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Second Patient Dies from Sarepta's Duchenne Therapy, Shares Plunge 40%

Bio Tech

Business Honor
17 June, 2025

Sarepta faces scrutiny after Elevidys treatment causes fatal liver failure in patients.

Sarepta Therapeutics, a Cambridge, Massachusetts, biotechnology firm, has reported the second death of a patient who had been administered its gene therapy for Duchenne muscular dystrophy (DMD). The patient died from acute liver failure caused by the treatment, Elevidys, a single infusion intended to treat the root cause of DMD, an ultra-rare and life-ending disease in young boys. The initial patient passed away in March after the same therapy.

Following the second death, Sarepta's shares declined by over 40% and the company temporarily suspended shipments of Elevidys to some patients. It also suspended a clinical trial as it attempts to determine the reasons behind these reactions and review its treatment procedures.

Elevidys, priced at $3.2 million per patient, has already been given to more than 900 patients. The treatment was approved in 2023 using the FDA's accelerated approval program, designed to give life-saving therapies to those with few other options. Its safety, however, was questioned, especially the possibility of causing liver failure, when it was approved.

The death has put families of Duchenne patients in a difficult situation, as they now have to weigh the danger of treatment against the advancement of the disease. Chief Scientific Officer at CureDuchenne, a patient organization, Michael Kelly said that the risk-benefit ratio has greatly shifted after these incidents.

Sarepta's Chief Scientific Officer, Louise Rodino-Klapac, expressed the fatalities and indicated that the company was collaborating with specialists to understand and prevent more instances of liver failure. The FDA has also taken note and is conducting a high-level review of the therapy.

As Sarepta continues to probe, the future of Elevidys hangs in the balance, and the company is under greater scrutiny regarding the approval process of its gene therapy.