United States: Elevidys gene therapy claimed the life of a patient diagnosed with Duchenne muscular dystrophy as the first death officially linked to this treatment.
What Happened
A Sarepta Therapeutics spokesperson claimed the patient developed acute liver injury, which functions as a documented drug side effect, according to information shared by The Associated Press. According to the company, Elevidys levels of disease severity remained unrecorded in previous patient encounters. Elevidys has been administered to more than 800 patients according to company data, as reported by HealthDay.
Sarepta did not identify the patient who developed acute liver injury although recent infections could have contributed to the problem. According to the company more than 800 people have received this therapy.
The company intends to modify Elevidys prescription information to include this evaluation.
Market Impact
The stock price of Sarepta, based in Cambridge, Massachusetts, declined by more than 27% throughout Tuesday to finish at $73 per share.
Elevidys obtained U.S. FDA approval as the initial gene therapy treatment for Duchenne muscular dystrophy, which requires a single infusion and costs $3.2 million.
Elevidys: A Controversial Approval
The FDA approved Elevidys with expedited status in 2023 while some scientists expressed doubts regarding its effectiveness.
In 2024, the agency granted full approval for Elevidys to treat patients aged four years and above, even if they did not have mobility restrictions.
The therapeutic method utilizes disabled viruses to deliver genes that enable the body to generate dystrophin proteins needed for muscle operation. According to The Associated Press, Sarepta announced that its drug Elevidys has been used by more than 800 patients up to this point, as reported by HealthDay.
Since 2016 Sarepta has obtained expedited FDA approval for three additional Duchenne drugs. The medical community continues studies to verify the effectiveness of these medications as ongoing research has not yet produced confirmed results.