The deadly saga of the controversial gene therapy Elevidys
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Summary
Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy (DMD) has been banned by the US Food and Drug Administration (FDA), following the deaths of two teenagers treated with the drug.
DMD causes muscles to waste away over time and mostly affects boys, with symptoms usually starting in early childhood.
The FDA approved the therapy, branded Elevidys, in 2023, for four- and five-year-olds with the disease, but on the basis that the company completed another clinical trial.
However, the results of that trial revealed the drug had failed to meet its primary endpoint.
The FDA approved the drug again in June 2024 for DMD sufferers over the age of four who can walk independently, but criticism of this approval led to disagreements within the FDA.
Following the recent deaths, the FDA has now requested the suspension of shipments of Elevidys and is investigating the risks of serious outcomes “including hospitalization and death” associated with the treatment.