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PFS 翻倍！tipifarnib获FDA突破性疗法资格

2021-02-26 医药魔方 医药魔方

铂类化疗后疾病
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2月24日，Kura宣布，tipifarnib已被FDA授予突破性疗法认定，用于治疗铂类化疗后疾病进展、等位基因变异频率≥20%，复发或转移性HRAS突变型头颈部鳞状细胞癌(HNSCC)。

2月24日，Kura宣布，tipifarnib已被FDA授予突破性疗法认定，用于治疗铂类化疗后疾病进展、等位基因变异频率≥20%，复发或转移性HRAS突变型头颈部鳞状细胞癌(HNSCC)。 头颈部鳞状细胞癌是世界上第七大最常见癌症，每年有超过88.5万的新发病例。尽管近年治疗取得了进展，但患者预后仍然很差，5年生存率低于40%。二线治疗对许多患者的临床获益有限，客观缓解率(ORR)仅为6-16%，中位无进展生存期(PFS)为2-3个月，中位总生存期(OS)为5-8个月。尽管HRAS早在40多年前就被发现了，但目前还没有针对这种蛋白突变形式的肿瘤的特定治疗方法。

HRAS是原癌基因，是一种正常基因，通过编码蛋白质，帮助调节细胞生长和分化。但可因突变或表达增加而成为癌基因。4-8%的头颈鳞状细胞癌患者存在HRAS突变。tipifarnib是一种有效的、高选择性的法尼酰基转移酶抑制剂，法尼酰基转移酶是HRAS活性的关键酶。

tipifarnib分子结构式

Tipifarnib突破性疗法认定是基于一项II期RUN-HN研究数据，该研究评估了tipifarnib在复发或转移性HRAS突变型头颈部鳞状细胞癌患者中的疗效。试验数据已于2020年5月在美国临床肿瘤学会上公布，18例疗效可评估患者的ORR为50%，中位PFS为5.9个月，中位OS为15.4个月。tipifarnib目前正在进行一项注册性临床研究（AIM-HN）。

Kura总裁兼首席执行官Troy Wilson博士说：“我们很高兴FDA授予tipifarnib突破性疗法称号，这是FDA对其治疗这种毁灭性疾病潜力的肯定。”我们将继续专注于AIM-HN注册性试验，并期待与FDA紧密合作，尽快将这种疗法应用于患者。

评论区 (3)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1448677, encodeId=d2ef14486e73c, content=<a href='/topic/show?id=aaf21412e7e' target=_blank style='color:#2F92EE;'>#PFS#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=110, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14127, encryptionId=aaf21412e7e, topicName=PFS)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=47f75418389, createdName=jeanqiuqiu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536216, encodeId=ac6f153621665, content=<a href='/topic/show?id=75fbe553800' target=_blank style='color:#2F92EE;'>#突破性疗法#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=102, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=75538, encryptionId=75fbe553800, topicName=突破性疗法)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7eca12667393, createdName=zhwj, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1624586, encodeId=5dab1624586b2, content=<a href='/topic/show?id=3f2d2e7087' target=_blank style='color:#2F92EE;'>#ARNI#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=106, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2770, encryptionId=3f2d2e7087, topicName=ARNI)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f41c20676233, createdName=gongliu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=)]
    2021-02-28 jeanqiuqiu

    #PFS#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1448677, encodeId=d2ef14486e73c, content=<a href='/topic/show?id=aaf21412e7e' target=_blank style='color:#2F92EE;'>#PFS#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=110, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14127, encryptionId=aaf21412e7e, topicName=PFS)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=47f75418389, createdName=jeanqiuqiu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536216, encodeId=ac6f153621665, content=<a href='/topic/show?id=75fbe553800' target=_blank style='color:#2F92EE;'>#突破性疗法#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=102, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=75538, encryptionId=75fbe553800, topicName=突破性疗法)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7eca12667393, createdName=zhwj, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1624586, encodeId=5dab1624586b2, content=<a href='/topic/show?id=3f2d2e7087' target=_blank style='color:#2F92EE;'>#ARNI#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=106, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2770, encryptionId=3f2d2e7087, topicName=ARNI)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f41c20676233, createdName=gongliu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=)]
    2021-02-28 zhwj

    #突破性疗法#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1448677, encodeId=d2ef14486e73c, content=<a href='/topic/show?id=aaf21412e7e' target=_blank style='color:#2F92EE;'>#PFS#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=110, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14127, encryptionId=aaf21412e7e, topicName=PFS)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=47f75418389, createdName=jeanqiuqiu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536216, encodeId=ac6f153621665, content=<a href='/topic/show?id=75fbe553800' target=_blank style='color:#2F92EE;'>#突破性疗法#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=102, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=75538, encryptionId=75fbe553800, topicName=突破性疗法)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=7eca12667393, createdName=zhwj, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1624586, encodeId=5dab1624586b2, content=<a href='/topic/show?id=3f2d2e7087' target=_blank style='color:#2F92EE;'>#ARNI#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=106, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2770, encryptionId=3f2d2e7087, topicName=ARNI)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f41c20676233, createdName=gongliu, createdTime=Sun Feb 28 01:10:07 CST 2021, time=2021-02-28, status=1, ipAttribution=)]
    2021-02-28 gongliu

    #ARNI#

    0