United States: The therapy of Elevidys ended in the death of a patient who suffered from Duchenne muscular dystrophy, marking the initial recorded death incident associated with this treatment.
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Sarepta Therapeutics declared through their company that their drug-induced patient developed acute liver injury, which aligns with already known side effects per Associated Press reports. The company reports no other case has shown this severe extent of harm.
So far, Elevidys has received treatment from more than 800 patients, according to company reports.
Sarepta stated that recent infections potentially contributed to the liver injury that happened to one of their patients while refusing to provide details about this individual.
According to the company’s reporting statistics, the therapy has been administered to more than 800 patients.
The company has decided to revise Elevidy’s prescribing manual to include information about this particular case.
The stock value for Sarepta dropped by more than 27% on Tuesday, which led to its closing price reaching approximately USD 73 per share.
FDA Approval and Growing Scrutiny
The US Food and Drug Administration approved Elevidys as the foremost gene therapy for Duchenne muscular dystrophy due to its USD 3.2 million one-time infusion cost.
This drug serves rare males who have muscle weakness, which later causes mobility loss and leads to early death.
The Food and Drug Administration (FDA) issued emergency authorization to Elevidys in 2023, but several scientists have expressed doubts about its therapeutic results.
The death of a 16-year-old taking Sarepta Therapeutics' ELEVIDYS has reignited concerns about the FDA's accelerated approval process: https://t.co/T4AHt2hC7t
— Caroline Catherman (@CECatherman) March 19, 2025
The agency obtained complete approval in 2024, which extended coverage to patients aged 4 and older who were not required to show locomotor disabilities.
A disabled virus introduces a gene that enables the body to generate dystrophin protein that is fundamental to muscular function through therapy.
Sarepta reported that Elevidys treatment has been administered to more than 800 patients, according to The Associated Press.
Sarepta obtained three additional Duchenne drug FDA accelerated approvals starting in 2016.
All clinical trials for new Duchenne treatments remain active, but scientists have yet to prove the effectiveness of any new intervention.