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The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne muscular dystrophy that has been linked to the deaths of two patients.
Sarepta Therapeutics’ infused therapy will carry a boxed warning — the most serious type — alerting doctors and patients to the risk of potentially fatal liver failure with the treatment. fda Said in a release.
The one-time therapy, Alevidis, is under FDA scrutiny after the first of two deaths in teenage boys was reported in March. After the second death reported in June, the FDA called for a summary halt to all shipments of the drug. But the agency soon changed course after facing opposition from patient families and libertarian activists close to the president. donald trump,
Alevidis is the first US-approved 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 who are 4 years of age and older and can still walk. The FDA previously approved the drug’s use in sedentary patients, whose disease is typically more advanced.
The new labeling will also recommend weekly liver function monitoring for the first three months of treatment, as well as other precautionary steps.
Alevidis is Sarepta’s best-selling product and recent headwinds against the drug have taken a huge toll on the company and its stock. In July, CambridgeThe Massachusetts-based company announced it would lay off 500 employees.
Shares of Sarepta Therapeutics Inc. rose 7.7% after the FDA announcement, reflecting better visibility for investors about the company’s outlook.
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