{"id":5428,"date":"2022-07-07T00:03:13","date_gmt":"2022-07-07T00:03:13","guid":{"rendered":"\/\/smallcapscn.x5view.co\/?p=5428"},"modified":"2023-08-29T17:54:43","modified_gmt":"2023-08-29T17:54:43","slug":"kazia-therapeutics-awarded-us-fda-rare-drug-designation-paxalisib-childhood-brain-cancer","status":"publish","type":"post","link":"https:\/\/smallcapscn.x5view.co\/?p=5428","title":{"rendered":"Kazia Therapeutics \u6388\u4e88\u7f8e\u56fd FDA \u7f55\u89c1\u836f\u7269\u6307\u5b9a\u7528\u4e8e\u6cbb\u7597\u513f\u7ae5\u8111\u764c\u7684 paxalisib"},"content":{"rendered":"<p>Kazia Therapeutics (ASX: KZA) \u7684\u4e3b\u8981\u5019\u9009\u836f\u7269 paxalisib \u5df2\u88ab\u6388\u4e88\u7f55\u89c1\u7684\u513f\u79d1\u75be\u75c5\u6307\u5b9a (RPDD)\uff0c\u7528\u4e8e\u6cbb\u7597\u79f0\u4e3a\u975e\u5178\u578b\u6a2a\u7eb9\u808c\u6837\u7578\u80ce\u7624 (AT\/RT) \u7684\u9ad8\u5ea6\u4fb5\u88ad\u6027\u513f\u7ae5\u8111\u764c\u3002<\/p>\n<p>\u8be5\u6307\u5b9a\u7531\u7f8e\u56fd\u98df\u54c1\u548c\u836f\u7269\u7ba1\u7406\u5c40 (US Food and Drug Administration; FDA) \u4e0b\u8fbe\uff0c\u8fd9\u610f\u5473\u7740\u5982\u679c paxalisib \u6700\u521d\u88ab\u6279\u51c6\u7528\u4e8e\u764c\u75c7\uff0cKazia \u53ef\u80fd\u6709\u6743\u83b7\u5f97\u513f\u79d1\u4f18\u5148\u5ba1\u67e5\u51ed\u8bc1 (pPRV)\u3002<\/p>\n<p>pPRV \u5141\u8bb8\u6301\u6709\u4eba\u5bf9\u65b0\u836f\u7533\u8bf7\u8fdb\u884c\u4e3a\u671f\u516d\u4e2a\u6708\u7684\u52a0\u6025\u5ba1\u67e5\u3002<\/p>\n<p>\u8fd9\u4e9b\u4ee3\u91d1\u5238\u662f\u53ef\u4ea4\u6613\u7684\uff0c\u5386\u53f2\u4e0a\u7684\u4ef7\u683c\u8d85\u8fc7 1.46 \u4ebf\u6fb3\u5143\u3002<\/p>\n<p>Paxalisib \u6b64\u524d\u4e8e 6 \u6708\u88ab\u7f8e\u56fd FDA \u6388\u4e88\u7528\u4e8e <a href=\"http:\/\/smallcaps-production.test\/kazia-therapeutics-us-fda-orphan-drug-designation-paxalisib-childhood-brain-cancer\/?lang=cn\">AT\/RT \u7684\u5b64\u513f\u836f\u6307\u5b9a (ODD)<\/a>\u3002<\/p>\n<h2>\u4e34\u5e8a\u524d\u6570\u636e<\/h2>\n<p>FDA \u6388\u4e88 RPDD \u4e4b\u524d\uff0cKazia \u5728\u7f8e\u56fd\u764c\u75c7\u7814\u7a76\u534f\u4f1a (American Association of Cancer Research \uff1bAACR) 4 \u6708\u4e3e\u884c\u7684\u5e74\u5ea6\u4f1a\u8bae\u4e0a\u4ecb\u7ecd\u4e86 paxalisib \u6cbb\u7597 AT\/RT \u7684\u6709\u5e0c\u671b\u7684\u4e34\u5e8a\u524d\u6570\u636e\u3002<\/p>\n<p>Kazia \u9996\u5e2d\u6267\u884c\u5b98 James Garner \u535a\u58eb\u8868\u793a\uff0c\u8fd9\u8bc1\u660e\u4e86\u513f\u7ae5\u8111\u764c\u5728\u6574\u4e2a paxalisib \u5f00\u53d1\u8ba1\u5212\u4e2d\u7684\u91cd\u8981\u6027\u3002<\/p>\n<p>\u201c\u8111\u764c\u662f\u513f\u7ae5\u764c\u75c7\u6b7b\u4ea1\u7684\u6700\u5e38\u89c1\u539f\u56e0\uff0c\u4f46\u8bb8\u591a\u5f62\u5f0f\u7684[\u8fd9\u79cd\u75be\u75c5]\u7684\u7ed3\u679c\u51e0\u5341\u5e74\u6765\u90fd\u6ca1\u6709\u6539\u5584\uff0c\u201d\u4ed6\u8bf4\u3002<\/p>\n<p>\u201c\u6211\u4eec\u975e\u5e38\u5e0c\u671b paxalisib \u80fd\u591f\u5bf9\u53d7 DIPG\uff08\u5f25\u6f2b\u6027\u8111\u6865\u8111\u80f6\u8d28\u7624\uff09\u548c AT\/RT \u5f71\u54cd\u7684\u5bb6\u5ead\u4ea7\u751f\u5f71\u54cd\uff0c\u6211\u4eec\u5c06\u4e0e\u4e34\u5e8a\u533b\u751f\u3001\u7814\u7a76\u4eba\u5458\u548c FDA \u5bc6\u5207\u5408\u4f5c\uff0c\u4ee5\u786e\u5b9a\u63a8\u52a8\u836f\u7269\u5411\u524d\u53d1\u5c55\u7684\u6700\u4f73\u65b9\u6cd5\u3002 \u201d<\/p>\n<h2>\u836f\u54c1\u540d\u79f0<\/h2>\n<p>\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u6307\u5b9a\u53ef\u6388\u4e88\u6b63\u5728\u5f00\u53d1\u7684\u7528\u4e8e\u6cbb\u7597\u4ee5\u4e0b\u75be\u75c5\u7684\u836f\u7269\uff1a \u4e3b\u8981\u5f71\u54cd\u513f\u7ae5\uff0818 \u5c81\u4ee5\u4e0b\uff09\uff1b\u4e25\u91cd\u6216\u5371\u53ca\u751f\u547d\uff1b\u5728\u7f8e\u56fd\u6bcf\u5e74\u62a5\u544a\u7684\u65b0\u75c5\u4f8b\u5c11\u4e8e 200,000 \u4f8b\u3002<\/p>\n<p>\u5177\u6709 RPDD \u7684\u836f\u7269\u7684\u7533\u529e\u8005\u53ef\u4ee5\u5728\u4e0a\u5e02\u7533\u8bf7\u65f6\u5411 FDA \u7d22\u53d6\u4f18\u5148\u5ba1\u8bc4\u51ed\u8bc1\u3002<\/p>\n<p>\u51ed\u5355\u6709\u6548\u5730\u5c06 FDA \u7684\u5ba1\u67e5\u671f\u4ece 12 \u4e2a\u6708\u7f29\u77ed\u5230 6 \u4e2a\u6708\u3002<\/p>\n<p>\u5bf9\u4e8e\u4e00\u5bb6\u63a8\u51fa\u5341\u4ebf\u6fb3\u5143\u836f\u7269\u7684\u5927\u516c\u53f8\u6765\u8bf4\uff0c\u4e3a\u671f\u516d\u4e2a\u6708\u7684\u52a0\u901f\u76d1\u7ba1\u5ba1\u67e5\u53ef\u80fd\u5177\u6709\u5de8\u5927\u7684\u7ecf\u6d4e\u4ef7\u503c\u3002<\/p>\n<p>2019 \u5e74\uff0cFDA \u6388\u4e88\u4e86\u4e94\u4e2a\u513f\u79d1 PRV\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Kazia Therapeutics (ASX: KZA) \u7684\u4e3b\u8981\u5019\u9009\u836f\u7269 paxalisib \u5df2\u88ab\u6388\u4e88\u7f55\u89c1\u7684\u513f\u79d1\u75be\u75c5\u6307\u5b9a (RPDD)\uff0c\u7528\u4e8e\u6cbb\u7597\u79f0\u4e3a\u975e\u5178\u578b\u6a2a\u7eb9\u808c\u6837\u7578\u80ce\u7624 (AT\/RT) \u7684\u9ad8\u5ea6\u4fb5\u88ad\u6027\u513f\u7ae5\u8111\u764c\u3002 \u8be5\u6307\u5b9a\u7531\u7f8e\u56fd\u98df\u54c1\u548c\u836f\u7269\u7ba1\u7406\u5c40 (US Food and Drug Administration; FDA) \u4e0b\u8fbe\uff0c\u8fd9\u610f\u5473\u7740\u5982\u679c paxalisib \u6700\u521d\u88ab\u6279\u51c6\u7528\u4e8e\u764c\u75c7\uff0cKazia \u53ef\u80fd\u6709\u6743\u83b7\u5f97\u513f\u79d1\u4f18\u5148\u5ba1\u67e5\u51ed\u8bc1 (pPRV)\u3002 pPRV \u5141\u8bb8\u6301\u6709\u4eba\u5bf9\u65b0\u836f\u7533\u8bf7\u8fdb\u884c\u4e3a\u671f\u516d\u4e2a\u6708\u7684\u52a0\u6025\u5ba1\u67e5\u3002 \u8fd9\u4e9b\u4ee3\u91d1\u5238\u662f\u53ef\u4ea4\u6613\u7684\uff0c\u5386\u53f2\u4e0a\u7684\u4ef7\u683c\u8d85\u8fc7 1.46 \u4ebf\u6fb3\u5143\u3002 Paxalisib \u6b64\u524d\u4e8e 6 \u6708\u88ab\u7f8e\u56fd FDA \u6388\u4e88\u7528\u4e8e AT\/RT \u7684\u5b64\u513f\u836f\u6307\u5b9a (ODD)\u3002 \u4e34\u5e8a\u524d\u6570\u636e FDA \u6388\u4e88 RPDD \u4e4b\u524d\uff0cKazia \u5728\u7f8e\u56fd\u764c\u75c7\u7814\u7a76\u534f\u4f1a (American Association of Cancer Research \uff1bAACR) 4 \u6708\u4e3e\u884c\u7684\u5e74\u5ea6\u4f1a\u8bae\u4e0a\u4ecb\u7ecd\u4e86 paxalisib \u6cbb\u7597 AT\/RT \u7684\u6709\u5e0c\u671b\u7684\u4e34\u5e8a\u524d\u6570\u636e\u3002 Kazia&hellip; <a class=\"more-link\" href=\"https:\/\/smallcapscn.x5view.co\/?p=5428\">Continue reading <span class=\"screen-reader-text\">Kazia Therapeutics \u6388\u4e88\u7f8e\u56fd FDA \u7f55\u89c1\u836f\u7269\u6307\u5b9a\u7528\u4e8e\u6cbb\u7597\u513f\u7ae5\u8111\u764c\u7684 paxalisib<\/span><\/a><\/p>\n","protected":false},"author":4,"featured_media":10095,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[3],"tags":[498],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v17.3 (Yoast SEO v21.0) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Kazia Therapeutics \u6388\u4e88\u7f8e\u56fd FDA \u7f55\u89c1\u836f\u7269\u6307\u5b9a\u7528\u4e8e\u6cbb\u7597\u513f\u7ae5\u8111\u764c\u7684 paxalisib<\/title>\n<meta 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content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Imelda Cotton\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Imelda Cotton\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/smallcapscn.x5view.co\/kazia-therapeutics-awarded-us-fda-rare-drug-designation-paxalisib-childhood-brain-cancer\/\",\"url\":\"https:\/\/smallcapscn.x5view.co\/kazia-therapeutics-awarded-us-fda-rare-drug-designation-paxalisib-childhood-brain-cancer\/\",\"name\":\"Kazia Therapeutics \u6388\u4e88\u7f8e\u56fd FDA \u7f55\u89c1\u836f\u7269\u6307\u5b9a\u7528\u4e8e\u6cbb\u7597\u513f\u7ae5\u8111\u764c\u7684 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