PDGFRα靶向新药avapritinib在中国台湾申请用于治疗晚期胃肠道间质瘤成人患者

2020-03-29 MedSci MedSci原创

已递交TFDA的临床数据显示,avapritinib在PDGFRα外显子18突变(包括D842V突变)的晚期GIST患者中ORR达 84%,且不良反应大多为1级或2级

  • 已递交TFDA的临床数据显示,avapritinib在PDGFRα外显子18突变(包括D842V突变)的晚期GIST患者中ORR达 84%,且不良反应大多为1级或2级

  • Avapritinib已于2020年1月获美国FDA批准,是目前首个且唯一上市的GIST精准靶向药物,用于治疗PDGFRα外显子18突变的、不可手术切除或转移性GIST患者

3月27日,基石药业宣布公司已向台湾卫生福利部食品药物管理署(TFDA)提交胃肠道间质瘤(GIST)精准靶向药物avapritinib的上市申请,且该药物已于2020年3月9日获TFDA新药优先审查资格认定。该药物将用于治疗携带血小板衍生生长因子受体α(PDGFRα)外显子18突变(包括PDGFRα D842V突变)的、不可手术切除或转移性GIST成人患者。Avapritinib由公司合作伙伴Blueprint Medicines开发,也是基石药业第二款递交上市申请的药物。
 
基石药业董事长兼首席执行官江宁军博士表示:“一年内,基石药业已先后递交了两个同类首创(first-in-class)精准靶向药物的上市申请,这不仅体现公司对于临床迫切需求的关注和回应,也显示了公司正顺利向商业化转型。GIST是一种罕见的肿瘤,且目前大中华区已获批的疗法针对PDGFRα D842V突变均无疗效。除台湾外,今年上半年我们计划针对同一适应症在中国大陆递交avapritinib的新药上市申请,以满足更多患者之需。”
 
基石药业首席医学官杨建新博士表示:“Avapritinib是一种强效、高选择性的在研口服KIT和PDGFRα抑制剂。今年1月,FDA已批准其用于治疗携带PDGFR外显子18突变的不可手术切除或转移性GIST成人患者,成为目前首个且唯一在美国上市的GIST精准靶向药物。目前向TFDA递交的临床数据显示,avapritinib在PDGFRα外显子18突变(包括D842V突变)的晚期GIST患者中显示出前所未有的显著抗肿瘤活性,客观缓解率(ORR)达 84%, 且大多数不良反应均为1级或2级,中位缓解持续时间(DOR)尚未达到,61%的患者DOR≥6个月。"
 
GIST为发生于胃壁或小肠中的肉瘤,高发年龄为50-80岁。大约90%的GIST的发生与KIT或PDGFRα基因的突变导致的细胞生长失调有关。由于GIST对化疗和放疗不敏感,目前晚期GIST的治疗方案主要为依次使用酪氨酸激酶抑制剂(TKIs)。然而,携带PDGFR外显子18 D842V突变的患者对现有大中华区已批准的TKI药物均不敏感,ORR为0%,中位无进展生存期仅3至5个月,总生存期约15个月。除了D842V之外,既往已批准的GIST治疗药物对于其它罕见的PDGFR外显子18突变疗效也并不理想。
 
基石药业已与BlueprintMedicines达成独家合作和授权,获得了包括avapritinib在内的多款药物在大中华区的独家开发和商业化授权。BlueprintMedicines保留在世界其他地区开发及商业化相关药物的权利。

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    2020-07-18 hxzhang
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    2020-06-15 hbwxf
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    2020-03-31 liuyiping

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