
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
Scientists solve the mystery of 'impossible' merger of 'forbidden' black holes - 2
The 10 Most Progressive Logical Disclosures - 3
6 Fun Urban areas For Seniors To Travel - 4
Manageable Living: Eco-Accommodating Decisions for Regular day to day existence - 5
韓国野球が日本に追いつく日は本当に来るのか?ドジャースのキム・ヘソンが語った“日韓格差”のリアル #エキスパートトピ(金明昱) - エキスパート - Yahoo!ニュース
Step by step instructions to Keep up with the Life span of Your Kona SUV's Battery Duration
車7社、米関税で7800億減益 9月中間、3社が赤字転落(共同通信)
In the background: Visiting Notable Film Areas All over the Planet
鈴木彩艶、骨折で長期離脱の影響は? 日本代表とパルマのセリエA残留にどう響くのか #エキスパートトピ(中村大晃) - エキスパート - Yahoo!ニュース
Most loved Occasion Dish: What Makes Your Merry Table?
早くもガソリン5円値下げも!きょうからガソリン補助金10円→15円に拡大し店頭価格引き下げへ(FNNプライムオンライン(フジテレビ系))
仙台育英高校サッカー部がいじめ問題で全国大会出場を辞退。弁護士の見解や代替出場の可能性は? #エキスパートトピ(下薗昌記) - エキスパート - Yahoo!ニュース
An Extended time of Careful Nurturing: Individual Bits of knowledge on Bringing up Youngsters
the Kinds of Thailand: Decision in favor of Your Number one Thai Dish!












