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RNAi疗法givosiran治疗急性肝性卟啉症的晚期研究达到主要终点

2019-03-07 不详 网络

肝性卟啉症givosiranRNAi
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Alnylam制药本周三（2019年3月6日）报道，用于治疗急性肝性卟啉症（AHP）的研究性RNAi疗法givosiran的III期研究符合其主要疗效终点和许多次要目标。

Alnylam制药本周三（2019年3月6日）报道，用于治疗急性肝性卟啉症（AHP）的研究性RNAi疗法givosiran的III期研究符合其主要疗效终点和许多次要目标。该公司表示，根据ENVISION试验的最佳结果，它计划在2019年中期向FDA提交营销申请并在欧洲提交申请。该试验随机分配了94例AHP患者，其中89例患有基因证实的急性间歇性卟啉症（AIP），这些患者接受了针对氨基乙酰丙酸合成酶1的givosiran或安慰剂治疗。该研究的主要终点为复合性卟啉症发作的年化率的降低。该研究的结果显示，与安慰剂相比，givosiran达到了降低复合性卟啉症发病年率的主要目标，并且在9个次要终点中的5个中取得了显著结果。

卟啉症是因为人体内的卟啉（Porphyrin）等物质异常累积所造成的身体病变。人体以卟啉为前驱物，透过特定酶的催化作用来制造血红素，一旦酶无法正常作用，卟啉等物质便会越积越多，最终引致卟啉症。卟啉症是一种罕见疾病，临床统计估计发生机率约30万分之1，目前仍无治愈方法，只能针对卟啉症的病征进行治疗。


原始出处：

http://www.firstwordpharma.com/node/1628182#axzz5hUFjzudx

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

评论区 (3)
#插入话题
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    2020-02-17 zhangj7108

    #主要终点#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1996944, encodeId=bcad199694465, content=<a href='/topic/show?id=b706232e831' target=_blank style='color:#2F92EE;'>#主要终点#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=81, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=23278, encryptionId=b706232e831, topicName=主要终点)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7387164, createdName=zhangj7108, createdTime=Mon Feb 17 06:14:00 CST 2020, time=2020-02-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1778473, encodeId=c8d41e7847325, content=<a href='/topic/show?id=f4c3803ed7' target=_blank style='color:#2F92EE;'>#givosiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8037, encryptionId=f4c3803ed7, topicName=givosiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=95bb39274732, createdName=zhs3895, createdTime=Thu Jul 18 06:14:00 CST 2019, time=2019-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751667, encodeId=688c1e516671b, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Mon May 27 19:14:00 CST 2019, time=2019-05-27, status=1, ipAttribution=)]
    2019-07-18 zhs3895

    #givosiran#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1996944, encodeId=bcad199694465, content=<a href='/topic/show?id=b706232e831' target=_blank style='color:#2F92EE;'>#主要终点#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=81, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=23278, encryptionId=b706232e831, topicName=主要终点)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7387164, createdName=zhangj7108, createdTime=Mon Feb 17 06:14:00 CST 2020, time=2020-02-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1778473, encodeId=c8d41e7847325, content=<a href='/topic/show?id=f4c3803ed7' target=_blank style='color:#2F92EE;'>#givosiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8037, encryptionId=f4c3803ed7, topicName=givosiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=95bb39274732, createdName=zhs3895, createdTime=Thu Jul 18 06:14:00 CST 2019, time=2019-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751667, encodeId=688c1e516671b, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Mon May 27 19:14:00 CST 2019, time=2019-05-27, status=1, ipAttribution=)]
    2019-05-27 yinxm8315

    #RNAi#

    0