
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
Mobility exercises are an important part of fitness as we age. Here are some tips - 2
Journalists killed by Israeli strike in southern Lebanon - 3
Israel reports second missile fire from Yemen since start of Iran war - 4
The most effective method to Explore Moral Situations in Brain research with Your Certification - 5
Brazil passes anti-gang law allowing seized crypto to fund security forces
Poll: Most are satisfied with their health insurance, but a quarter report denials or delays
Turkiye’s Erdogan calls Israel’s Somaliland recognition ‘unacceptable’
Where should we send a real 'Hail Mary' spacecraft? A new study has the answers
Popular Home Rug Series For You
What an expert on the gut microbiome eats in a day
'Stranger Things' series finale trailer shows Hawkins gang gearing up for last battle with Vecna
Eurovision Song Contest changes voting rules after controversial allegations against Israel
Unpaid caregiving work can feel small and personal, but that doesn’t take away its ethical value
Step by step instructions to Get the Best Vehicle Rent Arrangement: Insider Tips and Systems













