About Cholangiocarcinoma
Cholangiocarcinoma (CCA) is a rare, aggressive malignancy with a poor prognosis.1,2 Approximately 8000 people in the US are diagnosed with CCA each year.3
Abnormal FGFR2 signaling can be an oncogenic driver of CCA4
FGFRs can play a pivotal role in CCA5-9
- FGFRs 1-4 are involved in cell proliferation, cell differentiation, cell migration, and cell survival5
- Aberrant FGFR signaling has been linked to the potential development and maintenance of CCA5-9
- In CCA, genetic alterations, including FGFR2 fusions, are known to play a role in carcinogenesis by promoting cancer cell growth and survival6
FGFR2 is the primary therapeutic target for certain CCA treatments10
- Treatment that targets FGFR2 may lead to tumor inhibition for patients whose CCA is driven by an FGFR2 fusion or rearrangement10
However, there are off-tumor toxicities associated with inhibition of other FGFRs,5,11 including:
- Hyperphosphatemia–a pharmacodynamic effect of FGFR inhibition, mainly driven by inhibition of FGFR1 signaling11,12
- GI effects such as diarrhea–thought to be driven by inhibition of FGFR4 signaling11,13-15
Considering the role of FGFRs and the effects of their inhibition can support treatment decisions5,11
- Proactive molecular testing for FGFR2 fusions or other rearrangements can help you identify which patients with CCA may be appropriate for targeted therapy16,17
- FGFR2 fusions are detectable early in disease progression, making identifying these alterations in patients at diagnosis especially important4
FGFR-targeted therapies provide the opportunity for an individualized approach in the treatment of advanced CCA in patients with an FGFR2 fusion or other rearrangement5,11
Molecular testing for CCA
Proactive molecular testing can help you identify which patients may be appropriate for targeted therapy16,17

Next-generation sequencing can detect FGFR2 fusions
Patients with FGFR2 fusions and gene rearrangements may be appropriate for targeted therapy with an FGFR inhibitor.4NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend molecular testing for patients with unresectable or metastatic CCA17*†‡
*See the guidelines online at NCCN.org for the full recommendation.
†NCCN makes no warranties of any kind whatsoever regarding their content, use, or application and disclaims any responsibility for their application or use in any way.
‡Category 2A: Based upon lower-level evidence, there is uniform NCCN consensus that the intervention is appropriate.
NCCN=National Comprehensive Cancer Network.
FoundationOne® CDx for detection of FGFR2 fusions
Information on FDA-approved test(s) for the detection of FGFR2 fusions or rearrangements in CCA is available at www.fda.gov/CompanionDiagnostics.Learn about an FDA-approved treatment.
- Valle JW, et al. Cancer Discov. 2017;7(9):943-962
- Banales JM, et al. Nat Rev Gastroenterol Hepatol. 2020;17(9):557-588.
- American Cancer Society. https://www.cancer.org/cancer/bile-duct-cancer/about/key-statistics. Accessed March 1, 2021
- Rizvi S, Borad MJ. J Gastrointest Oncol. 2016;7(5):789-796.
- Touat M, et al. Clin Cancer Res. 2015;21(12):2684-2694.
- Pellino A, et al. Transl Gastroenterol Hepatol. 2018;3:40.
- Javle M, et al. Cancer. 2016;122(24):3838-3847.
- Borad MJ, et al. Curr Opin Gastroenterol. 2015;31(3):264-268.
- Goyal L, et al. Cancer Discov. 2019;9(8): 1064-1079.
- TRUSELTIQ Prescribing Information. Brisbane, CA: QED Therapeutics, Inc.; May 2021.
- Chae YK, et al. Oncotarget.2017;8(9):16052-16074.
- Lyou Y, et al. Eur Urol. 2020;78(6):916-924.
- Hierro C, et al. Semin Oncol. 2015;42(6):801-819.
- Mellor HR. Liver Int. 2014;34(6):e1-e9. doi:10.1111/liv.12462.
- Kim RD, et al. Cancer Discov. 2019;9:1696-1707.
- Avogadri F, et al. Presented at: AACR Virtual Annual Meeting II; June 22-24, 2020.
- Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Hepatobiliary Cancers V.3.2021. ©National Comprehensive Cancer Network, Inc. 2021. All rights reserved. Accessed June 15, 2021. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use, or application and disclaims any responsibility for their application or use in any way.
- Lowery MA, et al. Clin Cancer Res. 2018; 24(17):4154-4161. doi:10.1158/1078-0432.CCR-18-0078.
- Sia D, et al. Nat Communications. 2015;6:6087. doi: 10.1038/ncomms7087.
- Chun YS, et al. Cancer Control. 2017;24(3):1-7.
- Ross JS, et al. Oncologist. 2014;19(3):235-242.
- Lamarca A, et al. J Clin Med. 2020;9(9):2854. doi:10.3390/jcm9092854.
- Arai Y, et al. Hepatology. 2014;59(4):1427-1434.
- Kongpetch S, et al. JCO Glob Oncol. 2020;6:628-638.