
WASHINGTON (AP) — The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne’s muscular dystrophy that's been linked to two patient deaths.
The infused therapy from Sarepta Therapeutics will carry a boxed warning — the most serious type — alerting doctors and patients to the risk of potentially fatal liver failure with the treatment, the FDA said in a release.
The one-time therapy, Elevidys, has been under FDA scrutiny since the company reported the first of two deaths of teenage boys in March. Following a second death reported in June, the FDA briefly called for halting all shipments of the drug. But the agency quickly reversed course after facing pushback from patient families and libertarian activists close to President Donald Trump.
Elevidys is the first U.S.-approved gene therapy for Duchenne’s 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 old and up and can still walk. Previously the FDA had allowed the drug's use in immobile patients, who generally have more advanced disease.
New labeling will also recommend weekly liver function monitoring for the first three months of treatment, as well as other precautionary steps.
Elevidys 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 after the FDA announcement, reflecting improved visibility for investors about the company's outlook.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
LATEST POSTS
- 1
Meet Beef the bulldog, who takes slow walks with his 78-year-old friend - 2
The most effective method to Recognize a Great Lab Jewel - 3
Louisiana seeks California doctor’s extradition, testing the limits of shield laws - 4
Becoming the best at Discussion: Individual Procedures - 5
The Main 15 Applications for Efficiency and Association
Artemis II astronauts say they're "ready to go" for moon launch
Instructions to Pick the Right Gold Speculation Procedure: Exploring the Market
Bird flu poses risk of pandemic worse than COVID, France's Institut Pasteur says
German petrol stations hike prices as once-a-day rule takes effect
Fake new headlights rule steer Australian drivers astray
The Manual for Decent European Urban communities in 2024
Grasping the Elements of Medical caretaker Pay rates: Factors That Shape Your Pay
Oil, energy and food: Which countries in Europe are most exposed to higher food prices?
Etymological Investigation Disclosed: A Survey of \Dominating New Tongues\ Language Learning Application













