A Drug Company and the FDA in a Heated Dispute Over Patient Safety
In a surprising turn of events, Sarepta Therapeutics has chosen to defy the FDA's request to stop distributing its gene therapy drug, Elevidys. This decision comes after three patient deaths linked to the treatment. The FDA has expressed serious concerns about the safety of the drug and has asked Sarepta to halt all shipments. However, the company has refused, arguing that its own reviews show no new safety issues for younger patients.
FDA's Power and Sarepta's Defiance
The FDA has the power to remove drugs from the market, but this process can be slow. Typically, companies comply with informal requests from the agency. This time, however, Sarepta has decided to keep the drug available for certain patients. The company has also added a bold warning to the drug's label and plans to lay off a third of its employees.
Elevidys: A Breakthrough with Controversy
Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, a fatal muscle-wasting disease. The drug received accelerated approval in 2023, despite some FDA scientists doubting its effectiveness. Last year, the FDA granted full approval and expanded the therapy's use to older patients. However, the recent deaths have raised serious questions about the drug's safety.
Patient Deaths and Company Response
Sarepta has confirmed that all three patient deaths were linked to liver injury, a known side effect of the drug. The company has halted shipments of the therapy for older boys with Duchenne's and has placed a clinical trial on hold. Despite these setbacks, Sarepta remains committed to making the drug available for younger patients.
Market Reaction and Criticism
The company's decision to defy the FDA has sparked criticism from Wall Street analysts. Sarepta's shares fell more than 35% on Friday, reflecting the market's concerns about the drug's future. The company has faced criticism in the past for failing to complete studies needed to secure full FDA approval of its drugs.