BioMarin公司血友病基因疗法Ⅲ期临床5年的长期效果观察,数据积极

2022-01-16 网络 网络

BioMarin的疗法,称为valoctocogene roxaparvovec或'valrox',在第3阶段GENER8-1试验中,20名严重A型血友病成人患者中,8名凝血因子VIII的水平得到充分

BioMarin的疗法,称为valoctocogene roxaparvovec或'valrox',在第3阶段GENER8-1试验中,20名严重A型血友病成人患者中,8名凝血因子VIII的水平得到充分改善,满足了预先规定的监管审查标准。

2019年6月,BioMarin报告了其针对A型血友病基因疗法的三年数据,该数据用于支持其在欧洲和美国的申请,然而,其长期治疗价值也引起了质疑。详细见:BioMarin的血友病基因疗法III期临床达到终点,但其长期效应引起质疑

2022年1月9日,BioMarin Pharmaceutical公司宣布,valoctocogene roxaparvovec在一项正在进行的Ⅲ期临床试验中获得积极结果。

这是一款使用AAV5病毒载体,递送表达凝血因子VIII的转基因的在研基因疗法,用于治疗严重血友病A成人患者。试验结果表明,在112名受试者(中位随访时间为110周)中,与4.8的基线值相比,valoctocogene roxaparvovec使患者年出血率(ABR)降低85%。在整个评估期间,平均ABR为0.8。同时,该疗法使每年凝血因子VIII输注率较基线数据显著降低98%。在凝血因子VIII活性水平方面,第二年结束时,改良意向治疗(mITT)人群(n=132)的受试者中使用发色底物(CS)检测获得的平均内源性凝血因子VIII活性水平为23(中位数11.8)IU/dL,一步法(OS)检测获得的平均内源性凝血因子VIII活性水平为36.1(中位数21.6)IU/dL。n该疗法已经获得美国FDA授予的再生医学先进疗法认定(RMAT)和突破性疗法认定。基于试验数据,BioMarin计划在2022年第二季度重新向FDA递交生物制品许可申请(BLA)。

不过,也看到随着随访时间延长,FVIII的活性在持续降低。

图片

不过,这项基因治疗一旦问世,将具有高昂的价格,详细见:BioMarin计划为其A型血友病基因疗法Valrox,定价为200万至300万美元

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=1919373, encodeId=b7b219193e310, content=<a href='/topic/show?id=4f26341994' target=_blank style='color:#2F92EE;'>#Bio#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3419, encryptionId=4f26341994, topicName=Bio)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Sun May 29 19:03:11 CST 2022, time=2022-05-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1187701, encodeId=b233118e701f3, content=我记得之前看过一个用脂质体做载体的,想不太起来了, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3cd6406141, createdName=ms4000000819102095, createdTime=Mon Jan 24 14:19:50 CST 2022, time=2022-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2038237, encodeId=4b1c203823e54, content=<a href='/topic/show?id=3a3a9e011f2' target=_blank style='color:#2F92EE;'>#长期效果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97011, encryptionId=3a3a9e011f2, topicName=长期效果)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85df2500173, createdName=654699882_84926986, createdTime=Wed Aug 17 14:03:11 CST 2022, time=2022-08-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1495861, encodeId=26c314958615f, content=<a href='/topic/show?id=351f956559' target=_blank style='color:#2F92EE;'>#Ⅲ期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9565, encryptionId=351f956559, topicName=Ⅲ期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe6c9221933, createdName=dinorsor527, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569637, encodeId=ec7d156963e7b, content=<a href='/topic/show?id=31993446b3' target=_blank style='color:#2F92EE;'>#BioMarin#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3446, encryptionId=31993446b3, topicName=BioMarin)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=413a15536427, createdName=ysjykql, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=)]
    2022-05-29 sunylz
  2. [GetPortalCommentsPageByObjectIdResponse(id=1919373, encodeId=b7b219193e310, content=<a href='/topic/show?id=4f26341994' target=_blank style='color:#2F92EE;'>#Bio#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3419, encryptionId=4f26341994, topicName=Bio)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Sun May 29 19:03:11 CST 2022, time=2022-05-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1187701, encodeId=b233118e701f3, content=我记得之前看过一个用脂质体做载体的,想不太起来了, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3cd6406141, createdName=ms4000000819102095, createdTime=Mon Jan 24 14:19:50 CST 2022, time=2022-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2038237, encodeId=4b1c203823e54, content=<a href='/topic/show?id=3a3a9e011f2' target=_blank style='color:#2F92EE;'>#长期效果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97011, encryptionId=3a3a9e011f2, topicName=长期效果)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85df2500173, createdName=654699882_84926986, createdTime=Wed Aug 17 14:03:11 CST 2022, time=2022-08-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1495861, encodeId=26c314958615f, content=<a href='/topic/show?id=351f956559' target=_blank style='color:#2F92EE;'>#Ⅲ期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9565, encryptionId=351f956559, topicName=Ⅲ期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe6c9221933, createdName=dinorsor527, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569637, encodeId=ec7d156963e7b, content=<a href='/topic/show?id=31993446b3' target=_blank style='color:#2F92EE;'>#BioMarin#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3446, encryptionId=31993446b3, topicName=BioMarin)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=413a15536427, createdName=ysjykql, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=)]
    2022-01-24 ms4000000819102095

    我记得之前看过一个用脂质体做载体的,想不太起来了

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1919373, encodeId=b7b219193e310, content=<a href='/topic/show?id=4f26341994' target=_blank style='color:#2F92EE;'>#Bio#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3419, encryptionId=4f26341994, topicName=Bio)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Sun May 29 19:03:11 CST 2022, time=2022-05-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1187701, encodeId=b233118e701f3, content=我记得之前看过一个用脂质体做载体的,想不太起来了, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3cd6406141, createdName=ms4000000819102095, createdTime=Mon Jan 24 14:19:50 CST 2022, time=2022-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2038237, encodeId=4b1c203823e54, content=<a href='/topic/show?id=3a3a9e011f2' target=_blank style='color:#2F92EE;'>#长期效果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97011, encryptionId=3a3a9e011f2, topicName=长期效果)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85df2500173, createdName=654699882_84926986, createdTime=Wed Aug 17 14:03:11 CST 2022, time=2022-08-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1495861, encodeId=26c314958615f, content=<a href='/topic/show?id=351f956559' target=_blank style='color:#2F92EE;'>#Ⅲ期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9565, encryptionId=351f956559, topicName=Ⅲ期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe6c9221933, createdName=dinorsor527, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569637, encodeId=ec7d156963e7b, content=<a href='/topic/show?id=31993446b3' target=_blank style='color:#2F92EE;'>#BioMarin#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3446, encryptionId=31993446b3, topicName=BioMarin)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=413a15536427, createdName=ysjykql, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=)]
  4. [GetPortalCommentsPageByObjectIdResponse(id=1919373, encodeId=b7b219193e310, content=<a href='/topic/show?id=4f26341994' target=_blank style='color:#2F92EE;'>#Bio#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3419, encryptionId=4f26341994, topicName=Bio)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Sun May 29 19:03:11 CST 2022, time=2022-05-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1187701, encodeId=b233118e701f3, content=我记得之前看过一个用脂质体做载体的,想不太起来了, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3cd6406141, createdName=ms4000000819102095, createdTime=Mon Jan 24 14:19:50 CST 2022, time=2022-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2038237, encodeId=4b1c203823e54, content=<a href='/topic/show?id=3a3a9e011f2' target=_blank style='color:#2F92EE;'>#长期效果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97011, encryptionId=3a3a9e011f2, topicName=长期效果)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85df2500173, createdName=654699882_84926986, createdTime=Wed Aug 17 14:03:11 CST 2022, time=2022-08-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1495861, encodeId=26c314958615f, content=<a href='/topic/show?id=351f956559' target=_blank style='color:#2F92EE;'>#Ⅲ期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9565, encryptionId=351f956559, topicName=Ⅲ期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe6c9221933, createdName=dinorsor527, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569637, encodeId=ec7d156963e7b, content=<a href='/topic/show?id=31993446b3' target=_blank style='color:#2F92EE;'>#BioMarin#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3446, encryptionId=31993446b3, topicName=BioMarin)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=413a15536427, createdName=ysjykql, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=)]
  5. [GetPortalCommentsPageByObjectIdResponse(id=1919373, encodeId=b7b219193e310, content=<a href='/topic/show?id=4f26341994' target=_blank style='color:#2F92EE;'>#Bio#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3419, encryptionId=4f26341994, topicName=Bio)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Sun May 29 19:03:11 CST 2022, time=2022-05-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1187701, encodeId=b233118e701f3, content=我记得之前看过一个用脂质体做载体的,想不太起来了, beContent=null, objectType=article, channel=null, level=null, likeNumber=67, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c3cd6406141, createdName=ms4000000819102095, createdTime=Mon Jan 24 14:19:50 CST 2022, time=2022-01-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2038237, encodeId=4b1c203823e54, content=<a href='/topic/show?id=3a3a9e011f2' target=_blank style='color:#2F92EE;'>#长期效果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97011, encryptionId=3a3a9e011f2, topicName=长期效果)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85df2500173, createdName=654699882_84926986, createdTime=Wed Aug 17 14:03:11 CST 2022, time=2022-08-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1495861, encodeId=26c314958615f, content=<a href='/topic/show?id=351f956559' target=_blank style='color:#2F92EE;'>#Ⅲ期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9565, encryptionId=351f956559, topicName=Ⅲ期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe6c9221933, createdName=dinorsor527, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569637, encodeId=ec7d156963e7b, content=<a href='/topic/show?id=31993446b3' target=_blank style='color:#2F92EE;'>#BioMarin#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3446, encryptionId=31993446b3, topicName=BioMarin)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=413a15536427, createdName=ysjykql, createdTime=Tue Jan 18 09:03:11 CST 2022, time=2022-01-18, status=1, ipAttribution=)]
    2022-01-18 ysjykql

相关资讯

 Haemophilia: 血友病患者中内皮糖蛋白水平升高与血管生成相关血管生成素 2 相关

血友病是由于凝血因子缺乏而发生的出血性疾病,血管生成过程是血友病性关节病病理生理的重要过程。尽管今天在血友病的治疗中仍取得了非常重要的进展,但寻找能够预测关节出血和血友病长期增加的生物标志物,这继续决

罗氏重磅新药舒友立乐®在华斩获新适应症,助力更多A型血友病患者迈向“零出血”自由人生

舒友立乐®(英文商品名:HEMLIBRA®,化学通用名:艾美赛珠单抗)已获得中国国家药品监督管理局(NMPA)正式批准。

HAEMOPHILIA:血友病患者的全因死亡率和死亡原因: 系统评价和META分析

A型血友病和B型血友病分别是由因子VIII(FVIII)和因子IX(FIX)缺乏引起的遗传性出血疾病

信念医药血友病基因疗法获临床试验批准,开启国内AAV基因疗法新篇章

8月6日,上海信致医药科技有限公司(信念医药全资子公司)自主研发的

NEJM:AAV基因治疗血友病,效果显著、安全性良好

血友病(Hemophilia),是一类由于血液中某些凝血因子的缺乏而导致患者产生严重凝血障碍的遗传性出血性疾病,可分为血友病A型、血友病B型和血友病C型。