Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2020 Jun;80(9):923-929. doi: 10.1007/s40265-020-01330-y.
Pemigatinib (PEMAZYRE™), a small molecule inhibitor of fibroblast growth factor receptor (FGFR) 1, FGFR2 and FGFR3, received accelerated approval in April 2020 in the USA for the treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma and a FGFR2 fusion or other rearrangement, as detected by a US FDA-approved test. Developed by Incyte Corporation, it is the first targeted treatment for cholangiocarcinoma in the USA. The recommended dosage of pemigatinib is 13.5 mg once daily, administered orally with or without food, on days 1-14 of a 21-day cycle until disease progression or unacceptable toxicity. Pemigatinib received orphan designation for the treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB or FGFR1, or with PCM1-JAK2 in August 2019 in the USA. A regulatory assessment for pemigatinib as a treatment for adults with locally advanced or metastatic cholangiocarcinoma and a FGFR2 fusion or rearrangement that is relapsed or refractory after ≥ 1 line of systemic therapy is underway in the EU. Pemigatinib is also undergoing clinical development in various countries worldwide for use in several other FGFR-driven malignancies (e.g. solid tumour, urothelial carcinoma). This article summarizes the milestones in the development of pemigatinib leading to this first approval for the treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma and a FGFR2 fusion or other rearrangement, as detected by a US FDA-approved test.
培米替尼（Pemazyre™）是一种成纤维细胞生长因子受体（FGFR）1、FGFR2 和 FGFR3 的小分子抑制剂，于 2020 年 4 月在美国获得加速批准，用于治疗先前接受过治疗、不可切除的局部晚期或转移性胆管癌且存在 FGFR2 融合或其他重排的成年患者，该重排由美国食品和药物管理局（FDA）批准的检测方法检出。该药物由 Incyte 公司开发，是美国首个用于胆管癌的靶向治疗药物。培米替尼的推荐剂量为 13.5mg，每日一次，口服，无论是否与食物同服，21 天为一个周期，在第 1-14 天给药，直至疾病进展或出现不可接受的毒性。培米替尼于 2019 年 8 月在美国获得治疗伴有嗜酸性粒细胞增多和 PDGFRA、PDGFRB 或 FGFR1 重排或伴有 PCM1-JAK2 重排的髓系/淋巴肿瘤的孤儿药认定。一项针对培米替尼用于治疗先前接受过治疗、不可切除的局部晚期或转移性胆管癌且存在 FGFR2 融合或重排的成年患者的监管评估正在欧盟进行，这些患者在接受了≥1 线系统治疗后疾病复发或对治疗不耐受。培米替尼在全球多个国家也正在进行临床开发，用于治疗其他几种 FGFR 驱动的恶性肿瘤（如实体瘤、尿路上皮癌）。本文总结了培米替尼的开发里程碑，这些里程碑促成了该药在美国的首次批准，用于治疗先前接受过治疗、不可切除的局部晚期或转移性胆管癌且存在 FGFR2 融合或重排的成年患者，该重排由美国 FDA 批准的检测方法检出。
