欧洲潜在的治疗软骨发育不全的药物vosoritide:正式提交营销申请

2020-07-24 MedSci原创 MedSci原创

vosoritide是一种每日一次的C型利尿钠肽(CNP)注射剂,用于治疗软骨发育不良的儿童。

BioMarin已向欧洲药品管理局(EMA)提交了vosoritide的市场营销申请,vosoritide是一种每日一次的C型利尿钠肽(CNP)注射剂,用于治疗软骨发育不良的儿童

此次申请基于该药物功效和安全性的III期研究结果,并得到正在进行的II期和III扩展研究的长期安全性和有效性数据的进一步支持。

如果获得批准,vosoritide将成为欧洲首个治疗软骨发育不全的药物。

BioMarin全球研究与开发总裁Hank Fuchs表示:“我们的目标是提供一种治疗方法,以解决该病的根本问题,并随着时间的推移减少软骨发育不良所引起的并发症。”

关于软骨发育不良

软骨发育不良是不成比例矮小身材的最常见形式。软骨病的特征是软骨内骨化减慢,引起长骨骼、脊柱、面部和颅骨的骨骼矮小,结构紊乱。除了身材短小以外,软骨发育不全的人还会遭遇严重的健康并发症,包括睡眠呼吸暂停、双腿弯曲、面部中部发育不全、下背部永久性摇摆、椎管狭窄和复发性耳部感染

软骨炎是由成骨细胞生长因子受体3基因(FGFR3)突变引起,FGFR3是骨骼生长的负调节剂。

See the source image

图片来源:https://checkrare.com/first-participant-dosed-in-phase-2-study-of-vosoritide-for-children-with-achondroplasia/

关于Vosoritide

Vosoritide源自天然的人源肽,是骨骼生长的正向调节剂,通过与特定的受体结合,激活抑制FGFR3的细胞内信号。

原始出处:

http://www.pharmatimes.com/news/biomarin_submits_eu_marketing_application_for_vosoritide_1345428

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    2020-08-31 ms3000000352910740

    什么时候可以出售

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=881285, encodeId=c583881285c9, content=什么时候可以出售, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c2be5414636, createdName=ms3000000352910740, createdTime=Mon Aug 31 10:05:46 CST 2020, time=2020-08-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339340, encodeId=915e133934093, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1347491, encodeId=6855134e49169, content=<a href='/topic/show?id=88713e938c1' target=_blank style='color:#2F92EE;'>#发育#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37938, encryptionId=88713e938c1, topicName=发育)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=f4c124, createdName=neurowu, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804369, encodeId=3eb48043698f, content=多多学习啊, beContent=null, objectType=article, channel=null, level=null, likeNumber=96, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7d491808785, createdName=ms6779183192904278, createdTime=Fri Jul 24 20:28:50 CST 2020, time=2020-07-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804366, encodeId=ebdc8043664f, content=🐂🍺, beContent=null, objectType=article, channel=null, level=null, likeNumber=90, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200724/6c3c7d3af64b4dfa911c93d4b277aace/fafb7aec90dd4c9ab95b89c5dd89e514.jpg, createdBy=5bfa5390798, createdName=ms6000000681387880, createdTime=Fri Jul 24 20:06:51 CST 2020, time=2020-07-24, status=1, ipAttribution=)]
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    2020-07-26 neurowu
  4. [GetPortalCommentsPageByObjectIdResponse(id=881285, encodeId=c583881285c9, content=什么时候可以出售, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c2be5414636, createdName=ms3000000352910740, createdTime=Mon Aug 31 10:05:46 CST 2020, time=2020-08-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339340, encodeId=915e133934093, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1347491, encodeId=6855134e49169, content=<a href='/topic/show?id=88713e938c1' target=_blank style='color:#2F92EE;'>#发育#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37938, encryptionId=88713e938c1, topicName=发育)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=f4c124, createdName=neurowu, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804369, encodeId=3eb48043698f, content=多多学习啊, beContent=null, objectType=article, channel=null, level=null, likeNumber=96, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7d491808785, createdName=ms6779183192904278, createdTime=Fri Jul 24 20:28:50 CST 2020, time=2020-07-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804366, encodeId=ebdc8043664f, content=🐂🍺, beContent=null, objectType=article, channel=null, level=null, likeNumber=90, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200724/6c3c7d3af64b4dfa911c93d4b277aace/fafb7aec90dd4c9ab95b89c5dd89e514.jpg, createdBy=5bfa5390798, createdName=ms6000000681387880, createdTime=Fri Jul 24 20:06:51 CST 2020, time=2020-07-24, status=1, ipAttribution=)]
    2020-07-24 ms6779183192904278

    多多学习啊

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=881285, encodeId=c583881285c9, content=什么时候可以出售, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c2be5414636, createdName=ms3000000352910740, createdTime=Mon Aug 31 10:05:46 CST 2020, time=2020-08-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339340, encodeId=915e133934093, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1347491, encodeId=6855134e49169, content=<a href='/topic/show?id=88713e938c1' target=_blank style='color:#2F92EE;'>#发育#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37938, encryptionId=88713e938c1, topicName=发育)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=f4c124, createdName=neurowu, createdTime=Sun Jul 26 10:39:33 CST 2020, time=2020-07-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804369, encodeId=3eb48043698f, content=多多学习啊, beContent=null, objectType=article, channel=null, level=null, likeNumber=96, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7d491808785, createdName=ms6779183192904278, createdTime=Fri Jul 24 20:28:50 CST 2020, time=2020-07-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=804366, encodeId=ebdc8043664f, content=🐂🍺, beContent=null, objectType=article, channel=null, level=null, likeNumber=90, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200724/6c3c7d3af64b4dfa911c93d4b277aace/fafb7aec90dd4c9ab95b89c5dd89e514.jpg, createdBy=5bfa5390798, createdName=ms6000000681387880, createdTime=Fri Jul 24 20:06:51 CST 2020, time=2020-07-24, status=1, ipAttribution=)]
    2020-07-24 ms6000000681387880

    🐂🍺

    0

相关资讯

BioMarin计划今年向美国和欧洲提交C型钠尿肽vosoritide的营销申请,用于治疗软骨发育不全症的儿童

如果获得批准,vosoritide将成为美国和欧洲首个治疗软骨发育不全症的药物。