肾内科

订阅

MedSci主页 / 所有栏目 / 肾内科

APL-2获得FDA的孤儿药物认定用于治疗C3肾小球病

2018-12-22 MedSci MedSci原创

C3肾小球病APL-2孤儿药物认定
关注

Apellis是一家临床阶段的生物制药公司，专注于通过抑制补体系统开发治疗疾病的新型疗法，近日宣布其C3补体抑制剂APL-2已被FDA授予孤儿药物认定用于治疗C3肾小球病。

Apellis是一家临床阶段的生物制药公司，专注于通过抑制补体系统开发治疗疾病的新型疗法，近日宣布其C3补体抑制剂APL-2已被FDA授予孤儿药物认定用于治疗C3肾小球病。C3肾小球病（C3G）是一类罕见的肾脏疾病，其补体系统的活动过度导致肾小球中C3的沉积。大约一半的C3G患者在诊断后的10年内进展为终末期肾病，并且没有经证实可以预防进展的疗法。

通过靶向C3，即所有三种补体途径（经典、凝集素和替代途径），Apellis认为APL-2有可能消除C3G的过度和不适当的C3激活，从而避免肾小球C3的沉积和进展肾病。Apellis目前正在对四种类型的肾小球疾病进行II期试验，其中涉及C3肾小球病、IgA肾病、原发性膜性肾病和狼疮性肾炎。FDA孤儿药物计划旨在加快用于治疗罕见疾病（在美国影响少于200,000人的疾病）的新药的审批。该指定为制药商提供开发和商业激励措施，包括七年的美国市场独占权、FDA对临床研究设计的咨询、加速药物开发的潜力以及某些费用减免。


原始出处：

http://www.firstwordpharma.com/node/1613058#axzz5aJrWexZu

本文系梅斯医学（MedSci）原创编译整理，转载需授权！

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=1951902, encodeId=3e8d1951902b7, content=<a href='/topic/show?id=1fcb39098a' target=_blank style='color:#2F92EE;'>#C3肾小球病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3909, encryptionId=1fcb39098a, topicName=C3肾小球病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c950224, createdName=hjwang79, createdTime=Mon Apr 08 12:48:00 CST 2019, time=2019-04-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1740089, encodeId=bef31e4008981, content=<a href='/topic/show?id=f5a445431df' target=_blank style='color:#2F92EE;'>#孤儿药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45431, encryptionId=f5a445431df, topicName=孤儿药物)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a4f034650217, createdName=ms6980045490879608, createdTime=Fri Nov 08 07:48:00 CST 2019, time=2019-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509521, encodeId=72eb15095215b, content=<a href='/topic/show?id=6a8e264431' target=_blank style='color:#2F92EE;'>#APL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2644, encryptionId=6a8e264431, topicName=APL)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c81c10095307, createdName=风铃824, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1615959, encodeId=2f46161595981, content=<a href='/topic/show?id=519345433ea' target=_blank style='color:#2F92EE;'>#孤儿药物认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45433, encryptionId=519345433ea, topicName=孤儿药物认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8cfd19711155, createdName=若水129, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=356168, encodeId=2a9335616871, content=很有价值的靶点, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Sun Dec 23 07:56:59 CST 2018, time=2018-12-23, status=1, ipAttribution=)]
    2019-04-08 hjwang79

    #C3肾小球病#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1951902, encodeId=3e8d1951902b7, content=<a href='/topic/show?id=1fcb39098a' target=_blank style='color:#2F92EE;'>#C3肾小球病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3909, encryptionId=1fcb39098a, topicName=C3肾小球病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c950224, createdName=hjwang79, createdTime=Mon Apr 08 12:48:00 CST 2019, time=2019-04-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1740089, encodeId=bef31e4008981, content=<a href='/topic/show?id=f5a445431df' target=_blank style='color:#2F92EE;'>#孤儿药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45431, encryptionId=f5a445431df, topicName=孤儿药物)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a4f034650217, createdName=ms6980045490879608, createdTime=Fri Nov 08 07:48:00 CST 2019, time=2019-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509521, encodeId=72eb15095215b, content=<a href='/topic/show?id=6a8e264431' target=_blank style='color:#2F92EE;'>#APL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2644, encryptionId=6a8e264431, topicName=APL)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c81c10095307, createdName=风铃824, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1615959, encodeId=2f46161595981, content=<a href='/topic/show?id=519345433ea' target=_blank style='color:#2F92EE;'>#孤儿药物认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45433, encryptionId=519345433ea, topicName=孤儿药物认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8cfd19711155, createdName=若水129, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=356168, encodeId=2a9335616871, content=很有价值的靶点, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Sun Dec 23 07:56:59 CST 2018, time=2018-12-23, status=1, ipAttribution=)]
    2019-11-08 ms6980045490879608

    #孤儿药物#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1951902, encodeId=3e8d1951902b7, content=<a href='/topic/show?id=1fcb39098a' target=_blank style='color:#2F92EE;'>#C3肾小球病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3909, encryptionId=1fcb39098a, topicName=C3肾小球病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c950224, createdName=hjwang79, createdTime=Mon Apr 08 12:48:00 CST 2019, time=2019-04-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1740089, encodeId=bef31e4008981, content=<a href='/topic/show?id=f5a445431df' target=_blank style='color:#2F92EE;'>#孤儿药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45431, encryptionId=f5a445431df, topicName=孤儿药物)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a4f034650217, createdName=ms6980045490879608, createdTime=Fri Nov 08 07:48:00 CST 2019, time=2019-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509521, encodeId=72eb15095215b, content=<a href='/topic/show?id=6a8e264431' target=_blank style='color:#2F92EE;'>#APL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2644, encryptionId=6a8e264431, topicName=APL)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c81c10095307, createdName=风铃824, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1615959, encodeId=2f46161595981, content=<a href='/topic/show?id=519345433ea' target=_blank style='color:#2F92EE;'>#孤儿药物认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45433, encryptionId=519345433ea, topicName=孤儿药物认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8cfd19711155, createdName=若水129, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=356168, encodeId=2a9335616871, content=很有价值的靶点, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Sun Dec 23 07:56:59 CST 2018, time=2018-12-23, status=1, ipAttribution=)]
    2018-12-24 风铃824

    #APL#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1951902, encodeId=3e8d1951902b7, content=<a href='/topic/show?id=1fcb39098a' target=_blank style='color:#2F92EE;'>#C3肾小球病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3909, encryptionId=1fcb39098a, topicName=C3肾小球病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c950224, createdName=hjwang79, createdTime=Mon Apr 08 12:48:00 CST 2019, time=2019-04-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1740089, encodeId=bef31e4008981, content=<a href='/topic/show?id=f5a445431df' target=_blank style='color:#2F92EE;'>#孤儿药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45431, encryptionId=f5a445431df, topicName=孤儿药物)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a4f034650217, createdName=ms6980045490879608, createdTime=Fri Nov 08 07:48:00 CST 2019, time=2019-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509521, encodeId=72eb15095215b, content=<a href='/topic/show?id=6a8e264431' target=_blank style='color:#2F92EE;'>#APL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2644, encryptionId=6a8e264431, topicName=APL)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c81c10095307, createdName=风铃824, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1615959, encodeId=2f46161595981, content=<a href='/topic/show?id=519345433ea' target=_blank style='color:#2F92EE;'>#孤儿药物认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45433, encryptionId=519345433ea, topicName=孤儿药物认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8cfd19711155, createdName=若水129, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=356168, encodeId=2a9335616871, content=很有价值的靶点, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Sun Dec 23 07:56:59 CST 2018, time=2018-12-23, status=1, ipAttribution=)]
    2018-12-24 若水129

    #孤儿药物认定#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1951902, encodeId=3e8d1951902b7, content=<a href='/topic/show?id=1fcb39098a' target=_blank style='color:#2F92EE;'>#C3肾小球病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3909, encryptionId=1fcb39098a, topicName=C3肾小球病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c950224, createdName=hjwang79, createdTime=Mon Apr 08 12:48:00 CST 2019, time=2019-04-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1740089, encodeId=bef31e4008981, content=<a href='/topic/show?id=f5a445431df' target=_blank style='color:#2F92EE;'>#孤儿药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45431, encryptionId=f5a445431df, topicName=孤儿药物)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=a4f034650217, createdName=ms6980045490879608, createdTime=Fri Nov 08 07:48:00 CST 2019, time=2019-11-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509521, encodeId=72eb15095215b, content=<a href='/topic/show?id=6a8e264431' target=_blank style='color:#2F92EE;'>#APL#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2644, encryptionId=6a8e264431, topicName=APL)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c81c10095307, createdName=风铃824, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1615959, encodeId=2f46161595981, content=<a href='/topic/show?id=519345433ea' target=_blank style='color:#2F92EE;'>#孤儿药物认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45433, encryptionId=519345433ea, topicName=孤儿药物认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8cfd19711155, createdName=若水129, createdTime=Mon Dec 24 07:48:00 CST 2018, time=2018-12-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=356168, encodeId=2a9335616871, content=很有价值的靶点, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c4a5105539, createdName=lovetcm, createdTime=Sun Dec 23 07:56:59 CST 2018, time=2018-12-23, status=1, ipAttribution=)]
    2018-12-23 lovetcm

    很有价值的靶点

    0