{"id":4826,"date":"2025-10-21T14:58:09","date_gmt":"2025-10-21T11:58:09","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4826"},"modified":"2025-11-01T16:06:07","modified_gmt":"2025-11-01T13:06:07","slug":"why-elevidys-was-not-approved-by-the-european-medicines-agency-ema","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/","title":{"rendered":"\u4e3a\u4ec0\u4e48Elevidys\u672a\u83b7\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u6279\u51c6\uff1f"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Elevidys\uff08\u6d3b\u6027\u7269\u8d28\uff1adelandistrogene moxeparvovec\uff09\u57fa\u56e0\u7597\u6cd5\u672a\u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u6279\u51c6\u7528\u4e8e\u6cbb\u7597 3-7 \u5c81\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u513f\u7ae5\uff0c\u56e0\u4e3a\u4eba\u4eec\u5bf9\u5176\u5728\u8be5\u60a3\u8005\u7fa4\u4f53\u4e2d\u8868\u73b0\u51fa\u7684\u76ca\u5904\u5b58\u5728\u91cd\u5927\u62c5\u5fe7\u3002\uff08<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noopener\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3b\u8981\u539f\u56e0\u5982\u4e0b\uff1a<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u76ee\u5f55<\/h2><nav><ul><li><a href=\"#reasons-for-elevidys-refusal-by-the-ema\">EMA \u62d2\u7edd Elevidys&#039; \u7684\u539f\u56e0<\/a><ul><li><a href=\"#additional-context-and-concerns\">\u5176\u4ed6\u80cc\u666f\u4fe1\u606f\u548c\u62c5\u5fe7<\/a><\/li><li><a href=\"#summary\">\u6982\u62ec<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"reasons-for-elevidys-refusal-by-the-ema\">EMA \u62d2\u7edd Elevidys&#039; \u7684\u539f\u56e0<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6839\u636eEMA\u7684\u516c\u5f00\u8bc4\u4f30\uff0c\u5177\u4f53\u539f\u56e0\u5305\u62ec\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5728\u5173\u952e\u7814\u7a76\uff08\u63d0\u4ea4\u7684\u4e3b\u8981\u8bc1\u636e\uff09\u4e2d\uff0cElevidys\u7ec4\u548c\u5b89\u6170\u5242\u7ec4\u572812\u4e2a\u6708\u65f6\u7684\u8fd0\u52a8\u529f\u80fd\uff08\u4f7f\u7528\u5317\u6781\u661f\u95e8\u8bca\u8bc4\u4f30\u91cf\u8868 (NSAA) \u6d4b\u91cf\uff09\u6ca1\u6709\u7edf\u8ba1\u5b66\u663e\u8457\u6539\u5584\u3002\u572834\u5206\u5236\u91cf\u8868\u4e2d\uff0c\u5dee\u5f02\u4ec5\u4e3a0.65\u5206\uff0cEMA\u5f97\u51fa\u7ed3\u8bba\u201c\u53ef\u80fd\u662f\u5076\u7136\u7684\u201d\u3002(<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09<\/a>) <strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4ec0\u4e48\u662f\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30\uff1f<\/a><\/li>\n\n\n\n<li>\u5c3d\u7ba1\u8be5\u7597\u6cd5\u8bf1\u5bfc\u4e86\u8f83\u77ed\u5f62\u5f0f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\uff0c\u4f46\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u6c34\u5e73\u65e0\u6cd5\u53ef\u9760\u5730\u4e0e\u8fd0\u52a8\u80fd\u529b\u7684\u663e\u8457\u6539\u5584\u8054\u7cfb\u8d77\u6765\u3002\uff08<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09<\/a>)<\/li>\n\n\n\n<li>\u5373\u4f7f\u8003\u8651\u5230\u53cd\u5e94\u66f4\u597d\u7684\u60a3\u8005\u4e9a\u7ec4\uff0c\u8be5\u516c\u53f8\u4e5f\u65e0\u6cd5\u4ee4\u4eba\u4fe1\u670d\u5730\u8bc1\u660e\u8be5\u6cbb\u7597\u5728\u8fd9\u4e9b\u7fa4\u4f53\u4e2d\u63d0\u4f9b\u4e86\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u76ca\u5904\u3002\uff08<a href=\"https:\/\/www.institut-myologie.org\/en\/2025\/09\/22\/unfavorable-opinion-on-marketing-authorisation-and-suspension-of-clinical-trials-in-europe-for-elevidys-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u808c\u75c5\u7814\u7a76\u6240<\/a>)<\/li>\n\n\n\n<li>\u7531\u4e8e\u8be5\u516c\u53f8\u5df2\u7533\u8bf7\u6709\u6761\u4ef6\u4e0a\u5e02\u8bb8\u53ef (CMA)\uff0cEMA \u9002\u7528\u7684\u6807\u51c6\u662f\uff0c\u5fc5\u987b\u5b58\u5728\u79ef\u6781\u7684\u83b7\u76ca\u98ce\u9669\uff08\u5373\u83b7\u76ca\u8bc1\u636e+\u53ef\u63a7\u98ce\u9669\uff09\uff0c\u5e76\u6709\u4ee4\u4eba\u4fe1\u670d\u7684\u6570\u636e\u652f\u6301\u3002EMA \u7684\u7ed3\u8bba\u662f\uff0c\u5728\u8fd9\u79cd\u60c5\u51b5\u4e0b\uff0c\u83b7\u76ca\u5c1a\u672a\u5f97\u5230\u5145\u5206\u8bc1\u660e\uff0c\u56e0\u6b64\u62d2\u7edd\u4e86\u6388\u6743\u3002(<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09<\/a>)<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"additional-context-and-concerns\">\u5176\u4ed6\u80cc\u666f\u4fe1\u606f\u548c\u62c5\u5fe7<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5b89\u5168\u95ee\u9898\uff1a\u8be5\u836f\u7269\u4e5f\u5b58\u5728\u4e25\u91cd\u7684\u5b89\u5168\u9690\u60a3\uff0c\u5c24\u5176\u662f\u5728\u65e0\u6cd5\u5367\u5e8a\u7684\u60a3\u8005\u4e2d\u3002\u636e\u62a5\u9053\uff0c\u63a5\u53d7Elevidys\u6cbb\u7597\u7684\u65e0\u6cd5\u5367\u5e8a\u60a3\u8005\u4e2d\uff0c\u6709\u51e0\u4f8b\u56e0\u6025\u6027\u809d\u8870\u7aed\u6b7b\u4ea1\uff0c\u56e0\u6b64\u8be5\u4eba\u7fa4\u7684\u8bd5\u9a8c\u5df2\u88ab\u6682\u505c\u3002(<a href=\"https:\/\/dmdwarrior.com\/zh\/aan-report-reviews-the-state-of-evidence-on-elevidys-gene-therapy-cure-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD\u6218\u58eb<\/a>) \u867d\u7136 EMA \u62d2\u7edd\u7684\u4e3b\u8981\u539f\u56e0\u662f\u5176\u5bf9 3-7 \u5c81\u80fd\u8d70\u52a8\u7684\u513f\u7ae5\u4eba\u7fa4\u7684\u6709\u6548\u6027\uff0c\u4f46\u5b89\u5168\u4fe1\u53f7\u53ef\u80fd\u4f1a\u5bf9\u6574\u4f53\u76d1\u7ba1\u73af\u5883\u4ea7\u751f\u5f71\u54cd\u3002.<\/li>\n\n\n\n<li>\u63d0\u4ea4\u7ed9EMA\u7684\u7533\u8bf7\u9488\u5bf9\u7684\u662f3-7\u5c81\u7684\u95e8\u8bca\u60a3\u8005\u3002EMA\u8ba4\u4e3a\uff0c\u8be5\u8bd5\u9a8c\u7684\u8bbe\u8ba1\u548c\u4e3b\u8981\u7ec8\u70b9\uff08NSAA\u768412\u4e2a\u6708\u53d8\u5316\uff09\u7684\u9009\u62e9\u4e0d\u8db3\u4ee5\u8bc1\u660e\u8be5\u7ec4\u60a3\u8005\u7684\u75be\u75c5\u8fdb\u5c55\u5f97\u5230\u663e\u8457\u51cf\u7f13\u3002(<a href=\"https:\/\/www.institut-myologie.org\/en\/2025\/09\/22\/unfavorable-opinion-on-marketing-authorisation-and-suspension-of-clinical-trials-in-europe-for-elevidys-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u808c\u75c5\u7814\u7a76\u6240<\/a>)<\/li>\n\n\n\n<li>\u8be5\u7597\u6cd5\u5df2\u5728\u5176\u4ed6\u4e00\u4e9b\u5730\u533a\uff08\u4f8b\u5982\u65e5\u672c\u548c\u4e00\u4e9b\u4e2d\u4e1c\u56fd\u5bb6\uff09\u6839\u636e\u4e0d\u540c\u7684\u76d1\u7ba1\u6846\u67b6\u83b7\u5f97\u6279\u51c6\u3002\uff08<a href=\"https:\/\/dmdwarrior.com\/zh\/in-which-countries-is-elevidys-approved-is-elevidys-approved-\/\" target=\"_blank\" rel=\"noreferrer noopener\">dmdwarrior.com<\/a>)<\/li>\n\n\n\n<li>EMA \u7684\u610f\u89c1\uff08\u901a\u8fc7\u5176\u4eba\u7528\u836f\u54c1\u59d4\u5458\u4f1a (CHMP)\uff09\u5728\u6b27\u76df\u59d4\u5458\u4f1a\u505a\u51fa\u51b3\u5b9a\u4e4b\u524d\u4e0d\u5177\u6709\u6cd5\u5f8b\u7ea6\u675f\u529b\uff0c\u4f46\u5728\u5b9e\u8df5\u4e2d\uff0cCHMP \u7684\u8d1f\u9762\u610f\u89c1\u51e0\u4e4e\u603b\u662f\u5bfc\u81f4\u62d2\u7edd\u3002\uff08<a href=\"https:\/\/www.globenewswire.com\/de\/news-release\/2025\/08\/11\/3131260\/32716\/en\/sarepta-therapeutics-srpt-declines-again-on-ema-recommendation-to-refuse-elevidys-marketing-authorization-securities-class-action-pending-hagens-berman.html\" target=\"_blank\" rel=\"noreferrer noopener\">\u73af\u7403\u65b0\u95fb\u793e<\/a>)<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"summary\">\u6982\u62ec<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c3d\u7ba1Elevidys\u4ee3\u8868\u4e86\u4e00\u79cd\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u65b0\u578b\u57fa\u56e0\u6cbb\u7597\u65b9\u6cd5\uff0c\u4f46EMA\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7ed3\u8bba\u662f\uff0c\u63d0\u4ea4\u7684\u8bc1\u636e\u4e0d\u8db3\u4ee5\u8bc1\u660e\u5176\u80fd\u591f\u6539\u5584\u76ee\u6807\u513f\u7ae5\uff083-7\u5c81\uff09\u7684\u8fd0\u52a8\u529f\u80fd\uff0c\u4e14\u8be5\u4eba\u7fa4\u7684\u83b7\u76ca\u98ce\u9669\u6bd4\u5c1a\u672a\u786e\u5b9a\u3002\u56e0\u6b64\uff0c\u4ed6\u4eec\u5efa\u8bae\u62d2\u7edd\u5176\u4e0a\u5e02\u8bb8\u53ef\u3002\uff08<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/www.ema.europa.eu\/en\/documents\/assessment-report\/elevidys-epar-refusal-public-assessment-report_en.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">\u60a8\u53ef\u4ee5\u4ece\u6b64\u94fe\u63a5\u8bbf\u95ee EMA \u53d1\u5e03\u7684\u6709\u5173 Elevidys \u62d2\u7edd\u539f\u56e0\u7684\u7efc\u5408\u62a5\u544a\u3002.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>The Elevidys (active substance: delandistrogene\u202fmoxeparvovec) gene therapy was not approved by the European Medicines Agency (EMA) for the treatment of ambulatory children aged 3\u20117 years with Duchenne\u202fmuscular\u202fdystrophy (DMD) because of significant concerns about its demonstrated benefit in that patient population. (European Medicines Agency (EMA)) Here are the key reasons: Reasons for Elevidys&#8217; Refusal by the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4833,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[121,33,61,65],"class_list":["post-4826","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-gene-therapy","tag-sarepta"],"subtitle":"\u5982\u679c\u60a8\u6b63\u5728\u5bfb\u627e'Elevidys\u4e3a\u4f55\u672a\u83b7\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u6279\u51c6'\u7684\u7b54\u6848\uff0c\u90a3\u4e48\u60a8\u5e76\u4e0d\u5b64\u5355\u3002\u5728\u672c\u6587\u4e2d\uff0c\u6211\u4eec\u5c06\u63a2\u8ba8EMA\u672a\u83b7\u6279\u51c6\u7684\u5173\u952e\u539f\u56e0\uff0c\u4ee5\u53ca\u5b83\u5bf9\u60a3\u8005\u3001\u5bb6\u5ead\u4ee5\u53ca\u6b27\u76df\u57fa\u56e0\u6cbb\u7597\u672a\u6765\u7684\u5f71\u54cd\u3002.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4826","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=4826"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4826\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4833"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4826"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4826"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4826"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}