WASHINGTON (AP) – Salepta Therapeutics (SRPT) said Tuesday that the patient died while undergoing closely monitored gene therapy due to muscular dystrophy and will send shares in the company that will plummet in morning trading.
The young man died of acute liver damage, a known side effect, Salepta said in a statement. However, the company said the “severity” of patient cases was not previously seen in a treatment called Elevidys. The company said this was the first known patient death from treatments used in more than 800 patients.
2:52:18 PM EDT. Market open.
In 2023, Elevidi received US approval despite concerns from some Food and Drug Administration scientists about its effectiveness in the treatment of Duchenne muscular dystrophy. It is the first gene therapy approved in the US for rare muscle sucking conditions that cause male weakness, loss of mobility and early death.
Last year, the FDA granted full approval for patients with Duchenne with certain genetic mutations and expanded its use to patients over the age of four, regardless of whether they are still able to walk. Previously, it was only available to young patients who were still mobile.
In a statement, Salepta said the deceased patient had a recent infection that could contribute to liver damage. The company said it plans to update its prescription information for Elebidi to reflect the case.
Massachusetts-based Cambridge shares fell more than 23% per share in morning trading to around $78.
Elevidys uses the impaired virus to insert replacement genes into patient cells to generate dystrophin. A one-off treatment costs $3.2 million.
Sarepta has been FDA accelerated approval for three Duchenne drugs since 2016. The work has not been confirmed yet. Research is currently underway designed to ensure full FDA approval.
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