{"id":32122,"date":"2025-07-18T20:17:47","date_gmt":"2025-07-18T20:17:47","guid":{"rendered":"https:\/\/medexperts.pro\/?p=32122"},"modified":"2025-07-18T20:25:56","modified_gmt":"2025-07-18T20:25:56","slug":"fda-to-ask-sarepta-to-stop-shipping-muscular-dystrophy-drug-elevidys","status":"publish","type":"post","link":"https:\/\/medexperts.pro\/?p=32122","title":{"rendered":"FDA to Ask Sarepta to Stop Shipping Muscular Dystrophy Drug Elevidys"},"content":{"rendered":"
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Three patients with a muscle-wasting disease died from liver failure after taking the therapy, Ele\u00advidys, or a similar treatment.<\/p>\n

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The Food and Drug Administration will request that the biotech company Sarepta Therapeutics halt all shipments of its treatment for a deadly muscle-wasting disease after three patients died from liver failure after taking the product or a similar therapy.<\/p>\n

The troubled treatment, known as Ele\u00advidys, has fueled an ongoing debate over whether the F.D.A. has grown too lenient in approving drugs that are insufficiently safe or effective. New officials in the Trump administration, however, have rejected several new<\/a> drug applications<\/a> and narrowed the use of Covid vaccines over concerns about \u201cunknown\u201d side effects<\/a>.<\/p>\n

Ele\u00advidys, a gene therapy, is administered as a one-time intravenous infusion. It is intended to slow the progression of Duchenne mus\u00adcu\u00adlar dy\u00ads\u00adtro\u00adphy, which causes muscles to deteriorate. The disease often kills patients, typically young men, before their 30th birthday.<\/p>\n

Ele\u00advidys, first approved in 2023, is one of a handful of treatments for the disease that arrived in the past decade<\/a>, with the help of fierce advocacy from patients and their families. But the approval of Ele\u00advidys was controversial because of limited evidence that its benefits outweighed its risks.<\/p>\n<\/div>\n<\/div>\n

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In recent months, two teenage boys who had received Ele\u00advidys died of liver failure, deepening critics\u2019 concerns about the drug and prompting speculation that the F.D.A. would take strict action. In late June, the F.D.A. announced an investigation<\/a> into the deaths. Liver injuries were among the side effects<\/a> seen in clinical trials of the treatment.<\/p>\n

On Tuesday, the company said<\/a> it would halt shipments for patients with more advanced disease who use wheelchairs, a group that includes the teenagers who died and most Duchenne patients over age 12. At the time, the company planned to keep distributing the product for patients who could still walk.<\/p>\n

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Three patients with a muscle-wasting disease died from liver failure after taking the therapy, Ele\u00advidys, or a similar treatment.The Food and Drug Administration will request that the biotech company Sarepta Therapeutics halt all shipments of its treatment for a deadly muscle-wasting disease after three patients died from liver failure after taking the product or a similar therapy.The troubled treatment, known as Ele\u00advidys, has fueled an ongoing debate over whether the F.D.A. has grown too lenient in approving drugs that are insufficiently safe or effective. New officials in the Trump administration, however, have rejected several new drug applications and narrowed the use of Covid vaccines over concerns about \u201cunknown\u201d side effects.Ele\u00advidys, a gene therapy, is administered as a one-time intravenous infusion. It is intended to slow the progression of Duchenne mus\u00adcu\u00adlar dy\u00ads\u00adtro\u00adphy, which causes muscles to deteriorate. The disease often kills patients, typically young men, before their 30th birthday.Ele\u00advidys, first approved in 2023, is one of a handful of treatments for the disease that arrived in the past decade, with the help of fierce advocacy from patients and their families. But the approval of Ele\u00advidys was controversial because of limited evidence that its benefits outweighed its risks.In recent months, two teenage boys who had received Ele\u00advidys died of liver failure, deepening critics\u2019 concerns about the drug and prompting speculation that the F.D.A. would take strict action. In late June, the F.D.A. announced an investigation into the deaths. Liver injuries were among the side effects seen in clinical trials of the treatment.On Tuesday, the company said it would halt shipments for patients with more advanced disease who use wheelchairs, a group that includes the teenagers who died and most Duchenne patients over age 12. At the time, the company planned to keep distributing the product for patients who could still walk.We are having trouble retrieving the article content.Please enable JavaScript in your browser settings.Thank you for your patience while we verify access. If you are in Reader mode please exit and\u00a0log into\u00a0your Times account, or\u00a0subscribe\u00a0for all of The Times.Thank you for your patience while we verify access.Already a subscriber?\u00a0Log in.Want all of The Times?\u00a0Subscribe.<\/p>\n","protected":false},"author":1,"featured_media":32124,"comment_status":"close","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[],"class_list":["post-32122","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science"],"_links":{"self":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/32122","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=32122"}],"version-history":[{"count":2,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/32122\/revisions"}],"predecessor-version":[{"id":32125,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/32122\/revisions\/32125"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/media\/32124"}],"wp:attachment":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=32122"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=32122"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=32122"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}