
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
Hostages as leverage: Iran's secret demand aimed at crippling Israel's agriculture - 2
Figure out how to Team up with Your Auto Crash Legal advisor for Best Outcomes - 3
Building a Flourishing Business: Illustrations from Business people - 4
Ancient Pompeii construction site reveals the process for creating Roman concrete - 5
Norovirus is spreading earlier again this year, wastewater data shows
One lightly wounded after Iranian missile barrage targets northern Israel
African nations push to recognize crimes of colonialism in Algeria
This cafe takes orders in sign language. It's cherished by the Deaf community
Experience Unrivaled Sound: Top Speakers You Really want to Hear
Watch interstellar comet 3I/ATLAS make its closest approach to Earth in free livestream on Dec. 18
Moon fever hits DC as Artemis 2 rocket 'candle' lights up Washington Monument just 1 month before launch (photos)
Former school bus aide pleads guilty to assaulting 3 autistic students in Colorado
Interstellar comet 3I/ATLAS to pass closest to Earth on Friday
Ringleader of suspected human trafficking network arrested in Ethiopia













