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囊性纤维化(CF)新药!美国FDA批准Kalydeco:用于4-6个月婴儿,治疗疾病根本原因!

2020-09-28 00:46:50生物谷
核心提示:Vertex制药公司是囊性纤维化(CF)治疗领域的全球领导者。近日宣布,该公司宣布,美国食品和药物管理局(FDA)已批准Kalydeco(ivacaftor),用于治疗4-6个月大的囊性纤维化(CF)婴儿患者,具体为:基于临床和/或体外试验数据,在囊性纤维化跨膜电导调节子(CFTR)基因中存在至少一个对Kalydeco治疗有反应的CF婴儿患者。

  VertexZHIYAOGONGSISHINANGXINGXIANWEIHUA(CF)ZHILIAOLINGYUDEQUANQIULINGDAOZHE。JINRIXUANBU,GAIGONGSIXUANBU,MEIGUOSHIPINHEYAOWUGUANLIJU(FDA)YIPIZHUNKalydeco(ivacaftor),YONGYUZHILIAO4-6GEYUEDADENANGXINGXIANWEIHUA(CF)YINGERHUANZHE,JUTIWEI:JIYULINCHUANGHE/HUOTIWAISHIYANSHUJU,ZAINANGXINGXIANWEIHUAKUAMODIANDAODIAOJIEZI(CFTR)JIYINZHONGCUNZAIZHISHAOYIGEDUIKalydecoZHILIAOYOUFANYINGDECFYINGERHUANZHE。

  ZHIDEYITIDESHI,KalydecoSHIDIYIGEYESHIWEIYIYIGEZHILIAONIANLINGDIZHI4GEYUEYINGERHUANZHECFGENBENBINGYINDEYAOWU。ZAIMEIGUOHEOUMENG,KalydecoZHIQIANYIBEIPIZHUN,YONGYUZHILIAO≥6GEYUEDECFHUANZHE。JUTIWEI:(1)YONGYUZHILIAOCFTRJIYINZHONGCUNZAIR117HTUBIANDE18SUIJIYISHANGCFCHENGRENHUANZHE;(2)YONGYUZHILIAOTIZHONG≥5GONGJIN、NIANLING≥6GEYUE、CFTRJIYINZHONGCUNZAIXIALIE9ZHONGTUBIANZHIYI(G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N,S549R)DECFHUANZHE。

  CICIFDAPIZHUN,JIYU24ZHOU3QIKAIFANGBIAOQIANANQUANDUILIE(ARRIVAL)DESHUJU,GAIDUILIEYOU6MINGNIANLINGZAI4GEYUEDAO6GEYUEZHIJIANDECFYINGERZUCHENG,TAMENDECFTRJIYINCUNZAIXIALIE10GETUBIANZHONGDEYIGE(G551D、G178R、S549N、S549R、G551S、G1244E、S1251N、S1255P、G1349DHUOR117H)。ZHEIGEDUILIEXIANSHILEYIGELEISIYUZAIJIAODADEERTONGHECHENGRENZHONGGUANCHADAODEANQUANXING。

  VertexSHOUXIZHIXINGGUANJIANZONGCAIReshma KewalramaniYIXUEBOSHIBIAOSHI:“ZICONGKalydecoZAI8NIANDUOQIANSHOUCIHUOPIYILAI,WOMENYIZHIZAICHIXUTUIJINWOMENDELINCHUANGKAIFAJIHUA,MUBIAOSHIJINZAOZHILIAONANGXINGXIANWEIHUADEGENBENBINGYIN。JINTIANDEPIZHUN,DUIHUANYOUCFDEYINGERQUNTIHENZHONGYAO,YINWEIZAOQIGANYUHEZHILIAOZHEIZHONGHUIMIEXINGDEJIANJINXINGJIBINGSHIRANGHUANZHEGENGJIANKANGZHANGSHOUDEGUANJIAN。JINTIANDEPIZHUNZHENGMINGLEWOMENYULINCHUANGHEKEXUEJIEYIDAO,WEISUOYOUKENENGCONGWOMENDEYAOWUZHONGHUOYIDECFHUANZHEZUOCHUDEBUXIENULI。”

  HUASHENGDUNDAXUEYIXUEYUANXIYATUERTONGYANJIUSUOHEERKEXIMargaret RosenfeldYIXUEBOSHIBIAOSHI:“ZAOZAI4GEYUEDASHIJIUKAISHIZHILIAONANGXINGXIANWEIHUADEGENBENYUANYIN,YOUQIANLIGAIBIANJIBINGDEJINCHENG。”

  NANGXINGXIANWEIHUA(CF)SHIYIZHONGHANJIANDE、SUODUANSHENGMINGDEYICHUANBING,YINGXIANGQUANQIUYUE7.5WANREN。CFSHIYIZHONGLEIJINDEDUOXITONGJIBING,YINGXIANGFEIBU、GANZANG、WEICHANGDAO、BIDOU、HANXIAN、YIXIANHESHENGZHIDAO。CFSHIYOUCFTRJIYINDEMOUXIETUBIANDAOZHICFTRDANBAIGONGNENGQUEXIANHUOQUESHIYINQIDE。CFTRDANBAITONGCHANGDIAOJIEXIBAOMODELIZIYUNSHU,JIYINTUBIANNENGDAOZHIDANBAICHANWUGONGNENGDEPOHUAIHUOSANGSHI。DANGXIBAOMOLIZIYUNSHUBEIZHONGDUAN,MOUXIEQIGUANZHANYETUCENGDEZHANDUJIANGBIANCHOU。GAIBINGDEYIGEZHUYAOTEZHENGSHIHUXIDAOJIJUHOUHOUDEZHANYE,DAOZHIHUXIKUNNANJIFANFUGANRAN。

  SUIRANYOUXUDUOBUTONGLEIXINGDECFTRTUBIANKEDAOZHIJIBING,DANJUEDADUOSHUCFHUANZHEZHISHAOYOUYIGEF508delTUBIAN。ZHEIXIETUBIAN,KETONGGUOJIYINJIANCEHUOJIYINFENXINGLAIQUEDING。CFTRDANBAITONGCHANGDIAOJIEXIBAOMODELIZIYUNSHU,JIYINTUBIANNENGDAOZHIDANBAICHANWUGONGNENGDEPOHUAIHUOSANGSHI。DANGXIBAOMOLIZIYUNSHUBEIZHONGDUAN,MOUXIEQIGUANZHANYETUCENGDEZHANDUJIANGBIANCHOU。GAIBINGDEYIGEZHUYAOTEZHENGSHIHUXIDAOJIJUHOUHOUDEZHANYE,DAOZHIHUXIKUNNAN、FEIBUMANXINGFANFUGANRANHEJINXINGXINGFEISUNSHANG,ZUIZHONGDAOZHISIWANG。CFHUANZHEDESIWANGNIANLINGZHONGWEISHUWEI30CHUTOU。

  KalydecoSHIQUANQIUSHOUGEBAXIANGNANGXINGXIANWEIHUA(CF)GENBENBINGYINDEYAOWU,YU2012NIANZAIMEIGUOSHOUCIHUODEPIZHUN,GAIYAOYONGYUZHILIAOCFTRJIYINZHONGCUNZAITEDINGTUBIANDEHUANZHE。GAIYAOSHIYIZHONGCFTRZENGXIAOJI,KESHIHUANZHETINEIQUEXIANXINGCFTRDANBAIFAHUIZHENGCHANGGONGNENG。GAIYAOTONGGUOKOUFUJIYAO,QIMUDESHISHICFTRDANBAIZAIXIBAOBIAOMIANDEKAIFASHIJIANGENGZHANG,YIGAISHANYANHESHUIZAIXIBAOMOSHANGDEYUNSHU,ZHEIYOUZHUYUSHUIHEHEQINGCHUQIDAOZHANYE。

  JIEZHIMUQIAN,VertexGONGSIYISHANGSHI4KUANCFYAOWU:Kalydeco(ivacaftor)、Orkabi(lumacaftor/ivacaftor)、Symdeko(tezacaftor/ivacaftorHEivacaftor)、Trikafta(elexacaftor/tezacaftor/ivacaftorHEivacaftor),QIAN3KUANYAOWUKEZHILIAOQUANQIUYUE40000LIHUANZHE,YUEZHANSUOYOUCFHUANZHEDE50%。ERZUIXINPIZHUNDESANLIANLIAOFATrikaftaKEJIANGZHILIAOFANWEIKUODADAOQUANQIU90%DECFHUANZHE。

  JINNIAN6YUE,YIYAOSHICHANGDIAOYANJIGOUEvaluatePhamraFABUBAOGAOYUCE,TrikaftaZAI2026NIANJIANGCHENGWEIQUANQIUZUICHANGXIAOTOP10YAOWUZHIYI,XIAOSHOUEJIANGDADAO87.ag娱乐网址登录YIMEIYUAN,2019-2026NIANQIJIANDENIANFUHEZENGZHANGLVDADAOJINGRENDE54.3%。(SHENGWUGUBioon.com)

  YUANWENCHUCHU:FDA approves KALYDECO? (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With CF as Early as Four Months of Age

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