{"id":577,"date":"2022-11-11T15:10:14","date_gmt":"2022-11-11T07:10:14","guid":{"rendered":"https:\/\/xiaoenjiyao.com\/?p=577"},"modified":"2022-11-11T15:10:14","modified_gmt":"2022-11-11T07:10:14","slug":"%e5%a6%82%e4%bd%95%e8%ae%b0%e4%bd%8f%e6%89%80%e6%9c%89fda%e6%89%b9%e5%87%86%e7%94%9f%e7%89%a9%e8%8d%af%ef%bc%9f-%e7%ba%b3%e7%b1%b3%e6%8a%97%e4%bd%93-caplacizumab-%e5%8d%a1%e6%99%ae%e8%b5%9b","status":"publish","type":"post","link":"https:\/\/xiaoenjiyao.com\/?p=577","title":{"rendered":"\u5982\u4f55\u8bb0\u4f4f\u6240\u6709FDA\u6279\u51c6\u751f\u7269\u836f\uff1f| \u7eb3\u7c73\u6297\u4f53 | Caplacizumab \u5361\u666e\u8d5b\u73e0\u5355\u6297 | \u83b7\u5f97\u6027\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765caTTP"},"content":{"rendered":"\n

\u7528\u56fe\u50cf\u8bb0\u5fc6\u7684\u65b9\u6cd5\uff0c\u4e00\u4e2a\u836f\u4e00\u5f20\u56fe\uff0c\u8bb0\u4f4f\u6240\u6709FDA\u6279\u51c6\u4e0a\u5e02\u751f\u7269\u5236\u5242\u7684\u540d\u79f0\uff0c\u9776\u70b9\uff0c\u9002\u5e94\u75c7\u7b49\u4fe1\u606f\u3002<\/p>\n\n\n\n

Caplacizumab\u5361\u666e\u8d5b\u73e0\u5355\u6297\u662fAblynx\u516c\u53f8\u5f00\u53d1\u7684\u4e00\u6b3e\u7eb3\u7c73\u6297\u4f53\uff08nanobody\uff09\uff0c\u7528\u4e8e\u6cbb\u7597\u83b7\u5f97\u6027\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c\uff08aTTP\uff09\u3002<\/p>\n\n\n\n

2018\u5e74\u8d5b\u8bfa\u83f2\uff08Sanofi\uff09\u516c\u53f8\u4ee539\u4ebf\u6b27\u5143\u6536\u8d2dAblynx\uff0c\u83b7\u5f97\u8be5\u7eb3\u7c73\u6297\u4f53\u6280\u672f\u5e73\u53f0\u3002<\/p>\n\n\n\n

\u8be5\u836f2019\u5e74\u83b7\u7f8e\u56fdFDA\u6279\u51c6\u4e0a\u5e02\uff08CABLIVI\u00ae\u00a0<\/sup>caplacizumab\uff09\u3002\u56fd\u5185\u6682\u672a\u4e0a\u5e02\uff0c\u672a\u67e5\u8be2\u5230CDE\u6ce8\u518c\u7533\u62a5\u4fe1\u606f\u3002\u5728\u4e2d\u56fd\u300a\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c\u8bca\u65ad\u4e0e\u6cbb\u7597\u4e2d\u56fd\u6307\u5357\uff082022 \u5e74\u7248\uff09\u300b\u4e2d\u79f0\u5176\u4e3a\u5361\u666e\u8d5b\u73e0\u5355\u6297\u3002<\/p>\n\n\n\n

1\u3001\u4f5c\u7528\u673a\u5236<\/h2>\n\n\n\n

1993\u5e74\uff0c\u6bd4\u5229\u65f6\u79d1\u5b66\u5bb6\u5728\u81ea\u7136\u6742\u5fd7\u4e2d\u9996\u6b21\u62a5\u9053\u7f8a\u9a7c\u5916\u5468\u8840\u6db2\u4e2d\u5b58\u5728\u4e00\u79cd\u5929\u7136\u7f3a\u5931\u8f7b\u94fe\u7684\u6297\u4f53\u3002<\/p>\n\n\n\n

\u8be5\u79cd\u6297\u4f53\u53ea\u5305\u542b\u4e00\u4e2a\u91cd\u94fe\u53ef\u53d8\u533a\uff08VHH\uff09\u548c\u4e24\u4e2a\u5e38\u89c4\u7684CH2\u4e0eCH3\u533a\uff0c\u5374\u5177\u6709\u975e\u5e38\u597d\u7684\u7a33\u5b9a\u6027\u3002<\/p>\n\n\n\n

\u7eb3\u7c73\u6297\u4f53<\/strong><\/p>\n\n\n

\n
\"\"
https:\/\/www.sanofi.com\/en\/science-and-innovation\/research-and-development\/technology-platforms\/nanobody-technology-platform<\/em><\/figcaption><\/figure>\n<\/div>\n\n\n

\u91cd\u94fe\u53ef\u53d8\u533a\uff08VHH\uff09\u53ef\u5355\u72ec\u514b\u9686\u5e76\u8868\u8fbe\uff0c\u662f\u76ee\u524d\u5df2\u77e5\u53ef\u7ed3\u5408\u76ee\u6807\u6297\u539f\u7684\u6700\u5c0f\u5355\u4f4d\uff0c\u5176\u5206\u5b50\u91cf\u53ea\u670915KD\uff0c\u88ab\u79f0\u4f5c\u7eb3\u7c73\u6297\u4f53<\/strong>\u3002<\/p>\n\n\n\n

Caplacizumab \u5361\u666e\u8d5b\u73e0\u5355\u6297\u662f\u76ee\u524d\u4e0a\u5e02\u7684\u552f\u4e00\u7eb3\u7c73\u6297\u4f53\u836f\u7269\u3002<\/p>\n\n\n\n

\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c<\/strong><\/p>\n\n\n\n

\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c\uff08thromboticthrombocytopenic purpura, TTP\uff09\u4e3a\u4e00\u79cd\u5c11\u89c1\u3001\u4e25\u91cd\u7684\u8840\u6813\u6027\u5fae\u8840\u7ba1\u75c5\uff0c\u5176\u4e3b\u8981\u4e34\u5e8a\u7279\u5f81\u5305\u62ec\u5fae\u8840\u7ba1\u75c5\u6027\u6eb6\u8840\u6027\u8d2b\u8840\uff08MAHA\uff09\u3001\u8840\u5c0f\u677f\u51cf\u5c11\u3001\u795e\u7ecf\u7cbe\u795e\u75c7\u72b6\u3001\u53d1\u70ed\u548c\u80be\u810f\u53d7\u7d2f\u7b49\u3002<\/p>\n\n\n

\n
\"\"
Expert Opin Biol Ther . 2019 Nov;19(11):1127-1134.<\/em><\/figcaption><\/figure>\n<\/div>\n\n\n

\u6b63\u5e38\u72b6\u6001\u4e0b\uff1a<\/strong><\/p>\n\n\n\n

\u4eba\u4f53\u4e2d\u8840\u7ba1\u6027\u8840\u53cb\u75c5\u56e0\u5b50\uff08VWF\uff09<\/strong>\u4f1a\u81ea\u53d1\u7ed3\u5408\u8840\u5c0f\u677f\u4ea7\u751f\u8840\u5c0f\u677f\u805a\u96c6\u3002<\/p>\n\n\n\n

VWF\u88c2\u89e3\u9176\uff08ADAMTS13<\/strong>\uff09\u7684\u529f\u80fd\u50cf\u4e00\u628a\u526a\u5200\uff0c\u964d\u89e3VWF\uff0c\u907f\u514d\u8840\u5c0f\u677f\u805a\u96c6\u3002<\/p>\n\n\n\n

\u75c5\u7406\u72b6\u6001\u4e0b\uff1a<\/strong><\/p>\n\n\n\n

TTP\u7684\u53d1\u75c5\u673a\u5236\u4e3b\u8981\u6d89\u53ca ADAMTS13 \u6d3b\u6027\u7f3a\u4e4f\u3002<\/p>\n\n\n\n

\u5982\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u60a3\u8005\u5148\u5929\u6027ADAMTS13\u5408\u6210\u7f3a\u5931\u3002\u6216\u53d1\u751f\u611f\u67d3\u3001\u836f\u7269\u3001\u80bf\u7624\u3001\u81ea\u8eab\u514d\u75ab\u6027\u75be\u75c5\u3001\u9020\u8840\u5e72\u7ec6\u80de\u79fb\u690d\u7b49\u5bfc\u81f4\u60a3\u8005\u4ea7\u751fADAMTS13\u81ea\u8eab\u6297\u4f53\u3002<\/p>\n\n\n\n

\u8840\u6d46\u4e2dADAMTS13\u6d3b\u6027\u7f3a\u4e4f\u5bfc\u81f4 VWF \u4e0d\u80fd\u53ca\u65f6\u964d\u89e3\uff0cVWF\u53ef\u81ea\u53d1\u7ed3\u5408\u8840\u5c0f\u677f\uff0c\u5bfc\u81f4\u5fae\u8840\u7ba1\u5185\u8840\u6813\u5f62\u6210\u3002<\/p>\n\n\n\n

TTP\u6cbb\u7597\u836f\u7269<\/p>\n\n\n

\n
\"\"
Expert Opin Biol Ther . 2019 Nov;19(11):1127-1134.<\/em><\/figcaption><\/figure>\n<\/div>\n\n\n

\u6839\u636eTTP\u7684\u53d1\u75c5\u673a\u5236\uff0c\u6cbb\u7597\u836f\u7269\u4e3b\u8981\u6709\u4ee5\u4e0b3\u7c7b\uff1a<\/strong><\/p>\n\n\n\n

1\u3001ADAMTS13\u7684\u66ff\u4ee3\u6cbb\u7597<\/strong>\u6cbb\u7597\u6027\u8840\u6d46\u7f6e\u6362\uff0c\u66ff\u6362\u5305\u542b\u8fc7\u91cfVWF\u7684\u60a3\u8005\u8840\u6d46\u3002\u6216\u8005\u4f7f\u7528\u91cd\u7ec4\u4ebaADAMTS13\uff0c\u4eba\u4e3a\u589e\u52a0ADAMTS13\u3002<\/p>\n\n\n\n

2\u3001\u8017\u7aedB\u6dcb\u5df4\u7ec6\u80de<\/strong>\u5982\u5229\u59a5\u6614\u5355\u6297\u4f7f\u60a3\u8005B\u6dcb\u5df4\u7ec6\u80de\u8017\u7aed\uff0c\u4ece\u800c\u51cf\u5c11\u81ea\u8eab\u6297\u4f53\u7684\u4ea7\u751f\uff0c\u8fdb\u800c\u91cd\u65b0\u6fc0\u6d3bADAMTS13\u3002\u00a0\u00a0<\/p>\n\n\n\n

3\u3001Caplacizumab \u5361\u666e\u8d5b\u73e0\u5355\u6297<\/strong>\u7ade\u4e89VWF\u4e2d\u8840\u5c0f\u677f\u7ed3\u5408\u4f4d\u70b9\uff0c\u963b\u65adVWF\u4e0e\u8840\u5c0f\u677f\u7ed3\u5408\uff0c\u5bfc\u81f4\u8840\u5c0f\u677f\u4e0d\u80fd\u805a\u96c6\u3002<\/p>\n\n\n\n

2\u3001\u9002\u5e94\u75c7<\/h2>\n\n\n\n

\u5361\u666e\u8d5b\u73e0\u5355\u6297FDA\u9002\u5e94\u75c7<\/strong><\/p>\n\n\n

\n
\"\"
\u8be5\u836f\u76ee\u524d\u56fd\u5185\u6682\u672a\u6279\u51c6\u4e0a\u5e02<\/em><\/figcaption><\/figure>\n<\/div>\n\n\n

Caplacizumab \u5361\u666e\u8d5b\u73e0\u5355\u6297 FDA\u6279\u51c6\u9002\u5e94\u75c7\u4e3a:<\/p>\n\n\n\n

\u6210\u4eba\u83b7\u5f97\u6027\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c\uff08aTTP\uff09<\/p>\n\n\n\n

\u6839\u636eADAMTS13 \u7f3a\u4e4f\u673a\u5236\u4e0d\u540c\uff0cTTP\u5206\u4e3a\uff1a<\/strong><\/p>\n\n\n\n

    \n
  • \u9057\u4f20\u6027TTP\uff08cTTP\uff0c\u53c8\u79f0\u4e3aUpshaw-Schulman \u7efc\u5408\u5f81\uff09<\/li>\n<\/ul>\n\n\n\n

    cTTP\u7cfbADAMTS13 \u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u8840\u6d46ADAMTS13 \u6d3b\u6027\u7f3a\u4e4f\uff0c\u5e38\u5728\u611f\u67d3\u3001\u708e\u75c7\u6216\u598a\u5a20\u7b49\u4fc3\u53d1\u56e0\u7d20\u4e0b\u53d1\u75c5\u3002cTTP \u5448\u5e38\u67d3\u8272\u4f53\u9690\u6027\u9057\u4f20\u3002<\/p>\n\n\n\n

      \n
    • \u83b7\u5f97\u6027TTP\uff08aTTP\uff09<\/li>\n<\/ul>\n\n\n\n

      \u56e0\u60a3\u8005\u4f53\u5185\u4ea7\u751f\u6297ADAMTS13 \u81ea\u8eab\u6297\u4f53\uff0c\u6291\u5236ADAMTS13 \u6d3b\u6027\uff08\u4e2d\u548c\u6297\u4f53\uff09\u6216\u4e0eADAMTS13\u7ed3\u5408\u5f62\u6210\u6297\u539f\u6297\u4f53\u590d\u5408\u7269\u800c\u52a0\u901fADAMTS13 \u5728\u4f53\u5185\u6e05\u9664\u3002<\/p>\n\n\n\n

      3\u3001\u56fe\u50cf\u8bb0\u5fc6<\/h2>\n\n\n
      \n
      \"\"
      \u5361\u666e\u7a7a\u516c\u53f8\uff08CAPCOM\uff09\u6700\u65b03A\u5927\u4f5c\u6e38\u620f\u300a\u585e\u4f4f\u8840\u7ba1\u300b\uff0c\u73a9\u5bb6\u63a7\u5236\u7eb3\u7c73\u673a\u5668\u4eba\uff0c\u758f\u901a\u5df2\u7ecf\u88ab\u8840\u5c0f\u677f\u5835\u585e\u5e76\u4e14\u6e17\u900f\u51fa\u7d2b\u8272\u5706\u70b9\u7684\u8840\u7ba1\u3002<\/figcaption><\/figure>\n<\/div>\n\n\n

      \u5361\u666e\u7a7a\u516c\u53f8\u7684\u300a\u585e\u4f4f\u8840\u7ba1\u300b\u6e38\u620f\uff0c\u4ee3\u8868\u836f\u7269\u540d\u79f0\uff0c\u5361\u666e\u8d5b\u73e0\u5355\u6297\u3002<\/p>\n\n\n\n

      \u758f\u901a\u8840\u7ba1\uff0c\u4ee3\u8868\u836f\u7269\u4f5c\u7528\u673a\u5236\uff0c\u9632\u6b62\u8840\u5c0f\u677f\u805a\u96c6\u3002<\/p>\n\n\n\n

      \u7d2b\u8272\u5706\u70b9\u4ee3\u8868\u836f\u7269\u9002\u5e94\u75c7\uff0c\u83b7\u5f97\u6027\u8840\u6813\u6027\u8840\u5c0f\u677f\u51cf\u5c11\u6027\u7d2b\u765c\u3002<\/p>\n\n\n\n

      \u4f60\u8bb0\u4f4f\u4e86\u4e48\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"

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