¡¾ÌáÒªÐÎò¡¿¿ËÈÕ£¬¹«Ë¾Ö÷´ò²úÆ·Ö®Ò»£¬ÒÒõ£µ¨¼îõ¥Ã¸ÒÖÖÆ¼Á¡¾Ê¯É¼¼î¼×¡¿ÐÂÐͿڷþÖÆ¼Á£¨Ñз¢²úÆ·´úºÅWP107£©ÖÎÁÆÈ«ÉíÐÍÖØÖ¢¼¡ÎÞÁ¦µÄÁÙ´²ÊÔÑéÉêÇë»ñµÃÃÀ¹úʳÎïÒ©Æ·¼àÊÓ¹ÜÀí¾Ö£¨FDA£©ÊÜÀí£¬½øÈëרҵÉóÆÀ½×¶Î¡£
¡¾ÌáÒªÐÎò¡¿¿ËÈÕ£¬¹«Ë¾Ö÷´ò²úÆ·Ö®Ò»£¬ÒÒõ£µ¨¼îõ¥Ã¸ÒÖÖÆ¼Á¡¾Ê¯É¼¼î¼×¡¿ÐÂÐͿڷþÖÆ¼Á£¨Ñз¢²úÆ·´úºÅWP107£©ÖÎÁÆÈ«ÉíÐÍÖØÖ¢¼¡ÎÞÁ¦µÄÁÙ´²ÊÔÑéÉêÇë»ñµÃÃÀ¹úʳÎïÒ©Æ·¼àÊÓ¹ÜÀí¾Ö£¨FDA£©ÊÜÀí£¬½øÈëרҵÉóÆÀ½×¶Î¡£
¿ËÈÕ£¬¹«Ë¾Ö÷´ò²úÆ·Ö®Ò»£¬ÒÒõ£µ¨¼îõ¥Ã¸ÒÖÖÆ¼Á¡¾Ê¯É¼¼î¼×¡¿ÐÂÐͿڷþÖÆ¼Á£¨Ñз¢²úÆ·´úºÅWP107£©ÖÎÁÆÈ«ÉíÐÍÖØÖ¢¼¡ÎÞÁ¦µÄÁÙ´²ÊÔÑéÉêÇë»ñµÃÃÀ¹úʳÎïÒ©Æ·¼àÊÓ¹ÜÀí¾Ö£¨FDA£©ÊÜÀí£¬½øÈëרҵÉóÆÀ½×¶Î¡£

×Ô2023Äê12Ô»ñµÃFDAÊÚÓèµÄʯɼ¼î¼×ÖÎÁÆÖØÖ¢¼¡ÎÞÁ¦µÄ¹Â¶ùÒ©È϶¨£¨orphan drug designation, ODD£©ÒÔÀ´£¬¹«Ë¾µÄÑз¢ÍŶÓÒ¹ÒÔ¼ÌÈյĿªÕ¹ÊÂÇ飬Íê³ÉÁËÖ§³ÖÃÀ¹úÁÙ´²ÊÔÑ鿪չµÄËùÓÐÁÙ´²Ç°ÊÂÇ飬°üÀ¨ÖÊÁÏÒ©µÄ¹ú¼Ê»¯Ò©Ñ§Ñо¿£¬ÐÂÐͿڷþÈÜÒºÖÆ¼ÁµÄ¿ª·¢¡¢Ò©Ñ§Ñо¿¡¢¹¤ÒÕÑéÖ¤¡¢Ò©ÀíÓë¶¾ÀíѧÑо¿£¬ÁÙ´²Õ½ÂÔÓëÁÙ´²¼Æ»®µÄÍýÏëÓë׫д£¬ÒÔ¼°¹ú¼Ê¹æÔòÓë×¢²áÉ걨µÄ×¼±¸¡£ÖµµÃÒ»ÌáµÄÊÇ£¬¹«Ë¾½¨ÉèÁËʵÁ¦ÐÛºñ¡¢ÖÜÈ«µÄרҵ»¯Ñз¢È˲Ų½¶Ó£¬ÔÚWP107ÏîÄ¿ÖÐ×ÔÖ÷Íê³ÉÁ˴󲿷ÖÑз¢ÊÂÇ飬²¢ÓëÏà¹Ø×¨ÒµµÄ¹ú¼Ê¡¢º£ÄÚ×ÅÃûµÄר¼ÒÍŶӺÏ×÷£¬Ê©Õ¹ÕûÌåÓÅÊÆ£¬È·±£ÏîÄ¿µÄ˳Ëì¡¢¿ìËÙ¡¢¸ßÖÊÁ¿Íƽø¡£
ÃÀ¹úFDAÐÂÒ©ÁÙ´²ÊÔÑéÉêÇëµÄÀÖ³ÉÊÜÀí£¬ÊÇ×Ô»ñµÃ¹Â¶ùÒ©È϶¨ÒÔÀ´£¬¹«Ë¾Á¢ÒìҩתÐÍ¡¢Éý¼¶µÄÓÖÒ»Àï³Ì±®¡£¹«Ë¾½«¼ÌÐø¶¦Á¦´ó¾ÙÍÆ¶¯Á¢ÒìÒ©µÄÑз¢¡¢¹ú¼Ê»¯½á¹¹£¬Æð¾¢Îª»¼Õß´øÀ´Ô½·¢Çå¾²¡¢ÓÐÓõÄÁÙ´²ÖÎÁÆÑ¡Ôñ£¬´Ó¶ø´´ÔìÓÅÒìµÄ¿µ½¡¼ÛÖµ¡¢Éç»á¼ÛÖµºÍÉÌÒµ¼ÛÖµ¡£
±ü³Ö“ÃæÏò»¼Õߣ¬ÃæÏòÌìÏ£¬ÃæÏòδÀ´”µÄÑз¢ÀíÄWP107¿Ú·þÒºµÄÈ«ÇòÁÙ´²¿ª·¢£¬ÇÐʵ¹á³¹Á˹«Ë¾½ÅÔúʵµØ×öÒ©µÄÖÊÆÓÇ黳£ºÔÚFDA¹Â¶ùÒ©È϶¨µÄ¼Ó³ÖÏ£¨ÓÈÆäÊÇÃÀ¹ú7ÄêÉÏÊкóÊг¡¶ÀÍÌȨ£©£¬²»¿ÌÒâ×·Çó¸ß¶Ë¼ÁÐÍ£¬Ñ¡Ôñ×îÊʺϻ¼ÕߵĿڷþÈÜÒº¼Á£¬³ä·Ö˼Á¿ÖØÖ¢¼¡ÎÞÁ¦»¼Õß¿ÉÄÜ·ºÆðµÄÍÌÑÊÄÑÌâµÄÇéÐΣ¬ÒÔ¼°Ï൱²¿·Ö¶ùͯ»¼ÕßµÄÓÃÒ©ÄÜÁ¦ºÍÒÀ´ÓÐÔ£¬ÔÚ¹«Ë¾ÒÑÓÐʯɼ¼î¼××¢ÉäÒºµÄ´¦·½¡¢¹¤ÒÕ»ù´¡ÉÏ£¬¶à¿ìºÃÊ¡µÄʵÏÖÁ˿ڷþÈÜÒº¼ÁµÄÖÐÊÔ¡¢ÁÙ´²Åú´ÎµÄÉú²ú²¢»ñµÃÓÅÒìµÄÎȹÌÐÔÊý¾Ý£¬Ê©Õ¹¹«Ë¾ÒÑÓеÄÖÊÁÏÒ©£«ÖƼÁµÄǿʢÊÖÒÕÓëÉú²úÄÜÁ¦£¬½öÓò»µ½Ò»Äêʱ¼ä±ãÍê³ÉËùÓÐÇкÏFDA¡¢ICH¹æÔòÒªÇóµÄËùÓÐÏà¹ØÊÂÇ飬ÌåÏÖÐÂÒ©´´ÖÆÑз¢µÄ97¹ú¼ÊËÙÂÊ¡£
¹«Ë¾ÆÚ´ý¾ÍÁÙ´²ÊÔÑé»ñµÃFDAµÄÅú×¼£¬¿ìËÙÍÆ½øWP107µÄÁÙ´²ÊÔÑéÊÂÇ飬ÔçÈÕ½«Æä´ø¸øº£ÄÚÍâÉîÊÜÖØÖ¢¼¡ÎÞÁ¦À§ÈŵϼÕß¡£
¹ØÓÚʯɼ¼î¼×
ʯɼ¼î¼×Ö÷Òª×÷ÓûúÀíΪÒÒõ£µ¨¼îõ¥Ã¸ÒÖÖÆ£¬Í¬Ê±¾ßÓп¹Ñס¢¿¹Ñõ»¯Ó¦¼¤ÓëÉñ¾±£»¤µÈ×÷Óá£ÒòÆä»úÀíÃ÷È·¡¢Çå¾²ÐԺã¬ÓÐÍûÎªÖØÖ¢¼¡ÎÞÁ¦»¼Õß´øÀ´¸üÓŵÄÁÙ´²Ñ¡Ôñ¡£
97¹ú¼ÊÖÆÒ©¼¯ÍÅÓµÓÐʯɼ¼î¼×ÖÊÁÏÒ©µÄÅú×¼Îĺţ¬ÊǺ£ÄÚÖ÷ÒªµÄʯɼ¼î¼×ÖÊÁÏÒ©¹©Ó¦ÉÌ¡£¹«Ë¾º£ÄÚʯɼ¼î¼××¢ÉäÒºÒѾÉÏÊУ¬²¢ÓÐʯɼ¼î¼×¿ØÊÍÆ¬ÕýÔÚ¿ªÕ¹ÖÎÁư¢¶û´Äº£Ä¬Ö¢µÄÁÙ´²Ñо¿¡£
´Ë´ÎINDµÄÉ걨£¬×÷Ϊ¹«Ë¾Æð¾¢ÍØÕ¹Ê¯É¼¼î¼×˳Ӧ֢ÁìÓò¡¢½«ÕâÒ»ÖйúÔÁ¢ÒìÒ©ÍÆÏò¹ú¼Ê»¯µÄÓÖÒ»³É¼¨£¬³ÉΪ¹«Ë¾Õë¶Ôʯɼ¼î¼×µÄмÁÐÍ¡¢ÐÂ˳Ӧ֢¡¢ÐÂÊг¡µÈÁ¢ÒìÐԽṹµÄÖ÷Òª×é³É²¿·Ö¡£
¹ØÓÚÖØÖ¢¼¡ÎÞÁ¦
ÖØÖ¢¼¡ÎÞÁ¦£¨MG£©ÊÇÒ»ÖÖÓÐÊýµÄÂýÐÔ×ÔÉíÃâÒßÐÔÉñ¾¼¡Èâ¼²²¡£¬¿ÉÄÜ»áÑÏÖØ²¢Î£¼°ÉúÃü£¬µ¼ÖÂÒò¼¡ÈâÎÞÁ¦¶øÖ²У¬ÉõÖÁÒòºôÎüË¥½ß¶øéæÃü¡£ÖØÖ¢¼¡ÎÞÁ¦µÄÁÙ´²ÑÏÖØË®Æ½¿É´ÓÉÔ΢µÄÑÛ²¿Ö¢×´£¨ÀýÈçÑÛíúÏ´¹¡¢¸´ÊÓ£©µ½ºôÎüºÍÑÓËèΣÏó£¬Î£¼°ÉúÃü£¬ÐèÒªÖØÖ¢¼à»¤¡¢Í¨ÆøÖ§³ÖºÍ³¦ÄÚÎ¹Ñø¡£ÆäËûÖ¢×´¿ÉÄܰüÀ¨ÒòÃæ²¿¼¡ÈâÎÞÁ¦¶øÎÞ·¨¾ÙÐÐÃæ²¿ÐÄÇé¡¢ÍÌÑÊ»òÓïÑÔÄÑÌ⣬ÒÔ¼°ÉíÌåÆäËû²¿Î»£¨Èç¾±²¿¡¢ÉÏÖ«ºÍÏÂÖ«£©¼¡ÈâÎÞÁ¦¡£Ô¼Äª20%µÄ»¼ÕßÖ»»á·ºÆðÑÛ²¿ÎÞÁ¦£¨³ÆÎªÑÛ¼¡ÐÍMG£©£¬¶øÆäËûÈ«ÉíÐÔÖØÖ¢¼¡ÎÞÁ¦£¨gMG£©»¼ÕßÒ²»á·ºÆðÉíÌåÆäËû²¿Î»µÄÎÞÁ¦¡£ÔÚÈ«Çò¹æÄ£ÄÚ£¬MG »¼²¡ÂÊÔ¤¼ÆÎªÃ¿ 10 ÍòÈËÖÐÓÐ12.4Àý¡£ÃÀ¹úÖØÖ¢¼¡ÎÞÁ¦»ù½ð»áÔ¤¼Æ£¬ÃÀ¹úÖØÖ¢¼¡ÎÞÁ¦µÄ»¼²¡ÂÊԼΪÿ10ÍòÈËÖÐ14ÖÁ20Àý£¬Ï൱ÓÚÌìÏÂÔ¼36,000ÖÁ60,000 Àý¡£×î½üµÄÒ»·Ý±¨¸æÔ¤¼Æ£¬ÃÀ¹úµÄ×Ü»¼²¡ÂÊÒª¸ßµÃ¶à£¬µÖ´ïÿ10ÍòÈË37.0ÈË¡£È»¶ø£¬ÓÉÓÚMGÈÔδ»ñµÃ³ä·ÖÕï¶Ï£¬ÏÖʵÌìÏÂÖеϼ²¡ÂÊ¿ÉÄÜÈÔÈ»½Ï¸ß¡£
¹«Ë¾µÄʯɼ¼î¼×¿Ú·þÈÜÒº£¬ÓÐÍûΪ»¼ÕßÌṩÖÎÁÆÊ±¼ä¸ü³¤¡¢Çå¾²ÐÔ¸üºÃµÄÖÎÁÆÑ¡Ôñ¡£ÌìϹæÄ£ÄÚ£¬È«ÇòÖØÖ¢¼¡ÎÞÁ¦ÖÎÁÆÊг¡Ô¤¼Æµ½2032Ä꽫µÖ´ï67ÒÚÃÀÔªÒÔÉÏ¡¾ref¡¿£¬ÆäÖб±ÃÀÊÇÖØÖ¢¼¡ÎÞÁ¦µÄ×î´óÊг¡¡£
ɨ¶þάÂëÓÃÊÖ»ú¿´
Ïà¹ØÍÆ¼ö