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罕见病新药！辉瑞「氯苯唑酸软胶囊」在中国获批

2020-10-10 风华 医药魔方

辉瑞氯苯唑酸软胶囊转甲状腺素蛋白
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10月9日，辉瑞在中国提交的氯苯唑酸软胶囊上市申请获国家药监局批准上市，用于治疗成人野生型或遗传型转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM），以减少心血管死亡及心血管相关的住院治疗。

10月9日，辉瑞在中国提交的氯苯唑酸软胶囊上市申请获国家药监局批准上市，用于治疗成人野生型或遗传型转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM），以减少心血管死亡及心血管相关的住院治疗。

ATTR-CM是一种罕见的致死性疾病，由一种转运蛋白，转甲状腺素蛋白不稳定引起的。转甲状腺素蛋白由4个相同的部分（四聚体）组成。当不稳定的转甲状腺素蛋白四聚物解离，就会导致错误折叠的蛋白形成淀粉样纤维沉积于心脏，从而使心肌变得僵硬，最终导致心力衰竭。

2019年5月，辉瑞Vyndaqel（氯苯唑酸葡甲胺，tafamidis meglumine）和Vyndamax（氯苯唑酸）获FDA批准上市，这是转甲状腺素蛋白稳定剂氯苯唑酸的2种口服制剂，能够选择性地与转甲状腺素蛋白结合，稳定转甲状腺素蛋白的四聚体。2020年2月5日，辉瑞氯苯唑酸葡胺软胶囊（维达全，Vyndaqel）已率先获得NMPA批准上市，用于治疗转甲状腺素蛋白淀粉样变性多发性神经病（ATTR-PN）I期症状性成人患者，以延缓其周围神经功能损害。

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    2021-05-13 wgx306

    #氯苯唑酸#

    0

  2. 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    2020-10-24 jacob9231

    谢谢啦学习

    0

  3. 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createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-23 jacob9231

    学习了谢谢

    0

  4. 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createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Thu Oct 22 16:11:34 CST 2020, time=2020-10-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=893592, encodeId=413489359295, content=学习了赞赞赞, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Wed Oct 21 21:37:40 CST 2020, time=2020-10-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=893414, encodeId=571789341405, content=谢谢学习了, beContent=null, objectType=article, channel=null, level=null, 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createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-22 jacob9231

    学习了谢谢

    0

  5. 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createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-21 jacob9231

    学习了赞赞赞

    0

  6. 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GetPortalCommentsPageByObjectIdResponse(id=892560, encodeId=310789256012, content=好好好好好, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Fri Oct 16 22:28:05 CST 2020, time=2020-10-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892342, encodeId=566189234200, content=受教了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-20 jacob9231

    谢谢学习了

    0

  7. 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createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Thu Oct 22 16:11:34 CST 2020, time=2020-10-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=893592, encodeId=413489359295, content=学习了赞赞赞, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Wed Oct 21 21:37:40 CST 2020, time=2020-10-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=893414, encodeId=571789341405, content=谢谢学习了, beContent=null, objectType=article, channel=null, level=null, 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GetPortalCommentsPageByObjectIdResponse(id=892560, encodeId=310789256012, content=好好好好好, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Fri Oct 16 22:28:05 CST 2020, time=2020-10-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892342, encodeId=566189234200, content=受教了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-18 jacob9231

    学习了谢谢

    0

  8. 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createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-16 jacob9231

    好好好好好

    0

  9. 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createdName=jacob9231, createdTime=Thu Oct 15 22:48:08 CST 2020, time=2020-10-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=891868, encodeId=c4d2891868bc, content=学习了谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/Q3auHgzwzM66WRRAmeqIVSnDN88DpaJfEWzRZxg4yBbiaBmMRwcvViaA2C0sE0Ts3Lic24JSSI4YffujDtpnm0GTHpuiao9TytmNArrcYESnSXY/0, createdBy=7938104912, createdName=jacob9231, createdTime=Tue Oct 13 21:12:26 CST 2020, time=2020-10-13, status=1, ipAttribution=)]
    2020-10-15 jacob9231

    受教了谢谢

    0

  10. 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    2020-10-13 jacob9231

    学习了谢谢

    0

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