
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
In wrangling dark matter, some scientists find inspiration in the Torah, Krishna and Christ - 2
Coca-Cola Co. and bottlers to invest in South Africa operations - 3
The cheap health insurance promoted by Trump officials has this catch - 4
Catch the moon dancing with bright star Regulus tonight - 5
1,000-mile Saharan dust storm, from the sky and from the ground
NASA wants to build a base on the Moon by the 2030s – how and why it plans to build up to a long-term lunar presence
6 Modest and Strong Tire Brands
Turkey’s intel chief lays out country’s vision for Middle East, world
Whale stranded in the Baltic Sea swims free again. It still faces a tough task
Manual for extravagance SUVs for seniors
Space Condos to Lift Your Metropolitan Living
Should you get an RSV vaccine this fall? What to know and where to get a shot
Attacks on Iran's Bushehr nuclear plant and on Iraq border crossing
Poland identifies two Ukrainian suspects in railway sabotage blast












