Sarepta will resume gene therapy shipments after FDA review of recent
patient death
[July 29, 2025]
By MATTHEW PERRONE
WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics
jumped in afterhours trading Monday after the company said it would
resume shipping its gene therapy for some patients, following a brief
pause requested by regulators.
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.”
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A cyclist passes the headquarters of Sarepta Therapeutics , Monday,
July 28, 2025, in Cambridge, Mass. (AP Photo/Charles Krupa)
 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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