Gene therapy maker Sarepta Therapeutics has told the FDA it won’t stop shipping its treatment for muscular dystrophy despite a third patient’s death linked to liver injury. The company, which recently laid off 500 employees and cut prices of one of its existing treatments, claims there are no new safety concerns with its leading therapy, Elevidys.
Elevidys is the first FDA-approved gene therapy for Duchenne’s muscular dystrophy, a fatal muscle-wasting disease that primarily affects males. The treatment has faced scrutiny since its clearance in 2023 and was granted full approval last year after an accelerated review by some FDA scientists who doubted its effectiveness.
The FDA had requested Sarepta to suspend all sales of Elevidys due to the safety concerns, but the company refused. Instead, Sarepta plans to continue providing the treatment to patients with earlier stages of Duchenne’s muscular dystrophy. The FDA has also halted a trial for an experimental gene therapy that was linked to one of the patient deaths.
Sarepta’s decision not to comply with the FDA’s request has raised concerns about the availability of Elevidys, which is still under review by regulatory authorities. The company’s stock price plummeted 35% on Friday after news of the third patient death emerged.
Source: https://apnews.com/article/sarepta-muscular-dystophy-death-fda-gene-therapy-756b7ef43591b685fc197d6acb29c4c1