
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
10 Moving Design Frill for Summer 2023 - 2
Figure out How to Upgrade Your Gold Speculation Portfolio: Vital Bits of knowledge and Strategies - 3
Instructions to Investigate Different Open Record Extra Offers Actually - 4
Becoming amazing at Systems administration: Individual and Expert Tips - 5
The Way to Recuperation: Defeating Dependence
Audits of 6 American Busssiness Class Flights
A Manual for Nations to Head out To
Vote in favor of the subject that you see as generally captivating and intelligent!
The Significance of Prenuptial Arrangements in Separation Procedures
Commonsense Ways to work on Your Funds with a Restricted Pay
Trouvez La Carte De Cr\u00e9dit Id\u00e9ale Pour Vos Besoins En Belgique
Manual for Tracking down the Immaculate Magnificence of Focal Asia
Space Condos to Lift Your Metropolitan Living
Pick Your Favored kind of salad













