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Sarepta shares crash as second patient dies after receiving its gene therapy
By Christy Santhosh and Bhanvi Satija - 6/16/2025
By Christy Santhosh and Bhanvi Satija
(Reuters) -Shares of Sarepta Therapeutics <SRPT.O> slumped 42% on Monday after a second patient who had received its gene therapy, Elevidys, died in a span of three months, raising doubts the safety of the treatment.
It is the only gene therapy approved by U.S. drug regulators for treating Duchenne muscular dystrophy (DMD), a rare form of the disease, in patients aged four and above.
It was first approved in 2023 for patients who could walk and a year later was cleared for use in those who could not move independently.
The second patient died of acute liver failure, the company said on Sunday. Elevidys is a one-time gene therapy with a known risk of liver damage and both the deaths had similar complications.
"While these two deaths occurred in non-ambulatory patients, we view the risk profile as now heightened for all DMD patients," H.C. Wainwright analyst Raghuram Selvaraju wrote in a note.
He expects very little use of the therapy in patients who are unable to walk, which represents about 80% of patients.
The Department of Health and Human Services, which oversees the U.S. Food and Drug Administration, did not immediately respond to a Reuters request for comment.
William Blair analyst Sami Corwin said the liver toxicity risk might be limited to older patients who receive a higher dose, with dosage determined by a patient's weight.
Sarepta has temporarily suspended shipments of Elevidys to non-ambulatory patients and is working with regulators to develop a new treatment plan for them, aimed at suppressing an overactive immune response seen with the therapy.
Analysts said Sarepta could face tough scrutiny over its effort to get the new treatment plan approved and future gene therapies for DMD would be expected to meet a higher bar.
Prior to the patient deaths, Vinay Prasad, who heads the FDA's unit that regulates gene therapies and vaccines, had raised questions about the approval of Elevidys under former unit head Peter Marks, analysts said.
"The second patient death may raise serious questions on Elevidys' sales outlook," said Jefferies analyst Andrew Tsai.
As of last closing share price, Sarepta had a market value of $3.56 billion.
(Reporting by Rashika Singh, Christy Santhosh and Bhanvi Satija in Bengaluru; Editing by Shailesh Kuber and Arun Koyyur)