Sarepta will resume gene therapy shipments after FDA review of recent patient death
The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy.
Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product.
Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk.
The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.'
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy.
FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients.
'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement.
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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
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