Pemigatinib: First Approval
Pemigatinib: First Approval
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DOI：
10.1007/s40265-020-01330-y
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发表时间：
2020-06-01
期刊：
影响因子：
11.5
通讯作者：
Hoy, Sheridan M.
中科院分区：
文献类型：
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作者：
Hoy, Sheridan M.
培美替尼（PEMAZYRE（TM））是一种成纤维细胞生长因子受体（FGFR）1、FGFR 2和FGFR 3的小分子抑制剂，于2020年4月在美国获得加速批准，用于治疗患有既往治疗过的、不可切除的、局部晚期或转移性胆管癌以及FGFR 2融合或其他重排的成人，如美国FDA批准的检测所示。它由Incyte公司开发，是美国第一个针对胆管癌的靶向治疗。pemigatinib的推荐剂量为13.5 mg，每日一次，在21天周期的第1-14天口服给药，伴或不伴食物，直至疾病进展或出现不可接受的毒性。Pemigatinib于2019年8月在美国获得孤儿药资格，用于治疗伴嗜酸性粒细胞增多和PDGFRA、PDGFRB或FGFR 1重排或伴PCM 1-JAK 2的骨髓/淋巴肿瘤。欧盟正在对pemigatinib作为局部晚期或转移性胆管癌和FGFR 2融合或重排成人患者的治疗进行监管评估，这些患者在>= 1线全身治疗后复发或难治。Pemigatinib也正在全球多个国家进行临床开发，用于其他几种FGFR驱动的恶性肿瘤（例如实体瘤、尿路上皮癌）。这篇文章总结了pemigatinib开发的里程碑，导致首次批准用于治疗既往接受过治疗的、不可切除的、局部晚期或转移性胆管癌和FGFR 2融合或其他重排的成人，如美国FDA批准的检测所检测到的。
Pemigatinib (PEMAZYRE (TM)), 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.