胆管癌首个口服靶向药-培美替尼

培美替尼（又称培米替尼，英文商品名：pemazyre）是全球首款胆管癌靶向药物，2020年4月在美国上市，治疗既往接受过治疗、具有FGFR2融合/重排且不可手术切除的成人局部晚期或转移性胆管癌成患者。 Since the FGFR2 gene is almost exclusively found in intrahepatic cholangiocarcinoma, it is a targeted drug for intrahepatic cholangiocarcinoma.

Cholangiocarcinoma is a highly malignant tumor with strong invasiveness and poor prognosis. Many patients have lost the chance of surgery when diagnosed.晚期胆管癌系统治疗方案极其匮乏，一线标准化疗的客观缓解率（ORR）仅15%-26%，常发生耐药，且无标准二线治疗方案，临床亟需新的治疗方案改善患者生存状况。 2020年4月，美国FDA加速批准了靶向药培美替尼培美替尼（pemazyre）的上市申请，用于治疗FGFR2基因融合/重排的经治晚期胆管癌患者。 As the first targeted drug for cholangiocarcinoma, pemetinib is expected to change the clinical treatment landscape for patients with cholangiocarcinoma.

培美替尼在美国的获批基于代号为FIGHT-202(NCT02924376)的临床试验，试验结果显示，在107例存在FGFR2融合/重排的患者中，所有患者对治疗都有反应，培美替尼单药治疗的总缓解率为36%，其中包括2.8%的完全缓解和33%的部分缓解;疾病控制率为82%。 Median duration of response was 9.1 months; 63% of patients achieved response for 6 months or longer, and 18% maintained response for 12 months or longer.以上数据说明，用于存在FGFR2融合/重排的胆管癌二线治疗，有82%的患者肿瘤得到控制，并且有33%的患者肿瘤明显缩小或改善。

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.