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FDA Says It's Investigating Sarepta’s Duchenne Patient Deaths
- 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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FDA Probes Gene Therapy After Two Deaths
(MedPage Today) -- The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with delandistrogene moxeparvovec (Elevidys) gene therapy, the agency said. The investigation will focus on the...
·New York, United States
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Total News Sources16
Leaning Left2Leaning Right0Center10Last UpdatedBias Distribution83% Center
Bias Distribution
- 83% of the sources are Center
83% Center
L 17%
C 83%
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