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About PEMAZYRE — 2 of 3
PEMAZYRE is the first approved, molecularly targeted treatment for FGFR2 fusion-positive CCA
Pemigatinib is a potent inhibitor of FGFR1, 2 and 31,23
- Pemigatinib is a kinase inhibitor of FGFR1, 2 and 3. It inhibits FGFR phosphorylation and signalling and decreases cell viability in cells expressing FGFR genetic alterations, including point mutations, amplifications, and fusions or rearrangements1
- FGFR2 fusions are strong oncogenic drivers and are the most common FGFR alteration, occurring almost exclusively in 10–16% of iCCA1
Adapted from Swiss Professional Information PEMAZYRE® (pemigatinib) on www.swissmedicinfo.ch; ref. 1.
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CH/PEMA/P/23/0006
Date of preparation: April 2023
Date of preparation: April 2023
References
- Swiss Professional Information PEMAZYRE® (pemigatinib) on www.swissmedicinfo.ch.
- Rizvi S and Borad MJ. The rise of the FGFR inhibitor in advanced biliary cancer: the next cover of Time magazine? J Gastrointest Oncol. 2016;7:789–96.
- Ghouri YA, et al. Cancer review: Cholangiocarcinoma. J Carcinog. 2015;14:1.
- Sharma P and Yadav S. Demographics, tumor characteristics, treatment, and survival of patients with Klatskin tumors. Ann Gastroenterol. 2018;31:231–6.
- Blechacz B. Cholangiocarcinoma: Current knowledge and new developments. Gut Liver. 2017;11:13–26.
- Valle JW, et al. New horizons for precision medicine in biliary tract cancers. Cancer Discov. 2017;7:943–62.
- Bañales JM, et al. Cholangiocarcinoma 2020: The next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17:557–88.
- Bañales JM, et al. Expert consensus document: Cholangiocarcinoma: Current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol. 2016;13:261–80.
- Bertuccio P, et al. A comparison of trends in mortality from primary liver cancer and intrahepatic cholangiocarcinoma in Europe. Ann Oncol. 2013;24:1667–74.
- Blechacz B, et al. Clinical diagnosis and staging of cholangiocarcinoma. Nat Rev Gastroenterol Hepatol. 2011;8:512–22.
- Forner A, et al. Clinical presentation, diagnosis and staging of cholangiocarcinoma. Liver Int. 2019;39(suppl 1):98–107.
- Vogel A, et al. Biliary tract cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up. Ann Oncol. 2023;34:127–40.
- Lowery MA, et al. Comprehensive molecular profiling of intrahepatic and extrahepatic cholangiocarcinomas: potential targets for intervention. Clin Cancer Res. 2018;24:4154–61.
- Jain A, et al. Cholangiocarcinoma with FGFR genetic aberrations: A unique clinical phenotype. JCO Precis Oncol. 2018;2:1–12.
- Ross JS, et al. New routes to targeted therapy of intrahepatic cholangiocarcinomas revealed by next-generation sequencing. Oncologist. 2014;19:235–42.
- Farshidfar F, et al. Integrative genomic analysis of cholangiocarcinoma identifies distinct IDH-mutant molecular profiles. Cell Rep. 2017;18:2780–94.
- Graham RP, et al. Fibroblast growth factor receptor 2 translocations in intrahepatic cholangiocarcinoma. Hum Pathol. 2014;45:1630–8.
- Churi CR, et al. Mutation profiling in cholangiocarcinoma: Prognostic and therapeutic implications. PLoS One. 2014;9:e115383.
- Arai Y, et al. Fibroblast growth factor receptor 2 tyrosine kinase fusions define a unique molecular subtype of cholangiocarcinoma. Hepatology. 2014;59:1427–34.
- Borad MJ, et al. Fibroblast growth factor receptor 2 fusions as a target for treating cholangiocarcinoma. Curr Opin Gastroenterol. 2015;31:264–68.
- Silverman IM, et al. Clinicogenomic analysis of FGFR2-rearranged cholangiocarcinoma identifies correlates of response and mechanisms of resistance to pemigatinib. Cancer Discov. 2021;11:326–39.
- Barr FG. Fusion genes in solid tumors: The possibilities and the pitfalls. Expert Rev Mol Diagn. 2016;16:921–3.
- Liu PCC, 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:e0231877.
- Abou-Alfa GK, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: A multicentre, open-label, phase 2 study. Lancet Oncol. 2020;21:671–84.
All references are available upon request
Abbreviations
1L
first-line
5-FU
fluorouracil
AR
adverse reaction
BRAF
v-raf murine sarcoma viral oncogene homolog B1
BTC
biliary tract cancer
CCA
cholangiocarcinoma
CI
confidence interval
CNS
central nervous system
CR
complete response
CYP2B6
cytochrome P450 2B6
CYP3A4
cytochrome P450 3A4
dCCA
distal cholangiocarcinoma
dMMR
mismatch repair deficient
DOR
duration of response
eCCA
extrahepatic cholangiocarcinoma
ECOG PS
Eastern Cooperative Oncology Group Perfomance Status
EGFR
epidermal growth factor receptor
EMA
European Medicines Agency
ESCAT
ESMO Scale for Clinical Actionability of Molecular Targets
ESMO
European Society for Medical Oncology
EU
European Union
FDA
US Food and Drug Administration
FGFR
fibroblast growth factor receptor
FOLFOX
folinic acid, fluorouracil and oxaliplatin
HER
human epidermal growth factor receptor
iCCA
intrahepatic cholangiocarcinoma
IDH
isocitrate dehydrogenase
KM
Kaplan-Meier
KRAS
Kirsten rat sarcoma viral oncogene homolog
MATE1
multidrug and toxin extrusion protein 1
MDT
multidisciplinary team
mg
milligram
mg/dL
milligram per decilitre
MSI-H
microsatellite instability-high
NGS
next-generation sequencing
NTRK
neurotrophic tyrosine receptor kinase
OCT
optical coherence tomography
OCT2
organic cation transporter-2
ORR
overall response rate
OS
overall survival
pCCA
perihilar cholangiocarcinoma
PD-1
programmed cell death protein
PFS
progression-free survival
P-gp
P-glycoprotein
PI
Prescribing Information
PPES
palmar-plantar erythrodysaesthesia syndrome
PR
partial response
RECIST
Response Evaluation Criteria in Solid Tumours
Abbreviated Prescribing Information
▼ This medicinal product is subject to additional monitoring. For further information, see product information Pemazyre on www.swissmedicinfo.ch.
Revision date: January 2023. Marketing authorisation holder: Incyte Biosciences International Sàrl, CH-1110 Morges. Refer to www.swissmedicinfo.ch for detailed information.
Resources and support
For further information, please refer to the PEMAZYRE Swissmedic Professional Information.1
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