WASHINGTON — On Friday, the Food and Drug Administration said it would add a new warning and other restrictions to gene therapy for Duchenne muscular dystrophy it was due to two patient deaths.
Sarepta Therapeutics' infusion therapy will carry a boxed warning (the most severe type) warning doctors and patients of the risk of potentially fatal liver failure with the treatment, the FDA said.
One-time therapy with Elevidis is taken. FDA inspection as the company reported the first of two deaths of teenage boys in March. After reporting a second death in June, the FDA briefly called for a halt to all supplies of the drug. But the agency is fast reverse course faced with resistance from patients' families and libertarian activists close to President Donald Trump.
Elevidis is the first drug approved in the United States. gene therapy for Duchenne muscular dystrophy, a fatal muscle-wasting disease that affects boys and young men.
In addition to the boxed warning, the FDA is also limiting the drug's approved use to patients 4 years of age and older who are still able to walk. Previously, the FDA approved the use of the drug in immobile patients, who usually have more advanced disease.
The new labeling will also recommend weekly monitoring of liver function during the first three months of treatment, as well as other precautions.
Elevidis is Sarepta's best-selling product, and recent headwinds against the drug have weighed heavily on the company and its stock. In July, the Cambridge, Massachusetts-based company announced it would lay off 500 employees.
Sarepta Therapeutics Inc. shares rose 7.7% in trading following the FDA announcement, reflecting investors' improved understanding of the company's prospects.
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