{"id":4182,"date":"2025-07-23T18:14:43","date_gmt":"2025-07-23T15:14:43","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4182"},"modified":"2025-07-24T15:39:40","modified_gmt":"2025-07-24T12:39:40","slug":"fda-designates-avidity-biosciences-delpacibart-zotadirsen-del-zota-as-a-breakthrough-therapy-for-the-treatment-of-dmd-in-individuals-with-mutations-that-allow-for-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/fda-designates-avidity-biosciences-delpacibart-zotadirsen-del-zota-as-a-breakthrough-therapy-for-the-treatment-of-dmd-in-individuals-with-mutations-that-allow-for-exon-44-skipping\/","title":{"rendered":"FDA \u5c06 Avidity Biosciences&#039; Delpacibart Zotadirsen (del-zota) \u6307\u5b9a\u4e3a\u6cbb\u7597\u643a\u5e26\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7a81\u53d8\u7684 DMD \u60a3\u8005\u7684\u7a81\u7834\u6027\u7597\u6cd5"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences \u4eca\u5929\u5ba3\u5e03\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u5df2\u6388\u4e88 delpacibart zotadirsen (del-zota) \u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u643a\u5e26\u53ef\u8fdb\u884c\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7684\u7a81\u53d8 (DMD44) \u60a3\u8005\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Del-zota \u76ee\u524d\u6b63\u5728\u9488\u5bf9\u60a3\u6709 DMD44 \u7684\u4eba\u8fdb\u884c\u7b2c 2 \u9636\u6bb5 EXPLORE44 \u5f00\u653e\u6807\u7b7e\u6269\u5c55 (EXPLORE44-OLE\u2122) \u8bd5\u9a8c\u8bc4\u4f30\uff0c\u8fd9\u662f\u8be5\u516c\u53f8\u4e3a DMD \u5f00\u53d1\u7684\u591a\u79cd AOC \u4e2d\u7684\u7b2c\u4e00\u4e2a\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD \u662f\u4e00\u79cd\u7f55\u89c1\u7684\u9057\u4f20\u75c5\uff0c\u5176\u7279\u5f81\u662f\u7531\u4e8e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4e22\u5931\u800c\u5bfc\u81f4\u7684\u8fdb\u884c\u6027\u808c\u8089\u635f\u4f24\u548c\u65e0\u529b\uff0c\u8fd9\u79cd\u75be\u75c5\u901a\u5e38\u5728\u5f88\u5c0f\u7684\u65f6\u5019\u5c31\u5f00\u59cb\u4e86\u3002 <strong>Del-zota \u65e8\u5728\u5c06\u78f7\u9178\u4e8c\u9170\u80fa\u5417\u5549\u5be1\u805a\u4f53 (PMO) \u8f93\u9001\u5230\u9aa8\u9abc\u808c\u548c\u5fc3\u810f\u7ec4\u7ec7\uff0c\u4ee5\u4e13\u95e8\u8df3\u8fc7\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u7b2c 44 \u4e2a\u5916\u663e\u5b50\u5e76\u5b9e\u73b0\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u3002<\/strong> - \u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/avidity-biosciences-announces-positive-topline-del-zota-data-showing-consistent-statistically-significant-improvements-in-dystrophin\/\" target=\"_blank\" rel=\"noreferrer noopener\">Avidity Biosciences \u516c\u5e03 Del-zota \u7684\u6b63\u9762\u9876\u7ebf\u6570\u636e\uff0c\u663e\u793a\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u6301\u7eed\u3001\u7edf\u8ba1\u4e0a\u663e\u8457\u6539\u5584<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity\u9996\u5e2d\u533b\u7597\u5b98Steve Hughes\u533b\u5b66\u535a\u58eb\u8868\u793a\uff1a\u201c\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u8fdb\u4e00\u6b65\u51f8\u663e\u4e86FDA\u5bf9del-zota\u5728\u89e3\u51b3DMD44\u6839\u672c\u75c5\u56e0\u65b9\u9762\u7684\u5de8\u5927\u6f5c\u529b\u4ee5\u53ca\u4e3aDMD\u60a3\u8005\u7fa4\u4f53\u63d0\u4f9b\u521b\u65b0\u6cbb\u7597\u65b9\u6848\u7684\u8feb\u5207\u9700\u6c42\u7684\u8ba4\u53ef\u3002\u57281\/2\u671fEXPLORE44\u8bd5\u9a8c\u4e2d\uff0c\u6211\u4eec\u770b\u5230\u5305\u62ec\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5728\u5185\u7684\u591a\u79cd\u751f\u7269\u6807\u5fd7\u7269\u53d6\u5f97\u4e86\u663e\u8457\u4e14\u6301\u7eed\u7684\u6539\u5584\uff0c\u6211\u4eec\u81f4\u529b\u4e8e\u5c3d\u5feb\u5c06del-zota\u5e26\u7ed9DMD44\u60a3\u8005\uff0c\u5e76\u6309\u8ba1\u5212\u57282025\u5e74\u5e95\u63d0\u4ea4\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7(BLA)\u3002 \u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5df2\u5b8c\u6210\u7684\u9488\u5bf9DMD44\u60a3\u8005\u76841\/2\u671fEXPLORE44\u00ae\u8bd5\u9a8c\u4e2d\uff0cdel-zota\u663e\u793a\u51fa\u5916\u663e\u5b50\u8df3\u8dc3\u663e\u8457\u589e\u52a0\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210\u663e\u8457\u589e\u52a0\u3001\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u663e\u8457\u4e14\u6301\u7eed\u964d\u4f4e\u81f3\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73\uff0c\u5e76\u4e14\u59cb\u7ec8\u5177\u6709\u826f\u597d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u3002Avidity\u8ba1\u5212\u4e8e2025\u5e74\u7b2c\u56db\u5b63\u5ea6\u516c\u5e03\u6b63\u5728\u8fdb\u884c\u4e14\u5df2\u5168\u90e8\u5165\u7ec4\u76842\u671fEXPLORE44-OLE\u8bd5\u9a8c\u7684\u9876\u7ebf\u6570\u636e\u548c\u529f\u80fd\u6570\u636e\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u53ef\u901a\u8fc7\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u7a81\u53d8\u548c\u7f3a\u5931<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u516c\u53f8\u4ecd\u6309\u8ba1\u5212\u4e8e2025\u5e74\u5e95\u63d0\u4ea4\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7\uff08BLA\uff09\u3002Avidity\u516c\u53f8\u6b63\u5728\u4e3aFDA\u83b7\u6279\u540e\uff0c\u5728\u7f8e\u56fd\u63a8\u51fa\u7528\u4e8e\u6cbb\u7597DMD44\u7684del-zota\u8fdb\u884c\u5546\u4e1a\u5316\u51c6\u5907\u3002Del-zota\u7684\u9884\u671f\u4e0a\u5e02\u4e3aAvidity\u540e\u7eed\u63a8\u51fa\u5176\u4ed6\u795e\u7ecf\u808c\u8089\u9879\u76ee\u5960\u5b9a\u4e86\u57fa\u7840\uff0c\u4f8b\u5982\u7528\u4e8e\u6cbb\u75971\u578b\u5f3a\u76f4\u6027\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DM1\uff09\u7684del-desiran\u548c\u7528\u4e8e\u6cbb\u7597\u9762\u80a9\u80b1\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08FSHD\uff09\u7684del-brax\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u662f\u4e00\u79cd\u65e8\u5728\u52a0\u5feb\u5f00\u53d1\u548c\u5ba1\u67e5\u7528\u4e8e\u6cbb\u7597\u4e25\u91cd\u75be\u75c5\u7684\u836f\u7269\u7684\u8fc7\u7a0b\uff0c\u521d\u6b65\u4e34\u5e8a\u8bc1\u636e\u8868\u660e\u8be5\u836f\u7269\u53ef\u80fd\u5728\u4e34\u5e8a\u91cd\u8981\u7ec8\u70b9\u4e0a\u6bd4\u73b0\u6709\u7597\u6cd5\u6709\u663e\u8457\u6539\u5584\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9664\u4e86\u83b7\u5f97\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u5916\uff0cdel-zota \u6b64\u524d\u8fd8\u88ab FDA \u548c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u6388\u4e88\u5b64\u513f\u836f\u8ba4\u5b9a\uff0c\u5e76\u88ab FDA \u6388\u4e88\u7528\u4e8e\u6cbb\u7597 DMD44 \u7684\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u548c\u5feb\u901f\u901a\u9053\u8ba4\u5b9a\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/upcoming-exon-44-skipping-therapies-for-dmd\/\">\u5373\u5c06\u63a8\u51fa\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7597\u6cd5<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44). Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension (EXPLORE44-OLE\u2122) trial for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4185,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,141,127,246,299,359],"class_list":["post-4182","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44","tag-full-length-dystrophin"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4182","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=4182"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4182\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4185"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4182"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4182"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4182"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}