What's happened
Sarepta refuses to halt shipments of its Duchenne therapy Elevidys after the FDA requested a suspension following patient deaths linked to liver injury. The FDA's informal request was ignored, raising safety and regulatory concerns amid ongoing scrutiny and layoffs at Sarepta.
What's behind the headline?
The FDA's decision not to enforce a suspension despite Sarepta's refusal highlights a complex regulatory environment where safety signals are often contested. Sarepta's stance underscores the tension between regulatory authority and corporate independence. The deaths linked to liver injury, although acknowledged, have not prompted immediate market withdrawal, reflecting the FDA's cautious approach. This situation may lead to prolonged legal and regulatory battles, with the future of Elevidys hanging in the balance. The company's refusal to comply could set a precedent for other firms, emphasizing the importance of safety transparency and regulatory accountability. Ultimately, this standoff underscores the need for clearer, more enforceable safety protocols in gene therapy approvals, especially for high-risk treatments.
What the papers say
Bloomberg reports that Sarepta's refusal to suspend Elevidys sales follows patient deaths linked to liver injury, with the FDA requesting a halt. The Independent highlights the company's recent layoffs and safety concerns, noting the FDA's authority to pull drugs from the market but its cautious, informal approach. AP News emphasizes the ongoing safety issues and Sarepta's decision to keep the drug available for younger patients, despite the deaths and regulatory pressure. All sources agree that the situation underscores the tension between regulatory oversight and corporate decision-making, with safety concerns remaining unresolved and the future of Elevidys uncertain.
How we got here
The FDA approved Elevidys in 2023 for Duchenne's muscular dystrophy, despite safety concerns and scientific doubts. The agency requested a suspension after three deaths linked to liver injury, but Sarepta declined, citing its review showing no new safety signals. The company also recently laid off 500 employees amid stock declines and scrutiny.
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The Food and Drug Administration is a federal agency of the United States Department of Health and Human Services, one of the United States federal executive departments.
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Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States.
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Juul Labs, Inc. is an American electronic cigarette company which spun off from Pax Labs in 2017. It makes the Juul electronic cigarette, which atomizes nicotine salts from leaf tobacco supplied by one-time use cartridges.
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Martin Adel "Marty" Makary is a British-American surgeon, professor, and author. He practices surgical oncology and gastrointestinal laparoscopic surgery at the Johns Hopkins Hospital, is Mark Ravitch Chair in Gastrointestinal Surgery at Johns Hopkins Sch