The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne\u2019s muscular dystrophy that\u2019s been linked to two patient deaths. <\/p>\n
<\/p>\n
By MATTHEW PERRONE, Associated Press Health Writer<\/p>\n
WASHINGTON (AP) \u2014 <\/p>\n
The infused therapy from Sarepta Therapeutics will carry a boxed warning \u2014 the most serious type \u2014 alerting doctors and patients to the risk of potentially fatal liver failure with the treatment, the FDA said in a release.
\nThe 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.<\/p>\n
Elevidys is the first U.S.-approved gene therapy for Duchenne\u2019s muscular dystrophy, a fatal muscle-wasting disease that affects boys and young men.
\nIn addition to the boxed warning, the FDA is also limiting the drug\u2019s approved use to patients who are 4 years old and up and can still walk. Previously the FDA had allowed the drug\u2019s use in immobile patients, who generally have more advanced disease.
\nNew labeling will also recommend weekly liver function monitoring for the first three months of treatment, as well as other precautionary steps.<\/p>\n
Elevidys is Sarepta\u2019s 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.
\nSarepta Therapeutics Inc. shares rose 7.7% in trading after the FDA announcement, reflecting improved visibility for investors about the company\u2019s outlook.<\/p>\n
The FDA’s decision to add a boxed warning to Elevidys highlights the ongoing challenges and risks associated with gene therapies, particularly for severe and life-threatening conditions like Duchenne\u2019s muscular dystrophy. While the therapy offers hope for patients and their families, it’s crucial that both the benefits and risks are carefully considered and monitored.<\/p>\n
Elevidys is a gene therapy approved for the treatment of Duchenne\u2019s muscular dystrophy, a fatal muscle-wasting disease.<\/p>\n
The FDA has added a boxed warning, the most serious type, alerting to the risk of potentially fatal liver failure with the treatment.<\/p>\n
The drug\u2019s approved use is limited to patients who are 4 years old and up and can still walk, and new labeling recommends weekly liver function monitoring for the first three months of treatment.<\/p>\n
Sarepta Therapeutics Inc. shares rose 7.7% in trading after the FDA announcement, reflecting improved visibility for investors about the company\u2019s outlook.<\/p>\n","protected":false},"excerpt":{"rendered":"
FDA Adds Strongest Warning to Sarepta Gene Therapy Introduction to the Warning The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne\u2019s muscular dystrophy that\u2019s been linked to two patient deaths. By MATTHEW PERRONE, Associated Press Health Writer WASHINGTON (AP) \u2014 Background on […]<\/p>\n","protected":false},"author":3,"featured_media":24368,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"rank_math_lock_modified_date":false,"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[22],"tags":[],"class_list":{"0":"post-24366","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-health-wellness"},"jetpack_featured_media_url":"https:\/\/i2.wp.com\/www.dailynews.com\/wp-content\/uploads\/2025\/11\/MED-Duchennes-Therapy_98956.jpg?w=1400px&strip=all&w=1400&resize=1400,933&ssl=1","jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/posts\/24366"}],"collection":[{"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/comments?post=24366"}],"version-history":[{"count":1,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/posts\/24366\/revisions"}],"predecessor-version":[{"id":24369,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/posts\/24366\/revisions\/24369"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/media\/24368"}],"wp:attachment":[{"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/media?parent=24366"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/categories?post=24366"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/citystuff.com\/los-angeles\/wp-json\/wp\/v2\/tags?post=24366"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}