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RNAi疗法lumasiran获得欧盟批准治疗1型原发性高草酸尿症

2020-11-20 MedSci原创 MedSci原创

1型原发性高草酸尿症（PH1）RNAi疗法Lumasiran
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Alnylam Pharmaceuticals宣布，欧盟委员会批准了其RNAi治疗药物Oxlumo（lumasiran）用于治疗1型原发性高草酸尿症（PH1）。

Alnylam Pharmaceuticals宣布，欧盟委员会批准了其RNAi治疗药物Oxlumo（lumasiran）用于治疗1型原发性高草酸尿症（PH1）。Lumasiran通过降解HAO1 mRNA和消耗乙醇酸氧化酶来发挥作用，乙醇酸氧化酶则进一步抑制肝脏过度生产的草酸盐，草酸盐可促进PH1疾病进展。

图片来源：seekingalpha.com

首席执行官John Maraganore表示：“ Alnylam的Lumasiran从发现到获得监管机构批准仅仅花了六年时间。” 上个月，欧洲药品管理局人用药品委员会基于两项III期试验的数据发表了正面意见，此后获得了监管部门的批准。

在ILLUMINATE-A中，包括成人和六岁及以上的儿童，Oxlumo达到了主要终点，尿草酸盐相对于安慰剂平均减少了53.5％，并且草酸盐相对于基线平均减少了65.4％。与安慰剂组相比，大多数接受Oxlumo治疗患者的草酸尿水平也达到了正常或接近正常水平。同时，在六岁以下婴幼儿中评估的ILLUMINATE-B研究也显示Oxlumo的疗效结果和安全性与ILLUMINATE-A中观察到的相当。

图片来源：seekingalpha.com

该疗法是由医护人员每月一次皮下注射，持续三个月，然后每季度一次。Oxlumo也在美国被授予优先审查，FDA预计将于12月3日做出决定。

原始出处：

https://www.firstwordpharma.com/node/1775889?tsid=28&region_id=2

评论区 (6)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1846941, encodeId=15ab18469417a, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Mon Jun 07 08:41:49 CST 2021, time=2021-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751687, encodeId=8b631e5168768, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, 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=Sun Jan 24 00:41:49 CST 2021, time=2021-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=901503, encodeId=8f75901503d7, content=替代移植吗, beContent=null, objectType=article, channel=null, level=null, likeNumber=129, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://q.qlogo.cn/qqapp/1103841572/5A52F5B6BEDC82694F39CB1D2A5D1CF1/100, createdBy=2a7c2136456, createdName=chenwb091, createdTime=Mon Nov 23 17:22:21 CST 2020, time=2020-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536872, encodeId=44a715368e287, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sun Nov 22 05:41:49 CST 2020, time=2020-11-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1590036, encodeId=744e159003607, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Sun Nov 22 05:41:49 CST 2020, time=2020-11-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=900810, encodeId=dec3900810f5, content=<a href='/topic/show?id=370215539f6' target=_blank style='color:#2F92EE;'>#RNAi疗法#</a>lumasiran获得<a href='/topic/show?id=135a63113db' target=_blank style='color:#2F92EE;'>#欧盟#</a>批准治疗1型<a href='/topic/show?id=92f13e13717' target=_blank style='color:#2F92EE;'>#原发性高草酸尿症#</a>，RNAi疗法的新药很快就会大量问世了, beContent=null, objectType=article, channel=null, level=null, likeNumber=228, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15539, encryptionId=370215539f6, topicName=RNAi疗法), TopicDto(id=63113, encryptionId=135a63113db, topicName=欧盟), TopicDto(id=37137, encryptionId=92f13e13717, topicName=原发性高草酸尿症)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Fri Nov 20 15:41:26 CST 2020, time=2020-11-20, status=1, ipAttribution=)]
    2021-06-07 jiyangfei

    #原发性#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1846941, encodeId=15ab18469417a, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Mon Jun 07 08:41:49 CST 2021, time=2021-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751687, encodeId=8b631e5168768, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, 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=Sun Jan 24 00:41:49 CST 2021, time=2021-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=901503, encodeId=8f75901503d7, content=替代移植吗, beContent=null, objectType=article, channel=null, level=null, likeNumber=129, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://q.qlogo.cn/qqapp/1103841572/5A52F5B6BEDC82694F39CB1D2A5D1CF1/100, createdBy=2a7c2136456, createdName=chenwb091, createdTime=Mon Nov 23 17:22:21 CST 2020, time=2020-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536872, encodeId=44a715368e287, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sun Nov 22 05:41:49 CST 2020, time=2020-11-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1590036, encodeId=744e159003607, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Sun Nov 22 05:41:49 CST 2020, time=2020-11-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=900810, encodeId=dec3900810f5, content=<a href='/topic/show?id=370215539f6' target=_blank style='color:#2F92EE;'>#RNAi疗法#</a>lumasiran获得<a href='/topic/show?id=135a63113db' target=_blank style='color:#2F92EE;'>#欧盟#</a>批准治疗1型<a href='/topic/show?id=92f13e13717' target=_blank style='color:#2F92EE;'>#原发性高草酸尿症#</a>，RNAi疗法的新药很快就会大量问世了, beContent=null, objectType=article, channel=null, level=null, likeNumber=228, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15539, encryptionId=370215539f6, topicName=RNAi疗法), TopicDto(id=63113, encryptionId=135a63113db, topicName=欧盟), TopicDto(id=37137, encryptionId=92f13e13717, topicName=原发性高草酸尿症)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Fri Nov 20 15:41:26 CST 2020, time=2020-11-20, status=1, ipAttribution=)]
    2021-01-24 yinxm8315

    #RNAi#

    0

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    2020-11-23 chenwb091

    替代移植吗

    0

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    2020-11-22 fzwish20004

    #RNAi疗法Lumasiran#

    0

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    2020-11-22 chenwq14

    #欧盟批准#

    0

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    2020-11-20 lovetcm

    #RNAi疗法#lumasiran获得#欧盟#批准治疗1型#原发性高草酸尿症#，RNAi疗法的新药很快就会大量问世了

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