Vogel A, Bridgewater J, Edeline J, et al. Biliary tract cancer: ESMO clinical practice guideline for diagnosis, treatment and follow-up*. Ann Oncol. 2023;34(2):127–40.

Article  CAS  PubMed  Google Scholar 

Banales JM, Marin JJG, Lamarca A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17(9):557–88.

Article  PubMed  PubMed Central  Google Scholar 

Javle M, Lee S, Azad NS, et al. Temporal changes in cholangiocarcinoma incidence and mortality in the United States from 2001 to 2017. Oncologist. 2022;27(10):874–83.

Article  PubMed  PubMed Central  Google Scholar 

Saha SK, Zhu AX, Fuchs CS, et al. Forty-year trends in cholangiocarcinoma incidence in the US: intrahepatic disease on the rise. Oncologist. 2016;21:594–9.

Article  PubMed  PubMed Central  Google Scholar 

Bertuccio P, Malvezzi M, Carioli G, et al. Global trends in mortality from intrahepatic and extrahepatic cholangiocarcinoma. J Hepatol. 2019;71(1):104–14.

Article  PubMed  Google Scholar 

Florio AA, Ferlay J, Znaor A, et al. Global trends in intrahepatic and extrahepatic cholangiocarcinoma incidence from 1993 to 2012. Cancer. 2020;126(11):2666–78.

Article  PubMed  Google Scholar 

Ioffe D, Phull P, Dotan E. Optimal mnagement of patients with advanced or metastatic cholangiocarcinoma: an evidence-based review. Cancer Manag Res. 2021;13:8085–98.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bekaii-Saab TS, Valle JW, Van Cutsem E, et al. FIGHT-302: first-line pemigatinib vs gemcitabine plus cisplatin for advanced cholangiocarcinoma with FGFR2 rearrangements. Future Oncol. 2020;16(30):2385–99.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Harding JJ, Khalil DN, Fabris L, et al. Rational development of combination therapies for biliary tract cancers. J Hepatol. 2023;78(1):217–28.

Article  CAS  PubMed  Google Scholar 

Neumann O, Burn TC, Allgäuer M, et al. Genomic architecture of FGFR2 fusions in cholangiocarcinoma and its implication for molecular testing. Br J Cancer. 2022;127(8):1540–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Goyal L, Kongpetch S, Crolley VE, et al. Targeting FGFR inhibition in cholangiocarcinoma. Cancer Treat Rev. 2021;95: 102170.

Article  CAS  PubMed  Google Scholar 

Hoy SM. Pemigatinib: first approval. Drugs. 2020;80(9):923–9.

Article  PubMed  Google Scholar 

Incyte Biosciences Distribution B.V., Pemazyre 4.5 mg, 9 mg and 13.5 mg tablets : EU summary of product charcteristics. 2021. https://www.ema.europa.eu. Accessed 1 Nov 2023.

Incyte Corporation. PEMAZYRE™ (pemigatinib) tablets, for oral use: US prescribing information. 2022. https://www.pemazyre.com/pi/pi. Accessed 1 Nov 2023.

Incyte Corporation. Incyte announces approval of Pemazyre® (pemigatinib) in Japan for the treatment of patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy [media release]. 23 Mar 2021. http://www.incyte.com.

Incyte Corporation. Incyte announces Japanese approval of pemazyre® (Pemigatinib) for the treatment of patients with myeloid/lymphoid neoplasms (MLNs) [media release]. 27 Mar 2023. http://www.incyte.com.

Liu PCC, Koblish H, Wu L, et al. INCB054828 (pemigatinib), a potent and selective inhibitor of fibroblast growth factor receptors 1, 2, and 3, displays activity against genetically defined tumor models. PLoS ONE. 2020;15(4):1–16.

Article  CAS  Google Scholar 

Rodon J, Damian S, Furqan M, et al. Clinical and translational findings of pemigatinib in previously treated solid tumors with activating FGFR1-3 alterations in the FIGHT-207 study [abstract no. CT016 plus poster]. Cancer Res. 2023;83(8 Suppl):CT016.

Article  Google Scholar 

Kommalapati A, Tella SH, Borad M, et al. FGFR inhibitors in oncology: insight on the management of toxicities in clinical practice. Cancers (Basel). 2021;13(12):2968.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ji T, Chen X, Liu X, et al. Population pharmacokinetics analysis of pemigatinib in patients with advanced malignancies. Clin Pharmacol Drug Dev. 2022;11(4):454–66.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Subbiah V, Iannotti NO, Gutierrez M, et al. FIGHT-101, a first-in-human study of potent and selective FGFR 1–3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies. Ann Oncol. 2022;33(5):522–33.

Article  CAS  PubMed  Google Scholar 

Ji T, Rockich K, Epstein N, et al. Evaluation of the pharmacokinetics of pemigatinib in patients with impaired hepatic or renal function. Br J Clin Pharmacol. 2022;88(1):237–47.

Article  CAS  PubMed  Google Scholar 

Ji T, Rockich K, Epstein N, et al. Evaluation of drug-drug interactions of pemigatinib in healthy participants. Eur J Clin Pharmacol. 2021;77(12):1887–97.

Article  CAS  PubMed  Google Scholar 

Ji T, Chen X, Yeleswaram S. Evaluation of drug-drug interaction potential for pemigatinib using physiologically based pharmacokinetic modeling. CPT Pharmacometrics Syst Pharmacol. 2022;11(7):894–905.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020;21(5):671–84.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated locally advanced/metastatic cholangiocarcinoma (CCA): update of FIGHT-202 [abstract no. 4086 plus poster]. J Clin Oncol. 2021;39(15 Suppl):4086.

Article  Google Scholar 

Vogel A, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated locally advanced or metastatic cholangiocarcinoma: final results from FIGHT-202 [abstract no. O-2 plus poster]. Ann Oncol. 2022;33(S4):S379.

Article  Google Scholar 

Bibeau K, Féliz L, Lihou CF, et al. Progression-free survival in patients with cholangiocarcinoma with or without FGF/FGFR alterations: a FIGHT-202 post hoc analysis of prior systemic therapy response. JCO Precis Oncol. 2022;6:1–10.

Google Scholar 

Abou-Alfa G, Sahai V, Hollebecque A, et al. Progression-free survival in patients with cholangiocarcinoma with FGFR2 fusions or rearrangements: a FIGHT-202 post-hoc analysis of prior systemic therapy response [abstract no. SO-4 plus oral presentation]. Ann Oncol. 2021;32(Suppl 3):S203–4.

Article  Google Scholar 

European Medicines Agency. Pemazyre (pemigatinib): assessment report. https://www.ema.europa.eu. Accessed 17 Nov 2023.

Silverman IM, Hollebecque A, Friboulet L, et al. Clinicogenomic analysis of FGFR2-rearranged cholangiocarcinoma identifies correlates of response and mechanisms of resistance to pemigatinib. Cancer Discov. 2021;11(2):326–39.

Article  CAS  PubMed  Google Scholar 

Shi G, Huang X, Wen T, et al. Pemigatinib in Chinese patients with advanced/metastatic or surgically unresectable cholangiocarcinoma Including FGFR2 fusion or rearrangement: updated overall survival from an open-label, single-arm, multicenter phase II study [abstract no. CT153]. Cancer Res. 2023;83(8 Suppl):319.

Google Scholar 

Shi GM, Huang XY, Wen TF, et al. Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: a phase II study. Cancer Med. 2023;12(4):4137–46.

Article  CAS  PubMed  Google Scholar 

Subbiah V, Verstovsek S. Clinical development and management of adverse events associated with FGFR inhibitors. Cell Rep Med. 2023;4(10):101204.

Article  CAS  PubMed  PubMed Central  Google Scholar 

NCCN clinical practice guidelines in oncology (NCCN guidelines®) biliary tract cancers version 2.2023—May 10, 2023. https://www.nccn.org. Accessed 1 Nov 2023.

Normanno N, Martinelli E, Melisi D, et al. Role of molecular genetics in the clinical management of cholangiocarcinoma. ESMO Open. 2022;7(3):100505.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Angerilli V, Fornaro L, Pepe F, et al. FGFR2 testing in cholangiocarcinoma: translating molecular studies into clinical practice. Pathologica. 2023;115(2):71–82.

Article  PubMed  PubMed Central  Google Scholar 

Paine A, Thom H, Wacheck V, et al. Matching-adjusted indirect comparison of futibatinib versus chemotherapy and pemigatinib in cholangiocarcinoma patients with FGFR2 fusions/rearrangements [abstract no. CO78 plus poster]. Value Health. 2022;25(12 Suppl):S33.

Article  Google Scholar 

Lindley A, Prager G, Bitzer M, et al. Global expanded access program (EAP) to pemigatinib for patients (pts) with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) and FGFR2 fusions or rearrangements [abstract no. 335]. Oncol Res Treat. 2022;45(Suppl 3):81.

Google Scholar 

National Institute for Health Care Excellence. Pemigatinib for treating relapsed or refractory advanced cholangiocarcinoma with FGFR2 fusion or rearrangement. Technology appraisal guidance [TA722]. https://www.nice.org.uk. Accessed 1 Nov 2023.

Scottish Medicines Consortium. SMC2399. Pemigatinib 4. 5mg, 9 mg, and 13. 5mg tablets (Pemazyre®). https://www.scottishmedicines.org.uk. Accessed 17 Nov 2023.