{"id":4252,"date":"2025-07-29T11:18:46","date_gmt":"2025-07-29T08:18:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4252"},"modified":"2025-07-29T19:56:40","modified_gmt":"2025-07-29T16:56:40","slug":"metriopharm-mp1032-receives-ema-orphan-drug-designation-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/metriopharm-mp1032-receives-ema-orphan-drug-designation-in-duchenne-muscular-dystrophy\/","title":{"rendered":"MetrioPharm \u7684 MP1032 \u83b7\u5f97\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 EMA \u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a"},"content":{"rendered":"<p class=\"wp-block-paragraph\">MetrioPharm \u4eca\u5929\u5ba3\u5e03\uff0c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u5df2\u6388\u4e88\u5176\u4e3b\u8981\u5316\u5408\u7269 MP1032 \u5b64\u513f\u836f\u8d44\u683c (ODD)\uff0c\u7528\u4e8e\u6cbb\u7597\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u513f\u7ae5\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e00\u91cc\u7a0b\u7891\u662f\u7ee7\u7f8e\u56fd FDA \u83b7\u5f97\u9aa8\u8d28\u758f\u677e\u75c7 (ODD) \u4e4b\u540e\u53d6\u5f97\u7684\uff0c\u51f8\u663e\u4e86 MP1032 \u4f5c\u4e3a\u4e00\u6d41\u7597\u6cd5\u7684\u6f5c\u529b\uff0c\u53ef\u4ee5\u6700\u5927\u9650\u5ea6\u5730\u51cf\u5c11\u5bf9\u9ad8\u5242\u91cf\u76ae\u8d28\u7c7b\u56fa\u9187\u7684\u4f9d\u8d56\uff0c\u9ad8\u5242\u91cf\u76ae\u8d28\u7c7b\u56fa\u9187\u4f1a\u5bfc\u81f4\u9aa8\u8d28\u758f\u677e\u75c7\u3001\u751f\u957f\u53d1\u80b2\u8fdf\u7f13\u548c\u4ee3\u8c22\u95ee\u9898\u7b49\u4e25\u91cd\u526f\u4f5c\u7528\u3002<\/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=\"#key-findings-of-metrio-pharm-mp-1032\">MetrioPharm MP1032 \u7684\u4e3b\u8981\u53d1\u73b0<\/a><\/li><li><a href=\"#what-is-metrio-pharm-mp-1032\">MetrioPharm MP1032 \u662f\u4ec0\u4e48\uff1f<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-findings-of-metrio-pharm-mp-1032\">MetrioPharm MP1032 \u7684\u4e3b\u8981\u53d1\u73b0<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u8ba4\u53ef\u5148\u5bfc\u5316\u5408\u7269 MP1032 \u5728\u89e3\u51b3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u9886\u57df\u672a\u6ee1\u8db3\u7684\u533b\u7597\u9700\u6c42\u65b9\u9762\u7684\u6f5c\u529b<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u8be5\u8ba4\u5b9a\u4e3a\u66f4\u5b89\u5168\u7684\u957f\u671f\u6cbb\u7597\u94fa\u5e73\u4e86\u9053\u8def<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>MP1032 \u5df2\u8bc1\u660e\u80fd\u591f\u4fdd\u6301\u808c\u8089\u529f\u80fd\u5e76\u51cf\u5c11\u708e\u75c7\uff0c\u800c\u4e0d\u4f1a\u7834\u574f\u6b63\u5e38\u7684\u7ec6\u80de\u8fc7\u7a0b<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">EMA \u7684\u5b64\u513f\u836f (ODD) \u63d0\u4f9b\u4e86\u5173\u952e\u6fc0\u52b1\u63aa\u65bd\uff0c\u5305\u62ec EMA \u63d0\u4f9b\u7684\u65b9\u6848\u534f\u52a9\u3001\u8d39\u7528\u51cf\u514d\u4ee5\u53ca\u83b7\u6279\u540e\u5728\u6b27\u76df\u957f\u8fbe 12 \u5e74\u7684\u5e02\u573a\u72ec\u5360\u6743\uff08\u7531\u4e8e DMD \u516c\u53f8\u4e13\u6ce8\u4e8e\u513f\u79d1\u6cbb\u7597\uff0c\u539f\u5148\u7684\u5e02\u573a\u72ec\u5360\u6743\u4e3a 10 \u5e74\uff09\u3002\u6b64\u6b21\u76d1\u7ba1\u9a8c\u8bc1\u57fa\u4e8e\u4ee4\u4eba\u4fe1\u670d\u7684\u4e34\u5e8a\u524d\u6570\u636e\uff0c\u8fd9\u4e9b\u6570\u636e\u8868\u660e MP1032 \u80fd\u591f\u5728\u4e0d\u5e72\u6270\u6b63\u5e38\u7ec6\u80de\u8fc7\u7a0b\u7684\u60c5\u51b5\u4e0b\u7ef4\u6301\u808c\u8089\u529f\u80fd\u5e76\u51cf\u5c11\u708e\u75c7\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9996\u5e2d\u6267\u884c\u5b98 Thomas Christ\u00e9ly \u8868\u793a\uff1a\u201cFDA \u548c EMA \u7684\u53cc\u91cd\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u4ee5\u53ca FDA \u5df2\u83b7\u5f97\u7684\u513f\u79d1\u7f55\u89c1\u75c5\u8ba4\u5b9a\uff0c\u5c06\u5bf9 MetrioPharm \u548c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u9886\u57df\u4ea7\u751f\u91cd\u5927\u5f71\u54cd\u3002MP1032 \u7684\u72ec\u7279\u673a\u5236\u2014\u2014\u8c03\u8282\u8fc7\u5ea6\u6d3b\u8dc3\u514d\u75ab\u7ec6\u80de\u4e2d\u7684\u7ebf\u7c92\u4f53\u6d3b\u6027\u6c27 (ROS)\u2014\u2014\u53ef\u4ee5\u901a\u8fc7\u5927\u5e45\u51cf\u5c11\u76ae\u8d28\u7c7b\u56fa\u9187\u5242\u91cf\u548c\u526f\u4f5c\u7528\uff0c\u6539\u53d8\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u957f\u671f\u6cbb\u7597\u65b9\u6848\u3002\u6211\u4eec\u6b63\u5728\u52a0\u5feb MP1032 \u7684 II \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u4e0e\u60a3\u8005\u56e2\u4f53\u5408\u4f5c\uff0c\u5e76\u63a2\u7d22\u5efa\u7acb\u5408\u4f5c\u4f19\u4f34\u5173\u7cfb\uff0c\u4ee5\u4fbf\u5c06\u8fd9\u9879\u7597\u6cd5\u5e26\u7ed9\u9700\u8981\u66f4\u5b89\u5168\u6cbb\u7597\u65b9\u6848\u7684\u513f\u7ae5\u3002\u9664\u4e86\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e4b\u5916\uff0c\u6211\u4eec\u5e73\u53f0\u7684\u5bbf\u4e3b\u5bfc\u5411\u65b9\u6cd5\u5df2\u5728\u81ea\u8eab\u514d\u75ab\u6027\u75be\u75c5\u751a\u81f3\u6d41\u884c\u75c5\u6cbb\u7597\u4e2d\u5c55\u73b0\u51fa\u826f\u597d\u7684\u524d\u666f\uff0c\u8fd9\u4e00\u70b9\u5df2\u7531 HERA \u8d44\u52a9\u7684 COVID-19 IIa \u671f\u7814\u7a76\u5f97\u5230\u8bc1\u5b9e\u3002\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-metrio-pharm-mp-1032\">MetrioPharm MP1032 \u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>MetrioPharm \u7684\u4e3b\u8981\u5019\u9009\u836f\u7269 MP1032 \u662f\u4e00\u79cd\u72ec\u7279\u7684\u5c0f\u5206\u5b50\uff0c\u901a\u8fc7\u89e3\u51b3\u6fc0\u6d3b\u514d\u75ab\u7ec6\u80de\u4e2d\u53d1\u751f\u7684\u4e2d\u5fc3\u81ea\u7136\u4ee3\u8c22\u8fc7\u7a0b\u6765\u9488\u5bf9\u6fc0\u6d3b\u514d\u75ab\u7ec6\u80de\u7684\u8fc7\u5ea6\u53cd\u5e94\u3002<\/strong> \u8fd9\u4e9b\u7ec6\u80de\u7684\u7ebf\u7c92\u4f53\u4f1a\u901a\u8fc7\u4fc3\u8fdb\u80fd\u91cf\u4ee3\u8c22\u6765\u5e94\u5bf9\u8fd9\u79cd\u6fc0\u6d3b\uff0c\u8fdb\u800c\u5bfc\u81f4\u8fc7\u91cf\u6d3b\u6027\u6c27 (ROS) \u7684\u4ea7\u751f\u3002MP1032 \u662f\u4e00\u79cd\u9996\u521b\u7684\u6297\u708e\u836f\u7269\uff0c\u5b83\u80fd\u591f\u964d\u4f4e\u8fc7\u91cf\u7684 ROS\uff0c\u800c\u4e0d\u4f1a\u5e72\u6270\u5176\u4ed6\u7ec6\u80de\u7684\u751f\u7406\u6027 ROS \u6c34\u5e73\uff0c\u800c\u8fd9\u4e9b ROS \u5bf9\u7ec6\u80de\u7684\u6b63\u5e38\u529f\u80fd\u81f3\u5173\u91cd\u8981\uff0c\u56e0\u6b64\u5177\u6709\u5353\u8d8a\u7684\u5b89\u5168\u6027\u3002\u4f5c\u4e3a\u5355\u836f\u7597\u6cd5\uff0cMP1032 \u7684\u6cbb\u7597\u6548\u679c\u7c7b\u4f3c\u4e8e\u76ae\u8d28\u7c7b\u56fa\u9187\uff08\u57fa\u4e8e\u53ef\u7684\u677e\u7684\u7597\u6cd5\uff09\uff0c\u4f46\u6ca1\u6709\u4e25\u91cd\u7684\u526f\u4f5c\u7528\u3002\u4e0e\u4f20\u7edf\u6297\u708e\u836f\u7269\u4e0d\u540c\uff0c\u5b83\u6a21\u62df\u4e86\u76ae\u8d28\u7c7b\u56fa\u9187\u4f5c\u4e3a\u5355\u836f\u7597\u6cd5\u7684\u76ca\u5904\uff0c\u4f46\u907f\u514d\u4e86\u5176\u6bd2\u6027\uff0c\u8fd9\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\u3001\u591a\u53d1\u6027\u786c\u5316\u75c7\u3001\u5173\u8282\u708e\u7b49\u75be\u75c5\u7684\u4e34\u5e8a\u524d\u6a21\u578b\u4e2d\u5f97\u5230\u4e86\u8bc1\u5b9e\u3002<\/p>","protected":false},"excerpt":{"rendered":"<p>MetrioPharm announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA&#8217;s ODD, underscoring MP1032&#8217;s potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4253,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[91,213,398,399,195],"class_list":["post-4252","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ema","tag-european-medicines-agency","tag-metriopharm","tag-mp1032","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4252","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=4252"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4252\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4253"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4252"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4252"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4252"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}