First Death Reported in Patient Using Sarepta's Duchenne Gene Therapy

Sarepta Therapeutics investigates acute liver failure case, leading to a 23% stock drop and plans to update prescribing information.

A 16-year-old patient receiving Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy has died from acute liver failure, marking the first reported death linked to the treatment.
Sarepta stated that acute liver injury is a known potential side effect of Elevidys and noted a recent cytomegalovirus infection as a possible contributing factor in this case.
The company plans to update Elevidys' prescribing information to reflect this incident and has reported the death to health authorities.
Elevidys, approved under expedited FDA review in 2023, is the first gene therapy for Duchenne muscular dystrophy and costs $3.2 million for a one-time treatment.
Following the announcement, Sarepta's stock dropped 23%, highlighting significant financial and reputational impacts for the Cambridge-based biotech company.