# Pemigatinib

Source: https://onco.cc/drugs/pemigatinib/  
OnCo record `pemigatinib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Pemigatinib was the first targeted therapy for bile duct cancer, for tumours with an FGFR2 gene fusion.

## Summary

Pemigatinib is a selective oral inhibitor of FGFR1, 2 and 3, blocking the constitutively active receptor produced when FGFR2 is fused to another gene. It is used in previously treated cholangiocarcinoma with an FGFR2 fusion or rearrangement and was the first targeted therapy approved for bile duct cancer. FIGHT-202 showed an ORR of 37%, median PFS of 7.0 months and median OS of 17.5 months, with no responses in other FGF/FGFR alterations; accelerated approval followed in April 2020 (EU 2021), and it is also approved for FGFR1-rearranged myeloid and lymphoid neoplasms. Hyperphosphataemia (60%), alopecia, diarrhoea, nail toxicity and serous retinal detachment are on-target effects needing phosphate binders and eye checks. First-line FIGHT-302 was discontinued, so its place remains second line. For a newcomer, it is the first pill that targets a specific gene change in bile duct cancer.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Pemazyre
- Modality: Small-molecule kinase inhibitor (FGFR1-3)
- Mechanism: Selective oral FGFR1-3 inhibitor.
- Approvals: US 2020: Previously treated FGFR2-fusion/rearranged cholangiocarcinoma (accelerated); EU 2021: Same; UK 2021: Locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement after systemic therapy; NICE TA722 recommended 25 August 2021 with a commercial arrangement
- Dosing: Oral; 13.5 mg daily for 14 days of each 21-day cycle

## Notes

- Gallbladder cancer: the FDA indication is cholangiocarcinoma with an FGFR2 fusion or rearrangement (Pemazyre label), and FIGHT-202 enrolled cholangiocarcinoma only; FGFR2 fusions are a feature of intrahepatic disease and gallbladder cancer was not part of the approval population, so there is no gallbladder evidence for pemigatinib beyond case reports.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Pemigatinib
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pemigatinib
- NICE TA722: pemigatinib for FGFR2 fusion or rearrangement cholangiocarcinoma (25 August 2021): https://www.nice.org.uk/guidance/ta722

## Connected records

- biomarkers: [FGFR2 fusion or rearrangement](https://onco.cc/biomarkers/fgfr2-fusion-rearrangement/)
- cancers: [Biliary tract cancer (all types)](https://onco.cc/cancers/biliary-tract-cancer/), [Biliary tract cancer (cholangiocarcinoma)](https://onco.cc/cancers/cholangiocarcinoma/), [Gallbladder cancer](https://onco.cc/cancers/gallbladder/), [Intrahepatic cholangiocarcinoma](https://onco.cc/cancers/intrahepatic-cholangiocarcinoma/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [FGFR1](https://onco.cc/targets/fgfr1/), [FGFR2](https://onco.cc/targets/fgfr2/)
- companies: [Incyte](https://onco.cc/companies/incyte/)
- terms: [FGFR2 fusions and rearrangements](https://onco.cc/terms/fgfr2-fusion/)
- trials: [A Phase II Nationwide, Fully Decentralized, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreatic Cancer With FGFR Genetic](https://onco.cc/trials/nct06906562/), [A Phase II Study of Pemigatinib Plus Durvalumab in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients With FGFR-2 Fusion or Rearrangement](https://onco.cc/trials/nct06728410/), [Alternating Chemo-immunotherapy and FGFR Inhibitor for FGFR-positive Biliary or Cholangiocarcinoma](https://onco.cc/trials/nct07780838/), [FIGHT-202](https://onco.cc/trials/fight-202/), [FIGHT-302](https://onco.cc/trials/fight-302/), [Pemigatinib Combined With Durvalumab for Previously Treated Biliary Tract Carcinoma](https://onco.cc/trials/nct06530823/), [Pemigatinib Plus PD-1 Inhibitor With or Without Chemotherapy in FGFR2 Fusion/Rearrangement-Positive Cholangiocarcinoma](https://onco.cc/trials/nct07786766/)
- ideas: [ctDNA-guided switching among FGFR inhibitors](https://onco.cc/ideas/idea-btc-ctdna-fgfr-resistance/)
- pathways: [FGF / FGFR signalling](https://onco.cc/pathways/fgfr-signalling/), [Proteoglycans in cancer](https://onco.cc/pathways/proteoglycans-in-cancer/)
- drugs: [Infigratinib](https://onco.cc/drugs/infigratinib/)
- key papers: [FIGHT-202: pemigatinib for previously treated cholangiocarcinoma with FGFR2 fusions or rearrangements](https://onco.cc/key-papers/paper-fight-202-pemigatinib-lancet-oncol-2020/)
- roadmaps: [Targeted therapy roadmap: imatinib → designed for resistance → the undruggable drivers fall](https://onco.cc/roadmaps/targeted-therapy-roadmap/)

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