First Death Reported from Sarepta's Duchenne Gene Therapy Raises Safety Concerns

Sarepta Therapeutics confirms the death of a 16-year-old patient from acute liver failure linked to Elevidys, prompting updates to prescribing information and a significant stock decline.

• A 16-year-old patient treated with Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy died from acute liver failure, marking the first fatality among over 800 treated patients.
• Acute liver injury is a known side effect of Elevidys and similar gene therapies, though the severity of this case is unprecedented for the treatment.
• Sarepta attributes the incident to a combination of the therapy's risks and a recent cytomegalovirus infection, which may have exacerbated the liver failure.
• The company is updating Elevidys' prescribing information to reflect the incident's severity and has reported the case to health authorities, clinical investigators, and prescribing physicians.
• Following the announcement, Sarepta's stock fell over 23%, reflecting investor concerns about the therapy's safety profile and future prospects.

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