
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
The Red Sea strategy: What does Israel stand to gain from recognizing Somaliland? - 2
Journalists killed by Israeli strike in southern Lebanon - 3
How Seniors Can Use Refunds and Motivators to Purchase a Hyundai Ioniq EV - 4
The Response Uncovered: Disentangling the Secrets of the Universe - 5
Hamas Navy head, engineer of Khan Yunis tunnel network killed in Gaza, IDF confirms
The Best Computer games Ever
Giant ‘toothed’ birds flew over Antarctica 40 million to 50 million years ago
A Timeline of Rising Antisemitism in Australia
Getting ready for a Mechanized World: 10 Positions That computer based intelligence Could Dominate
New movies to watch this weekend: See 'Predator: Badlands' in theaters, rent 'Black Phone 2,' stream Guillermo del Toro's 'Frankenstein' on Netflix
Jason Kelce opens about wife Kylie Kelce's past pregnancy loss
Students were skipping my astrophysics class to play video games – so I turned the class itself into a video game
Discussion on deployment of foreign troops ongoing, two sources tell 'Post'
IDF destroys two-kilometer-long Gaza terror tunnel in Beit Lahiya













