
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
Illustrations Gained from a Crosscountry Excursion - 2
The most effective method to Offset Album Rates with Liquidity Needs - 3
College students are now slightly less likely to experience severe depression, research shows – but the mental health crisis is far from over - 4
Netflix's Eddie Murphy documentary explains 'Saturday Night Live' beef: 'That's why I didn't go back for years' - 5
8 Fundamental Stages: Novice's Manual for Secure Your Android with a VPN
A new mom skipped a routine appointment. An infected cut led to a devastating diagnosis
Step by step instructions to Guarantee the Strength and Life span of Your Pre-assembled Home
Interstellar comet 3I/ATLAS to pass closest to Earth on Friday
Was This Driver Simply Having Some good times Or Behaving Like An Ass?
Figure out How to Recognize Early Indications of Depressions
Kids get diseases like lupus, too. As researchers hunt better treatments, this camp brings joy
Somaliland denies trading recognition with Israel for accepting Gazans
Taylor Swift's 'The End of an Era' docuseries: Everything you need to know, plus how to watch for less
What you need to know about Trump accounts as Michael and Susan Dell donate $6 billion to the new early childhood investment program












