
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
Public Parks in the USA - 2
Toddler given just 3 years to live after strange symptoms makes full recovery - 3
Figure out How to Amplify the Resale Worth of Your Kona SUV - 4
Mummified cheetahs found in Saudi caves shed light on lost populations - 5
Where America’s CO2 emissions come from – what you need to know, in charts
Polls open in tense Uganda election amid widespread delays
Figure out How to Reveal Stowed away Open Record Rewards
The most effective method to Refresh the Infotainment Framework in the Volvo XC40
Robert Irwin on winning 'Dancing With the Stars' 10 years after sister Bindi: 'This was everything I dreamed it would be and so much more'
Yasser Abu Shabab's killing raises questions about Israel's militia strategy in Gaza
Brazilian cardinal orders a popular Catholic priest to go offline following right-wing attacks
A definitive Frozen yogurt Standoff: Which Flavor Rules?
New materials, old physics – the science behind how your winter jacket keeps you warm
New York to require social media platforms to display mental health warnings












