United States: A suffering individual died when receiving Elevidys as their treatment for Duchenne muscular dystrophy, which represents the first known death related to the therapy.
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The company, which is associated with the drug, Sarepta Therapeutics, declared that the deceased patient developed acute liver injury, which represents an identified adverse effect, according to The Associated Press.
According to the company, there has yet to be another case with this extreme level of severity.
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 Elevidys 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.
Patient death resurfaces safety concerns for Sarepta's gene therapy, but it's unclear that market access will be affected https://t.co/W75E0pNYWH
— BioCentury (@BioCentury) March 19, 2025
The Food and Drug Administration (FDA) issued emergency authorization to Elevidys in 2023, but several scientists have expressed doubts about its therapeutic results.
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.