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欧洲委员会批准SYMKEVI联合KALYDECO治疗6-11岁囊性纤维化患儿

2020-11-28 Allan MedSci原创

囊性纤维化F508delSYMKEVI（tezacaftor / ivacaftor）KALYDECO（ivacaftor）
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欧盟委员会已批准SYMKEVI（tezacaftor / ivacaftor）联合KALYDECO（ivacaftor）的适应症扩展，以包括治疗6-11岁囊性纤维化患儿。

囊性纤维化（CF）是一种常见的遗传疾病，此病症最常影响肺脏，但也常发生于胰脏、肝脏、肾脏以及肠。长期影响包含肺部感染所导致的呼吸困难以及积痰。

制药公司Vertex今天宣布，欧盟委员会已批准SYMKEVI（tezacaftor / ivacaftor）联合KALYDECO（ivacaftor）的适应症扩展，以包括治疗6-11岁囊性纤维化患儿，其囊性纤维化跨膜电导调节剂（CFTR）基因中有两个F508del突变拷贝，或一个F508del突变拷贝和一个CFTR基因突变拷贝：P67L、R117C、L206W、R352Q、A455E、D579G、711 + 3A→G、S945L、S977F、R1070W、D1152H、2789 + 5G→A、3272-26A→G和3849 + 10kbC→T。

 Vertex首席执行官兼总裁Reshma Kewalramani博士说：“获得批准后，在欧洲具有F508del的6至11岁CF患儿将有新的治疗选择，而具有某些残留功能突变的儿童将首次有针对性的治疗选择”。

SYMKEVI（tezacaftor / ivacaftor）联合KALYDECO（ivacaftor）将立即在德国使用，并将很快在与Vertex签订了创新性长期补偿协议的国家（包括英国、丹麦和爱尔兰共和国）使用。

 

原始出处：

https://www.firstwordpharma.com/node/1778171?tsid=4

评论区 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=2019424, encodeId=323c20194245a, content=<a href='/topic/show?id=fb11630689a' target=_blank style='color:#2F92EE;'>#欧洲委员会#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=92, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63068, encryptionId=fb11630689a, topicName=欧洲委员会)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/DYAIOgq83erCiat0j36p5MejdM9yCLtcVcY8WS5VIKuCMqXFNs56U6iacZib5CJvh6mVxGERicM6ZAkl91jMgpcDrw/132, createdBy=37622500012, createdName=ms2999106592497887, createdTime=Tue Aug 31 19:35:01 CST 2021, time=2021-08-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1675941, encodeId=3c2316e5941e8, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=智智灵药, createdTime=Fri Feb 26 16:35:01 CST 2021, time=2021-02-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=906026, encodeId=112b90602679, content=学习了 ，谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=157, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201015/772d85037a2d46ba9bf4247431437625/21829fa294ce4f3da4561f683e0d0a3d.jpg, createdBy=bd6a5426009, createdName=ms1000001355695609, createdTime=Tue Dec 08 15:15:35 CST 2020, time=2020-12-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339367, encodeId=db65133936eab, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=107, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Mon Nov 30 02:35:01 CST 2020, time=2020-11-30, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621472, encodeId=a25416214e23c, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=101, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Mon Nov 30 02:35:01 CST 2020, time=2020-11-30, status=1, ipAttribution=)]
    2021-08-31 ms2999106592497887

    #欧洲委员会#

    0

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    2021-02-26 智智灵药

    #囊性#

    0

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    2020-12-08 ms1000001355695609

    学习了 ，谢谢

    0

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    2020-11-30 ms9474365431325850

    #欧洲#

    0

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    2020-11-30 windight

    #Kalydeco#

    0

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本研究发现CF兔表现出内分泌系统的病理变化。这一新的动物模型将促进CF相关胰腺内分泌功能障碍的研究，为进一步阐明CF的病理机制提供了宝贵的实验工具，还可能推动相关的药物开发。

CKJ：三联药物（ETI）治疗对成年囊性纤维化患者肾功能、容量状态及尿路结石风险因素的影响

ETI对成年囊性纤维化患者的短期肾功能无显著影响，且该药物似乎能够通过恢复汗腺中CFTR的功能来纠正因汗液过度丢失引发的继发性醛固酮增多症。

超棒的分子治疗和基因治疗研究思路！美国在研的84项囊性纤维化基金给我们这些启发（2024）

我们仅对美国国立卫生研究院（NIH）资助的在研囊性纤维化相关项目进行梳理，希望给同仁们的选题思路提供一点启发。