Patient dies after muscular dystrophy -therapy, reports Sarepta

Washington (AP) – Sarepta Therapeutics said on Tuesday that a patient died when he took his closely observed gene therapy for muscular dystrophy and sends the company’s shares in morning trade.

The young man died of an acute liver injury, a well -known side effect, said Sarepta in an explanation. However, the company said that the “severity” of the patient’s case had not been observed beforehand during the therapy called Elefidys. It is the first known patient death with the therapy used in more than 800 patients, the company said.

In 2023, Elefidys received an accelerated US approval, although some scientists from Food and Drug Administration on his effectiveness in the treatment of Duchenne muscle dystrophy. It is the first gene therapy that was approved in the USA for the rare muscle -consuming state that causes weakness, loss of mobility and early death in men.

Last year, the FDA gave the patients of Duchenne full approval with a certain genetic mutation and expanded their use to patients from 4 years, regardless of whether they can still run. Before that, it was only available for younger patients who were still mobile.

In a statement, Sarepta said that the patient who died had an infection recently contributed to the liver injury. The company said it plans to update the regulations for ELEDYS to reflect the case.

The companies based in Cambridge, Massachusetts, fell by more than 23% to around $ 78 per share in the morning trade.

Elevidys uses a disabled virus to insert a replacement gene for the production of dystrophin in patient cells. It costs 3.2 million US dollars for one-time treatment.

Sarepta has received an accelerated FDA approval for three medication from Duchenne since 2016. Nobody was confirmed for the work. Studies to secure the full FDA approval have not yet been completed.

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