{"id":5310,"date":"2025-12-11T16:56:58","date_gmt":"2025-12-11T13:56:58","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5310"},"modified":"2025-12-11T16:57:00","modified_gmt":"2025-12-11T13:57:00","slug":"atossa-therapeutics-receives-fda-rare-pediatric-disease-designation-for-z-endoxifen-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/atossa-therapeutics-receives-fda-rare-pediatric-disease-designation-for-z-endoxifen-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Atossa Therapeutics \u83b7\u5f97 FDA \u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 (Z)-Endoxifen"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Atossa Therapeutics \u5ba3\u5e03\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08\u201cFDA\u201d\uff09\u5df2\u6388\u4e88 (Z)-Endoxifen \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08\u201cDMD\u201d\uff09\u7684\u7f55\u89c1\u513f\u79d1\u75be\u75c5\uff08\u201cRPD\u201d\uff09\u8ba4\u5b9a\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u8fd9\u9879\u8ba4\u5b9a\u662fAtossa\u7684\u4e00\u4e2a\u91cd\u8981\u76d1\u7ba1\u91cc\u7a0b\u7891\uff0c\u6211\u4eec\u76f8\u4fe1\u8fd9\u6709\u529b\u5730\u9a8c\u8bc1\u4e86(Z)-Endoxifen\u4f5c\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6cbb\u7597\u836f\u7269\u7684\u6f5c\u529b\uff0c\u201dAtossa Therapeutics\u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98Steven Quay\u533b\u5b66\u535a\u58eb\u8868\u793a\u3002\u201c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u513f\u7ae5\u65f6\u671f\u6700\u5177\u7834\u574f\u6027\u7684\u75be\u75c5\u4e4b\u4e00\u3002\u60a3\u8005\u5bb6\u5ead\u8feb\u5207\u9700\u8981\u9664\u7c7b\u56fa\u9187\u548c\u57fa\u56e0\u9776\u5411\u7597\u6cd5\u4e4b\u5916\u7684\u66f4\u597d\u9009\u62e9\u3002\u867d\u7136\u80bf\u7624\u5b66\u4ecd\u7136\u662f\u6211\u4eec\u7684\u6838\u5fc3\u5173\u6ce8\u70b9\uff0c\u4f46\u8fd9\u4e00\u91cc\u7a0b\u7891\u51f8\u663e\u4e86(Z)-Endoxifen\u4f5c\u4e3a\u5e73\u53f0\u7597\u6cd5\u5728\u764c\u75c7\u548c\u7f55\u89c1\u75c5\u9886\u57df\u7684\u6f5c\u529b\uff0c\u4e3a\u901a\u8fc7\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u9879\u76ee\u521b\u9020\u6f5c\u5728\u7684\u975e\u7a00\u91ca\u6027\u4ef7\u503c\u6253\u5f00\u4e86\u5927\u95e8\u3002\u201d<\/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=\"#what-is-endoxifen\">Endoxifen\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#why-endoxifen-in-dmd\">\u4e3a\u4ec0\u4e48DMD\u4e2d\u4f7f\u7528Endoxifen\uff1f<\/a><\/li><li><a href=\"#unmet-need-and-treatment-landscape\">\u672a\u6ee1\u8db3\u7684\u9700\u6c42\u548c\u6cbb\u7597\u73b0\u72b6<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-endoxifen\">Endoxifen\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Endoxifen \u662f\u4e00\u79cd\u7814\u7a76\u6027\u5c0f\u5206\u5b50\u7597\u6cd5\uff0c\u4e5f\u662f\u4ed6\u83ab\u6614\u82ac\u7684\u4e3b\u8981\u6d3b\u6027\u4ee3\u8c22\u7269\uff0c\u4ed6\u83ab\u6614\u82ac\u662f\u4e00\u79cd\u9009\u62e9\u6027\u96cc\u6fc0\u7d20\u53d7\u4f53\u8c03\u8282\u5242 (SERM)\uff0c\u5df2\u5e7f\u6cdb\u7528\u4e8e\u5176\u4ed6\u9002\u5e94\u75c7\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7531\u4e8e\u5185\u591a\u6614\u82ac\u76f4\u63a5\u4e0e\u96cc\u6fc0\u7d20\u53d7\u4f53\u7ed3\u5408\uff0c\u56e0\u6b64\u5b83\u6709\u53ef\u80fd\u8c03\u8282\u4e0e\u808c\u8089\u5065\u5eb7\u76f8\u5173\u7684\u901a\u8def\uff0c\u5305\u62ec\u808c\u8089\u751f\u957f\u3001\u4fee\u590d\u548c\u7ea4\u7ef4\u5316\u3002Atossa\u516c\u53f8\u6b63\u5728\u5f00\u53d1\u4e00\u79cd\u53e3\u670d\u5185\u591a\u6614\u82ac\u5236\u5242\uff0c\u65e8\u5728\u5b9e\u73b0\u7a33\u5b9a\u7684\u836f\u7269\u66b4\u9732\uff0c\u5e76\u65b9\u4fbf\u5730\u6bcf\u65e5\u4e00\u6b21\u7ed9\u836f\uff0c\u76ee\u524d\u8be5\u5236\u5242\u6b63\u5904\u4e8e\u6301\u7eed\u7684\u7814\u53d1\u548c\u8bc4\u4f30\u9636\u6bb5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Endoxifen \u5c1a\u672a\u83b7\u51c6\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u6216\u4efb\u4f55\u5176\u4ed6\u808c\u8425\u517b\u4e0d\u826f\u75c7\u3002\u5176\u7528\u4e8e\u8fd9\u4e9b\u7528\u9014\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u5c1a\u672a\u786e\u5b9a\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-endoxifen-in-dmd\">\u4e3a\u4ec0\u4e48DMD\u4e2d\u4f7f\u7528Endoxifen\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9aa8\u9abc\u808c\u5bf9\u6fc0\u7d20\u4fe1\u53f7\uff08\u5305\u62ec\u96cc\u6fc0\u7d20\u53d7\u4f53\u901a\u8def\uff09\u6709\u53cd\u5e94\uff0c\u8fd9\u4e9b\u4fe1\u53f7\u53ef\u4ee5\u5f71\u54cd\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u808c\u8089\u518d\u751f\u4e0e\u4fee\u590d<\/li>\n\n\n\n<li>\u708e\u75c7\u548c\u6c27\u5316\u5e94\u6fc0<\/li>\n\n\n\n<li>\u7ea4\u7ef4\u5316\u548c\u7ec4\u7ec7\u91cd\u5851<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u9009\u62e9\u6027\u96cc\u6fc0\u7d20\u53d7\u4f53\u8c03\u8282\u5242\uff08SERMs\uff09\u5df2\u5728\u808c\u8089\u4e2d\u663e\u793a\u51fa\u751f\u7269\u6d3b\u6027\uff0c\u76ee\u524d\u6b63\u5728\u5404\u79cd\u795e\u7ecf\u808c\u8089\u75be\u75c5\u9886\u57df\u8fdb\u884c\u63a2\u7d22\u3002. <strong>\u4f5c\u4e3a\u4e00\u79cd\u5f3a\u6548\u6d3b\u6027\u4ee3\u8c22\u7269\uff0c\u5185\u591a\u6614\u82ac\u53ef\u80fd\u5177\u6709\u4ee5\u4e0b\u4f5c\u7528\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u808c\u8089\u7ec4\u7ec7\u4e2d\u96cc\u6fc0\u7d20\u53d7\u4f53\u7684\u76f4\u63a5\u8c03\u8282<\/li>\n\n\n\n<li>\u53e3\u670d\u7ed9\u836f\u7684\u6f5c\u529b\uff0c\u5982\u679c\u6700\u7ec8\u8bc1\u5b9e\u5b89\u5168\u6709\u6548\uff0c\u5219\u652f\u6301\u957f\u671f\u7ed9\u836f\u3002<\/li>\n\n\n\n<li>\u8fd9\u662f\u4e00\u79cd\u4e0e\u7a81\u53d8\u65e0\u5173\u7684\u673a\u5236\uff0c\u8fd9\u610f\u5473\u7740\u539f\u5219\u4e0a\u53ef\u4ee5\u5728\u66f4\u5e7f\u6cdb\u7684DMD\u60a3\u8005\u7fa4\u4f53\u4e2d\u8fdb\u884c\u7814\u7a76\uff0c\u800c\u65e0\u9700\u8003\u8651\u6f5c\u5728\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7a81\u53d8\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Atossa \u7684\u8ba1\u5212\u65e8\u5728\u901a\u8fc7\u4e34\u5e8a\u524d\u7814\u7a76\u4ee5\u53ca\uff08\u5982\u679c\u5408\u9002\uff09\u672a\u6765\u7684\u4e34\u5e8a\u7814\u7a76\uff0c\u4e25\u683c\u8bc4\u4f30\u8fd9\u4e00\u79d1\u5b66\u539f\u7406\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"unmet-need-and-treatment-landscape\">\u672a\u6ee1\u8db3\u7684\u9700\u6c42\u548c\u6cbb\u7597\u73b0\u72b6<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524dDMD\u7684\u6cbb\u7597\u901a\u5e38\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u76ae\u8d28\u7c7b\u56fa\u9187\u53ef\u4ee5\u5e2e\u52a9\u5ef6\u7f13\u75be\u75c5\u8fdb\u5c55\uff0c\u4f46\u4f1a\u5e26\u6765\u4e25\u91cd\u7684\u526f\u4f5c\u7528\u3002<\/li>\n\n\n\n<li>\u7a81\u53d8\u7279\u5f02\u6027\u7597\u6cd5\uff0c\u4f8b\u5982\u5916\u663e\u5b50\u8df3\u8dc3\u6216\u57fa\u56e0\u9776\u5411\u7597\u6cd5\uff0c\u4ec5\u9002\u7528\u4e8e\u643a\u5e26\u7279\u5b9a\u57fa\u56e0\u53d8\u5f02\u7684\u60a3\u8005\u3002<\/li>\n\n\n\n<li>\u652f\u6301\u6027\u62a4\u7406\uff0c\u5305\u62ec\u5fc3\u810f\u548c\u547c\u5438\u7cfb\u7edf\u7ba1\u7406\u3001\u7269\u7406\u6cbb\u7597\u548c\u9aa8\u79d1\u6cbb\u7597<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\u663e\u7136\u9700\u8981\u66f4\u591a\u901a\u7528\u7684\u6cbb\u7597\u65b9\u6848\uff0c\u8fd9\u4e9b\u65b9\u6848\u65e2\u53ef\u5355\u72ec\u4f7f\u7528\uff0c\u4e5f\u53ef\u4e0e\u73b0\u6709\u7597\u6cd5\u8054\u5408\u4f7f\u7528\u3002\u5982\u679c\u4e00\u79cd\u4e0d\u9488\u5bf9\u7279\u5b9a\u57fa\u56e0\u7a81\u53d8\u7684\u53e3\u670d\u7597\u6cd5\u88ab\u8bc1\u5b9e\u5b89\u5168\u6709\u6548\uff0c\u5219\u6709\u671b\u60e0\u53ca\u66f4\u5e7f\u6cdb\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u7fa4\u4f53\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5173\u6ce8\u6b64\u9875\u9762<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6240\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Atossa Therapeutics announced that the U.S. Food and Drug Administration (&#8220;FDA&#8221;) has granted Rare Pediatric Disease (&#8220;RPD&#8221;) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (&#8220;DMD&#8221;). &#8220;This designation is an important regulatory milestone for Atossa, and we believe a strong validation of the science supporting the potential of (Z)-Endoxifen as a treatment for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5312,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[461,462,464,463],"class_list":["post-5310","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-z-endoxifen","tag-atossa-therapeutics","tag-endoxifen","tag-tamoxifen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5310","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=5310"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5310\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5312"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5310"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5310"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5310"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}