{"id":4794,"date":"2025-10-13T16:14:23","date_gmt":"2025-10-13T13:14:23","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4794"},"modified":"2025-11-03T20:17:29","modified_gmt":"2025-11-03T17:17:29","slug":"avidity-biosciences-announces-positive-pre-bla-meeting-with-u-s-fda-for-del-zota-in-dmd44-with-a-submission-planned-for-q1-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/avidity-biosciences-announces-positive-pre-bla-meeting-with-u-s-fda-for-del-zota-in-dmd44-with-a-submission-planned-for-q1-2026\/","title":{"rendered":"Avidity Biosciences \u5ba3\u5e03\u4e0e\u7f8e\u56fd FDA \u5c31 DMD44 \u4e2d\u7684 del-zota \u4e3e\u884c\u79ef\u6781\u7684 BLA \u524d\u4f1a\u8bae\uff0c\u8ba1\u5212\u4e8e 2026 \u5e74\u7b2c\u4e00\u5b63\u5ea6\u63d0\u4ea4"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences\u5ba3\u5e03\uff0c\u516c\u53f8\u5df2\u4e0e\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u5c31\u5373\u5c06\u63d0\u4ea4\u7684delpacibart zotadirsen\uff08del-zota\uff09\u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7\uff08BLA\uff09\u9884\u5ba1\u4f1a\u8bae\uff08FDA\uff09\u987a\u5229\u5b8c\u6210\u3002Del-zota\u5df2\u83b7\u5f97\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\uff0c\u662f\u4e00\u6b3e\u5728\u7814\u836f\u7269\uff0c\u76ee\u524d\u6b63\u5728\u8bc4\u4f30\u5176\u4f5c\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD44\uff09\u60a3\u8005\u7684\u6f5c\u5728\u6cbb\u7597\u65b9\u6848\uff0c\u8be5\u60a3\u8005\u643a\u5e26\u53ef\u8fdb\u884c\u5916\u663e\u5b5044\u8df3\u8dc3\u57fa\u56e0\u7a81\u53d8\u7684\u57fa\u56e0\u7a81\u53d8\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u9002\u5408\u5916\u663e\u5b5044\u8df3\u8dc3\u7597\u6cd5\u7684\u7a81\u53d8\u548c\u7f3a\u5931<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">BLA \u63d0\u4ea4\u7684\u65f6\u95f4\u5df2\u4ece\u4e4b\u524d\u7684 2025 \u5e74\u5e95\u66f4\u65b0\u4e3a 2026 \u5e74\u7b2c\u4e00\u5b63\u5ea6\uff0c\u4ee5\u786e\u4fdd FDA \u5728\u63d0\u4ea4\u65f6\u6536\u5230\u989d\u5916\u7684\u6570\u636e\u6765\u652f\u6301\u5316\u5b66\u3001\u5236\u9020\u548c\u63a7\u5236 (CMC) \u5305\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity Biosciences \u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98 Sarah Boyce \u8868\u793a\uff1a\u201c\u6211\u4eec\u6700\u8fd1\u4e0e FDA \u7684\u4f1a\u8bae\u9ad8\u5ea6\u534f\u4f5c\uff0c\u4e3a\u6211\u4eec\u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA) \u63d0\u4ea4\u63d0\u4f9b\u4e86\u6e05\u6670\u7684\u63a8\u8fdb\u8def\u5f84\u3002\u5bf9\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u800c\u8a00\uff0c\u7f8e\u56fd\u7684\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u662f\u5c06 del-zota \u5e26\u7ed9\u6709\u9700\u8981\u7684\u60a3\u8005\u7684\u6700\u4f73\u4e14\u6700\u5feb\u6377\u7684\u65b9\u5f0f\u3002\u6211\u4eec\u975e\u5e38\u611f\u8c22 FDA \u5c31\u5176\u63d0\u4ea4\u65f6\u6240\u9700\u7684 CMC \u6570\u636e\u7ed9\u4e88\u4e86\u660e\u786e\u7684\u6307\u5bfc\uff0c\u6211\u4eec\u76f8\u4fe1\u8fd9\u79cd\u534f\u8c03\u5c06\u6709\u52a9\u4e8e BLA \u6d41\u7a0b\u7684\u6210\u529f\u3002\u6211\u4eec\u975e\u5e38\u611f\u8c22 FDA \u5185\u90e8\u591a\u4e2a\u804c\u80fd\u90e8\u95e8\u656c\u4e1a\u3001\u52e4\u52c9\u7684\u5ba1\u6838\u4eba\u5458\uff0c\u4ed6\u4eec\u4f7f\u6211\u4eec\u80fd\u591f\u4ee5\u5e94\u6709\u7684\u7d27\u8feb\u611f\u63a8\u8fdb\u8be5\u9879\u76ee\u3002\u6211\u4eec\u77e5\u9053\uff0c\u5bf9\u4e8e\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u7537\u5b69\u548c\u5e74\u8f7b\u7537\u6027\u6765\u8bf4\uff0c\u6bcf\u4e00\u5206\u949f\u90fd\u81f3\u5173\u91cd\u8981\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity \u5bf9 del-zota \u7684\u6f5c\u529b\u5145\u6ee1\u4fe1\u5fc3\uff0c\u5e76\u671f\u5f85\u5728 2026 \u5e74\u7b2c\u4e00\u5b63\u5ea6\u63d0\u4ea4 del-zota \u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA)\u3002\u8fd9\u5c06\u662f\u516c\u53f8\u8ba1\u5212\u5728 12 \u4e2a\u6708\u5185\u63d0\u4ea4\u7684\u4e09\u4efd BLA \u7533\u8bf7\u4e2d\u7684\u9996\u4efd\u3002\u6b64\u5916\uff0cAvidity \u6b63\u5728\u7ee7\u7eed\u51c6\u5907\u4e00\u9879\u9a8c\u8bc1\u6027\u7814\u7a76\uff0c\u4ee5\u652f\u6301 del-zota \u7684\u5168\u7403\u5168\u9762\u6279\u51c6\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\uff08\u6240\u6709\u7814\u7a76\u5217\u8868\uff09<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences announced that the Company completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota). Del-zota, which received Breakthrough Therapy designation, is an investigational drug being evaluated as a potential treatment for people living with Duchenne muscular dystrophy who have gene mutations [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4796,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,129,141,127,246,88],"class_list":["post-4794","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-dmd44","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-fda"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4794","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=4794"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4794\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4796"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4794"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4794"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4794"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}