内分泌科

订阅

MedSci主页 / 所有栏目 / 内分泌科

Arimoclomol治疗丙型尼曼-皮克病：已完成新药申请

2020-07-22 Allan MedSci原创

Arimoclomol丙型尼曼-皮克病
关注

生物制药公司Orphazyme致力于开发热休克蛋白应答技术，Orphazyme近日宣布已向美国FDA提交了Arimoclomol的新药申请（NDA），用于治疗丙型尼曼-皮克病（NPC）。

生物制药公司Orphazyme致力于开发热休克蛋白应答技术，Orphazyme近日宣布已向美国FDA提交了Arimoclomol的新药申请（NDA），用于治疗丙型尼曼-皮克病（NPC）。NPC又称鞘磷脂沉积病，为先天性糖脂代谢性疾病，NPC的特点是全单核巨噬细胞和神经系统有大量的含有神经鞘磷脂的泡沫细胞。

Arimoclomol是一种小分子热休克应激反应诱导剂，可以提高热休克蛋白的产生。热休克反应是一种负责处理细胞应激的保护系统，并参与维护蛋白质的适当折叠。HSP可以挽救错误折叠的蛋白、清除蛋白聚集体、改善溶酶体功能。Arimoclomol已获得治疗NPC的快速通道资格、突破性疗法称号以及孤儿药称号。完成滚动提交后，FDA最多有60天的时间来确定是否接受申请。

明尼苏达州罗切斯特市梅奥诊所儿童中心神经内科Marc Patterson教授称：“Arimoclomol治疗丙型尼曼匹克病的随机对照临床试验的数据支持了该药物在减缓疾病进展中的积极作用，特别是在四岁以上的患者亚组中”。

 

原始出处：

https://www.firstwordpharma.com/node/1741707?tsid=4

版权声明：
本网站所有内容来源注明为“梅斯医学”或“MedSci原创”的文字、图片和音视频资料，版权均属于梅斯医学所有。非经授权，任何媒体、网站或个人不得转载，授权转载时须注明来源为“梅斯医学”。其它来源的文章系转载文章，或“梅斯号”自媒体发布的文章，仅系出于传递更多信息之目的，本站仅负责审核内容合规，其内容不代表本站立场，本站不负责内容的准确性和版权。如果存在侵权、或不希望被转载的媒体或个人可与我们联系，我们将立即进行删除处理。
在此留言
评论区 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1744215, encodeId=a59a1e44215ba, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Nov 08 12:19:35 CST 2020, time=2020-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=895309, encodeId=298989530920, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>新药！, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Thu Oct 29 10:00:11 CST 2020, time=2020-10-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1649489, encodeId=847d16494898f, content=<a href='/topic/show?id=27a52e6127' target=_blank style='color:#2F92EE;'>#Arimoclomol#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2761, encryptionId=27a52e6127, topicName=Arimoclomol)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b3cc23842214, createdName=waiwai3038, createdTime=Sun May 02 21:19:35 CST 2021, time=2021-05-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=808619, encodeId=8ac9808619d3, content=<a href='/topic/show?id=5a8be955621' target=_blank style='color:#2F92EE;'>#罕见病#</a><a href='/topic/show?id=75cfe9561c4' target=_blank style='color:#2F92EE;'>#罕见病新药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=135, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79556, encryptionId=5a8be955621, topicName=罕见病), TopicDto(id=79561, encryptionId=75cfe9561c4, topicName=罕见病新药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=docwu2019, createdTime=Thu Aug 13 22:49:15 CST 2020, time=2020-08-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803978, encodeId=db518039e8d9, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>可能只有一点点缓解作用吧, beContent=null, objectType=article, channel=null, level=null, likeNumber=108, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Wed Jul 22 19:50:06 CST 2020, time=2020-07-22, status=1, ipAttribution=)]
    2020-11-08 wangbingxhy

    #新药申请#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1744215, encodeId=a59a1e44215ba, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Nov 08 12:19:35 CST 2020, time=2020-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=895309, encodeId=298989530920, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>新药！, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Thu Oct 29 10:00:11 CST 2020, time=2020-10-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1649489, encodeId=847d16494898f, content=<a href='/topic/show?id=27a52e6127' target=_blank style='color:#2F92EE;'>#Arimoclomol#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2761, encryptionId=27a52e6127, topicName=Arimoclomol)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b3cc23842214, createdName=waiwai3038, createdTime=Sun May 02 21:19:35 CST 2021, time=2021-05-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=808619, encodeId=8ac9808619d3, content=<a href='/topic/show?id=5a8be955621' target=_blank style='color:#2F92EE;'>#罕见病#</a><a href='/topic/show?id=75cfe9561c4' target=_blank style='color:#2F92EE;'>#罕见病新药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=135, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79556, encryptionId=5a8be955621, topicName=罕见病), TopicDto(id=79561, encryptionId=75cfe9561c4, topicName=罕见病新药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=docwu2019, createdTime=Thu Aug 13 22:49:15 CST 2020, time=2020-08-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803978, encodeId=db518039e8d9, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>可能只有一点点缓解作用吧, beContent=null, objectType=article, channel=null, level=null, likeNumber=108, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Wed Jul 22 19:50:06 CST 2020, time=2020-07-22, status=1, ipAttribution=)]
    2020-10-29 lovetcm

    #尼曼-皮克病#新药！

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1744215, encodeId=a59a1e44215ba, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Nov 08 12:19:35 CST 2020, time=2020-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=895309, encodeId=298989530920, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>新药！, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Thu Oct 29 10:00:11 CST 2020, time=2020-10-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1649489, encodeId=847d16494898f, content=<a href='/topic/show?id=27a52e6127' target=_blank style='color:#2F92EE;'>#Arimoclomol#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2761, encryptionId=27a52e6127, topicName=Arimoclomol)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b3cc23842214, createdName=waiwai3038, createdTime=Sun May 02 21:19:35 CST 2021, time=2021-05-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=808619, encodeId=8ac9808619d3, content=<a href='/topic/show?id=5a8be955621' target=_blank style='color:#2F92EE;'>#罕见病#</a><a href='/topic/show?id=75cfe9561c4' target=_blank style='color:#2F92EE;'>#罕见病新药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=135, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79556, encryptionId=5a8be955621, topicName=罕见病), TopicDto(id=79561, encryptionId=75cfe9561c4, topicName=罕见病新药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=docwu2019, createdTime=Thu Aug 13 22:49:15 CST 2020, time=2020-08-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803978, encodeId=db518039e8d9, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>可能只有一点点缓解作用吧, beContent=null, objectType=article, channel=null, level=null, likeNumber=108, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Wed Jul 22 19:50:06 CST 2020, time=2020-07-22, status=1, ipAttribution=)]
    2021-05-02 waiwai3038

    #Arimoclomol#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1744215, encodeId=a59a1e44215ba, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Nov 08 12:19:35 CST 2020, time=2020-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=895309, encodeId=298989530920, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>新药！, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Thu Oct 29 10:00:11 CST 2020, time=2020-10-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1649489, encodeId=847d16494898f, content=<a href='/topic/show?id=27a52e6127' target=_blank style='color:#2F92EE;'>#Arimoclomol#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2761, encryptionId=27a52e6127, topicName=Arimoclomol)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b3cc23842214, createdName=waiwai3038, createdTime=Sun May 02 21:19:35 CST 2021, time=2021-05-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=808619, encodeId=8ac9808619d3, content=<a href='/topic/show?id=5a8be955621' target=_blank style='color:#2F92EE;'>#罕见病#</a><a href='/topic/show?id=75cfe9561c4' target=_blank style='color:#2F92EE;'>#罕见病新药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=135, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79556, encryptionId=5a8be955621, topicName=罕见病), TopicDto(id=79561, encryptionId=75cfe9561c4, topicName=罕见病新药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=docwu2019, createdTime=Thu Aug 13 22:49:15 CST 2020, time=2020-08-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803978, encodeId=db518039e8d9, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>可能只有一点点缓解作用吧, beContent=null, objectType=article, channel=null, level=null, likeNumber=108, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Wed Jul 22 19:50:06 CST 2020, time=2020-07-22, status=1, ipAttribution=)]
    2020-08-13 docwu2019

    #罕见病##罕见病新药#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1744215, encodeId=a59a1e44215ba, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Nov 08 12:19:35 CST 2020, time=2020-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=895309, encodeId=298989530920, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>新药！, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Thu Oct 29 10:00:11 CST 2020, time=2020-10-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1649489, encodeId=847d16494898f, content=<a href='/topic/show?id=27a52e6127' target=_blank style='color:#2F92EE;'>#Arimoclomol#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2761, encryptionId=27a52e6127, topicName=Arimoclomol)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b3cc23842214, createdName=waiwai3038, createdTime=Sun May 02 21:19:35 CST 2021, time=2021-05-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=808619, encodeId=8ac9808619d3, content=<a href='/topic/show?id=5a8be955621' target=_blank style='color:#2F92EE;'>#罕见病#</a><a href='/topic/show?id=75cfe9561c4' target=_blank style='color:#2F92EE;'>#罕见病新药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=135, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79556, encryptionId=5a8be955621, topicName=罕见病), TopicDto(id=79561, encryptionId=75cfe9561c4, topicName=罕见病新药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=docwu2019, createdTime=Thu Aug 13 22:49:15 CST 2020, time=2020-08-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803978, encodeId=db518039e8d9, content=<a href='/topic/show?id=ae974e367e0' target=_blank style='color:#2F92EE;'>#尼曼-皮克病#</a>可能只有一点点缓解作用吧, beContent=null, objectType=article, channel=null, level=null, likeNumber=108, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=47367, encryptionId=ae974e367e0, topicName=尼曼-皮克病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Wed Jul 22 19:50:06 CST 2020, time=2020-07-22, status=1, ipAttribution=)]
    2020-07-22 lovetcm

    #尼曼-皮克病#可能只有一点点缓解作用吧

    0

相关资讯

Orphazyme的热休克蛋白疗法Arimoclomol治疗肌萎缩性侧索硬化症，获得美国快速通道指定

Arimoclomol通过增加热休克蛋白的产生，帮助挽救错误折叠的蛋白，清除蛋白聚集体来治疗肌萎缩性侧索硬化症。

拓展阅读

罕见病例｜B型尼曼-皮克病及其肝脏受累的异质性表现1例

现将收治的1例NPD-B病例报道如下，并着重对NPD-B肝脏病变的异质性表现进行文献复习，旨在提高疑难罕见肝脏疾病的临床诊治水平。

Autophagy：环糊精通过自噬治疗C型尼曼-皮克病

C型尼曼-皮克病是一种NPC1或NPC2突变引起的溶酶体贮积病。NPC1或NPC2蛋白的缺失会导致溶酶体内未酯化的胆固醇聚积。药物2-羟丙基-β-环糊精（HPβCD）能够减少C型尼曼-皮克病患者中溶酶体胆固醇的积累，并已进入人体临床试验。