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FDA Says It's Investigating Sarepta’s Duchenne Patient Deaths

UNITED STATES, JUN 24 – Two non-ambulatory Duchenne muscular dystrophy patients died from acute liver failure after receiving Sarepta's Elevidys, prompting halted shipments and an FDA safety investigation.

  • The FDA is investigating two deaths of non-ambulatory Duchenne muscular dystrophy patients treated with Sarepta’s gene therapy Elevidys in 2025.
  • These deaths occurred after Elevidys was expanded to non-ambulatory patients despite its initial approval for ambulatory children, raising concerns about regulatory fast-tracking and risk communication.
  • Sarepta and Roche paused use and shipments of Elevidys in non-ambulatory patients and initiated an internal review while the FDA considers further regulatory action and protocol updates for ongoing trials.
  • Advocates and legal experts call for greater transparency and stronger informed consent, warning that the risks of liver failure and death were not fully reflected in Elevidys prescribing information.
  • This investigation highlights systemic issues in approving and monitoring gene therapies for vulnerable patients, emphasizing the need to understand treatment consequences before wider use.
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The Business Journals broke the news in United States on Tuesday, June 24, 2025.
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