WASHINGTON (AP) – Sarepta Therapeutics said on Tuesday that a patient died while he was closely observing Genetic therapy to attend musclesSending the shares of the company decreased in morning trading.
In a statement, I said in a statement that the young man died due to an acute liver injury, which is known side effects. But the company said that the “intensity” of the patient’s case was not previously seen with the treatment, which is called Qualitys. The company said it was the first known patient death with treatment, which was used in more than 800 patients.
In 2023, Elevidys received the US expedited approval despite fears from some food and drug management scientists about its effectiveness in treating muscle atrophy Duchenne. It is the first genetic treatment approved in the United States for a rare muscle condition, which causes weakness and loss of mobility and early death in males.
The Food and Drug Administration (FDA) granted full approval last year to Duchene patients with a specific genetic mutation, expanding its use of patients 4 or larger, regardless of whether they are still able to walk. Previously, it was only available for younger patients who were still mobile.
Sacripa said in a statement that the patient who died suffering from a recent infection could have contributed to the injury of the liver. The company said it plans to update the information described for Quistidys to reflect the case.
The shares of Cambridge, based in Massachusetts, decreased by more than 23 % to about $ 78 per share in morning trading.
Elevidys is used by a disabled virus to insert an alternative gene to produce guarantees in the patient’s cells. It costs $ 3.2 million for one -time treatment.
SAREPTA receives FDA’s acceleration approval threelast Duchene medications since 2016. Nothing has been confirmed to work; Studies designed to ensure full FDA approval.
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