Shares of Sarepta Therapeutics plummeted after the company reported a second patient death linked to its gene therapy, Elevidys, for Duchenne muscular dystrophy. This is the first gene therapy approved in the U.S. for the rare, muscle-wasting condition, but it has faced growing scrutiny since its accelerated approval in 2023.
The second death, following a similar incident in March, involved a teenage boy who suffered fatal acute liver injury, a known side effect of the therapy. Elevidys is administered to children and older patients with a genetic variant of Duchenne’s, but those who are no longer able to walk receive a higher dose, which has been tied to the liver issues.
In response, Sarepta announced it would halt shipments of the therapy to non-ambulatory patients and pause a related study. The company also plans to assemble an expert panel to refine safety protocols, including increased use of immune-suppressing drugs to mitigate the immune system’s reaction that likely triggers the liver damage.
Elevidys, which costs $3.2 million per treatment, was granted expedited approval despite concerns over its effectiveness. The FDA gave it full approval last year and expanded its use to patients aged 4 and older, regardless of whether they could still walk. The latest deaths have sparked concerns among analysts and the public, leading to speculation that the FDA could impose further restrictions or even revoke its approval for non-ambulatory patients.
Sarepta is cooperating with the FDA to assess the situation and determine next steps. The company’s stock fell by more than 42% following the announcement, reflecting investor fears of additional regulatory hurdles.
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