapy for last year.
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“The FDA is aware of the second reported fatal case of severe liver failure and are treating this situation with the highest level of concern,” said Andrew Nixon, a spokesman for the Department of Health and Human Services. “The FDA will take all appropriate regulatory actions to protect patients during our review of gene therapy products.”
The FDA first approved Elevidys on a limited basis in 2023, and it was controversial. An independent committee of experts advising the FDA voted 8-6 to recommend approving Elevidys, though some who supported it still expressed doubts. The agency’s staff also raised concerns about its effectiveness and safety, including the risk of liver failure associated with the same class of gene therapy.
The FDA approved Elevidys using its “accelerated approval” pathway, making a medicine widely available to patients with life-threatening diseases and few good treatment options, despite having less evidence than it usually requires. The agency typically requires additional trials post-approval to confirm that patients benefit in such cases.
In recent years, some public health watchdogs have argued that the FDA has been too willing to approve drugs based on ambiguous evidence, citing the approval of the Alzheimer’s drug Aduhelm and a drug treating amyotrophic lateral sclerosis, both of which were later pulled from the market by their manufacturers.
“These are horrible diseases, and we all understand that,” said Diana Zuckerman, president of the nonprofit National Center for Health Research, who has been critical of the FDA exercising flexibility to approve drugs despite ambiguous evidence. “But it does patients no favor to approve a product that can kill them and isn’t proven to potentially help them,” she said.
The first Trump administration was supportive of regulatory flexibility for devastating diseases that lacked effective treatments, signing legislation in 2018 to give patients an alternative way to try drugs not approved by the FDA.
The FDA expanded its approval for Elevidys in June 2024. Though a clinical trial with 218 male participants failed to meet its primary measure of success, the agency found secondary measures “to be compelling and to indicate clinical benefit compared to placebo.” In broadening the approval to treat patients who couldn’t walk and were at least 4 years old, the FDA required an additional trial to confirm the benefits.
Sarepta in March disclosed the death of a young man after treatment with Elevidys, saying that while risk to the liver was a known side effect, the death “represents a severity of acute liver injury not previously reported.”
On Sunday, Sarepta disclosed the second death. The company said it would immediately convene an independent group of experts to discuss a regimen for suppressing the immune system in conjunction with Elevidys. The FDA agreed with the decision to pause the trial, Sarepta said.
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