Sarepta shares slump after report of third patient death this year
The patient, who died last month, was enrolled in an early-stage study testing Sarepta's gene therapy to treat a muscle-wasting disorder called limb-girdle muscular dystrophy, the report said, citing a company spokesperson.
Two teenage boys died earlier this year from liver toxicities after they received Sarepta's Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy. Both patients were non-ambulatory, i.e., they were unable to walk independently.
The fatalities have brought Sarepta under heightened regulatory scrutiny, and cast doubts on the safety and future demand for Elevidys.
Sarepta did not immediately respond to a Reuters request for comment outside of its business hours. It has informed regulatory authorities and the trial investigators about the patient's death, according to the report.
The company said on Wednesday it had halted development of several experimental gene therapies for limb-girdle muscular dystrophy.
It has also said it would cut 500 jobs, and add a serious warning on the label of Elevidys for the risk of acute liver injury and liver failure in patients with Duchenne muscular dystrophy who can walk.
The U.S. Food and Drug Administration gave traditional approval to Elevidys for patients aged four and above who can walk, and conditional approval for those who cannot, in 2024 despite the therapy failing to meet the main goal in a late-stage study.
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