FDA 授予 NT102治疗Dravet 综合征的孤儿药称号

2021-10-31 Allan MedSci原创

Dravet 综合征是一种罕见的儿童期综合征,每 15,700 名在美国出生的婴儿中就有 1 人受到影响,其特征是脑病和癫痫。很难诊断,因为许多症状在孩子出生后的第一年并不明显。

Dravet 综合征是一种罕见的儿童期综合征,每 15,700 名在美国出生的婴儿中就有 1 人受到影响,其特征是脑病和癫痫。很难诊断,因为许多症状在孩子出生后的第一年并不明显。癫痫发作是最普遍的症状,可在核心温度升高(例如发烧、洗热水澡)后出现,此后继续频繁发生,严重程度通常会增加。全面诊断需要神经科医生下令进行基因检测,但根据 Dravet 综合征基金会的说法,最终确诊的患者至少包括以下症状:

  • 癫痫发作前具有正常或接近正常的认知和运动发育。
  • 在一岁之前,有或没有发烧的两次或多次癫痫发作。
  • 病史包括肌阵挛、半阵挛或全身强直-阵挛发作活动。
  • 两次或多次癫痫发作持续时间超过 10 分钟。
  • 对一线抗癫痫药物治疗无反应,两岁后癫痫持续发作。

在 80-90% 的 Dravet 综合征病例中,该疾病是由 SCN1A 基因突变引起的,该基因编码电压门控钠通道 Nav1.1 的一个亚基。这种钠通道的活性不足会改变大脑中的兴奋/抑制平衡,导致过度兴奋。

Neuroene Therapeutics近日宣布,FDA 已授予该公司专有化合物 NT102 的孤儿药称号。NT102 是作为线粒体和神经保护物质开发的,用于治疗神经系统疾病,如耐药性癫痫。在由美国国立卫生研究院资助的临床前研究中,NT102被证明具有良好的耐受性和口服生物利用度,具有出色的大脑渗透性。NT102 在多种临床前动物模型中具有广泛的癫痫发作保护,尤其是对 Dravet 综合征(一种以严重脑病和癫痫为特征的罕见儿童综合征)的癫痫发作具有出色的保护作用。

Neuroene 在犹他大学抗惊厥药物开发计划 (ADD) 发现,虽然只有 FDA 批准的抗癫痫药物组合才能有效减少疾病模型中的癫痫发作,但 NT102 显示出强大的单药疗效。

原始出处:

https://www.firstwordpharma.com/node/1875598?tsid=4

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    2021-11-01 ms4000000672105675

    非常好,非常感谢

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1365263, encodeId=e0ef13652632d, content=<a href='/topic/show?id=dfa045434e1' target=_blank style='color:#2F92EE;'>#孤儿药称号#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45434, encryptionId=dfa045434e1, topicName=孤儿药称号)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=cd0d217, createdName=huiwelcome, createdTime=Tue Nov 02 13:34:16 CST 2021, time=2021-11-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1432179, encodeId=cdfc14321e934, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Tue Nov 02 13:34:16 CST 2021, time=2021-11-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1065767, encodeId=bdf21065e675c, content=非常好,非常感谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=6a085645468, createdName=ms4000000672105675, createdTime=Mon Nov 01 13:02:15 CST 2021, time=2021-11-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1065676, encodeId=fee510656e6fa, content=谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=644b2182796, createdName=1207866fm50(暂无昵称), createdTime=Mon Nov 01 07:18:41 CST 2021, time=2021-11-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1065628, encodeId=349e1065628b2, content=已读已读已读已读已读已读已读已读已读已读已读已读已读已读已读, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f03a5433387, createdName=ms5000000518166734, createdTime=Sun Oct 31 22:49:11 CST 2021, time=2021-10-31, status=1, ipAttribution=)]
    2021-11-01 1207866fm50(暂无昵称)

    谢谢分享

    0

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    2021-10-31 ms5000000518166734

    已读已读已读已读已读已读已读已读已读已读已读已读已读已读已读

    0

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