Sarepta Therapeutics Inc, developer of the gene therapy Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy, faced a further setback on 18 July after the US Food and Drug Administration was reportedly poised to stop all shipments of the therapy. This followed the deaths of three patients with the disease. Two of the deaths were reported early in the year and involved young people. The third, disclosed on 18 July, involved a 51-year-old man, according to the Bloomberg news service. Sarepta had already announced plans to pause a new clinical study for the therapy while suspending shipments of Elevidys for a subset of non-ambulatory patients. The newest fatality and prospective regulatory action caused the company’s stock price to tumble by 36%.
Earlier in the week, the company announced plans to restructure in order to save $400 million. It also plans to reduce its workforce by 36% which is equivalent to about 500 employees. Elevidys was given an accelerated approval by the FDA in June 2023, and an expanded approval in June 2024. Following the first two deaths, the FDA began investigating the risk of acute liver failure for people receiving the therapy.
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