
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
Unwind: Four Extraordinary Spa Resorts On the planet - 2
4 Energy-Proficient Clothes washers to Consider in 2024 - 3
Overlooked infertility care should be part of national health services, says WHO - 4
Presenting Nintendo's New Pastel Satisfaction Con Tones for Switch Gamers: 3 Upscale Choices - 5
35 million tons of food go to waste yearly in the US. Experts share tips to help stop it
The race to mine the moon is on – and it urgently needs some clear international rules
NASA's Artemis 2 astronauts are cruising to the moon. So why are they doing CPR tests today?
7 Strange Devices to Make Your Party Stick Out!
Historical mysteries solved by science in 2025
Would you ever turn to AI for companionship? 6% of Americans say they could — or already have.
Shah Capital pushes for Novavax sale, warns of proxy fight
Mojtaba Khamenei unconscious in Qom, not actually running Iran
James Webb Space Telescope watches our Milky Way galaxy's monster black hole fire out a flare
They want better health care — so they're turning to crypto startups













