OnCo
cancersCancer

Biliary tract cancer (cholangiocarcinoma)

Cholangiocarcinoma is cancer of the bile ducts or gallbladder. It is rare and often found late, but it turned out to carry more targetable mutations than almost any other gastrointestinal cancer, and immunotherapy now adds to chemotherapy from the first treatment.

Biliary tract cancers comprise intrahepatic cholangiocarcinoma (iCCA, rising in incidence), perihilar and distal extrahepatic cholangiocarcinoma, and gallbladder cancer. They share late presentation (jaundice, weight loss), a poor prognosis (five-year survival under 20%), and dependence on surgical resection as the only cure, achievable in a minority. Risk factors differ by region: liver flukes and hepatolithiasis in East Asia, primary sclerosing cholangitis in the West, gallstones and chronic inflammation for gallbladder cancer. Biliary drainage is usually a prerequisite for any treatment.

The systemic landscape was gemcitabine-cisplatin alone from ABC-02 (2010) until TOPAZ-1 (durvalumab, 2022) and KEYNOTE-966 (pembrolizumab, 2023) added PD-(L)1 blockade with a modest median benefit but a doubling of two-year survival. Adjuvant capecitabine (BILCAP) is standard after resection. What sets biliary cancer apart is its genomic actionability: roughly 40% of intrahepatic tumours carry FGFR2 fusions (pemigatinib, futibatinib), IDH1 mutations (ivosidenib), HER2 amplification or overexpression (zanidatamab, trastuzumab deruxtecan), NRG1 fusions (zenocutuzumab, approved 2026), BRAF V600E, or MSI-high status, so molecular profiling at diagnosis is guideline-mandated.

The frontier is moving targeted agents into first line (HERIZON-BTC-302 for zanidatamab), overcoming FGFR-inhibitor resistance (tinengotinib in FIRST-308, lirafugratinib), ctDNA-guided sequencing, and finding a biomarker for the immunotherapy long-tail. Liver transplantation for unresectable perihilar tumours (Mayo protocol) and for selected intrahepatic disease is expanding. Open problems include second-line therapy after chemo-immunotherapy, gallbladder cancer's neglect in trials, and early detection in high-risk groups such as primary sclerosing cholangitis.

State of the art today

  • Genotype-directed therapy in ~40% of patients.
  • Four biomarker-directed drug classes approved (FGFR2, IDH1, HER2, NRG1), plus tumour-agnostic BRAF, MSI-H and NTRK options: ~40% of intrahepatic tumours have an actionable alteration.
  • Next-generation FGFR inhibitors targeting resistance mutations are in phase 3 (FIRST-308).
  • Zanidatamab moving into first line for HER2-positive disease (HERIZON-BTC-302).
  • Liver transplantation protocols extend curative options to selected unresectable perihilar tumours.
Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • Chemo-immunotherapy first line with a doubling of two-year survival (TOPAZ-1, KEYNOTE-966).
Who it affects

About 210,000 cases per year worldwide; incidence of intrahepatic cholangiocarcinoma has roughly doubled in Western countries over 30 years; gallbladder cancer is endemic in Chile, northern India, and among Indigenous Americans.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. Intrahepatic cholangiocarcinoma is inside the liver total (C22); gallbladder cancer (C23) is reported separately; extrahepatic bile duct cancer (C24) is not reported as its own site. No country-level estimate exists for biliary tract cancer as OnCo defines it.

Standard of care

12top
Resectable

Surgery + adjuvant capecitabine.

NCCN Guidelines: Biliary Tract Cancers
Advanced

Gem-cis + PD-(L)1; targeted therapy by genotype second line.

NCCN Guidelines: Biliary Tract Cancers
DurvalumabPembrolizumabZanidatamabZenocutuzumabTrastuzumab deruxtecan
Diagnosis and staging

Contrast CT/MRI with MRCP; ERCP or EUS-guided biopsy; molecular profiling (DNA + RNA NGS) for all advanced disease; biliary drainage if jaundiced.

NCCN · Molecular testing recommended (category 2A)
Comprehensive genomic profilingRNA sequencing & expression profilingBiliary stenting and drainageMRI
Resectable

Margin-negative resection (hepatectomy, Whipple, or radical cholecystectomy) with lymphadenectomy; adjuvant capecitabine 6 months (BILCAP).

NCCN · Capecitabine category 2A (preferred)ESMO-MCBS · B
BILCAPRobotic & minimally invasive surgery
Unresectable perihilar in selected patients

Neoadjuvant chemoradiation then liver transplantation (Mayo protocol) at experienced centres.

NCCN · Category 2B, transplant centres only
Liver transplantation for cancer (Milan criteria and beyond)
Advanced, first line

Gemcitabine-cisplatin + durvalumab (TOPAZ-1) or + pembrolizumab (KEYNOTE-966); zanidatamab added in HER2+ disease within HERIZON-BTC-302.

NCCN · Category 1 (preferred)ESMO-MCBS · TOPAZ-1 grade 3
TOPAZ-1KEYNOTE-966Gemcitabine + cisplatinDurvalumabPembrolizumabHERIZON-BTC-302
Advanced, FGFR2 fusion after chemotherapy

Pemigatinib or futibatinib; tinengotinib in FIRST-308 after progression.

NCCN · Category 2A
PemigatinibFutibatinibTinengotinibFIGHT-202FOENIX-CCA2FIRST-308
Advanced, IDH1 mutation after chemotherapy

Ivosidenib (ClarIDHy).

NCCN · Category 1
IvosidenibClarIDHy
Advanced, HER2-positive after chemotherapy

Zanidatamab (IHC 3+ or amplified); trastuzumab deruxtecan (IHC 3+, tumour-agnostic).

NCCN · Category 2A
ZanidatamabTrastuzumab deruxtecan
Advanced, other alterations

Zenocutuzumab (NRG1 fusion, approved 2026); dabrafenib-trametinib (BRAF V600E); pembrolizumab or dostarlimab (MSI-H/dMMR); larotrectinib/entrectinib (NTRK).

NCCN · Category 2A (tumour-agnostic)
ZenocutuzumabDabrafenib + trametinibPembrolizumabDostarlimab
Second-line, no target

FOLFOX (ABC-06, modest benefit); liposomal irinotecan/5-FU had conflicting results (NIFTY positive, NALIRICC negative); clinical trials preferred.

NCCN · FOLFOX category 1
NALIRICC (AIO)Cytotoxic chemotherapy
Locoregional (intrahepatic, liver-confined)

Y-90 radioembolisation, hepatic arterial infusion pump chemotherapy, or SBRT at specialised centres; no phase 3 proof of survival benefit.

NCCN · Category 2B
Radioembolisation (TARE / SIRT, yttrium-90)SBRT / SABR (stereotactic radiotherapy)

Subtypes & biomarkers

top
Subtypes
  • Intrahepatic cholangiocarcinoma (FGFR2 fusion ~10-15%, IDH1 ~15%)
  • Perihilar (Klatskin) cholangiocarcinoma
  • Distal extrahepatic cholangiocarcinoma
  • Gallbladder carcinoma (HER2 ~15-20%)
  • Fluke-associated (Opisthorchis, Clonorchis)
  • PSC-associated
Biomarkers clinicians test

Target prevalence in this cancer

Target / alterationPrevalenceSource
HER2
Higher in gallbladder/extrahepatic
10-20%
Wikipedia
IDH1 / IDH2
10-20%
cBioPortal (TCGA)
FGFR2
10-15%
cBioPortal (TCGA)

How common each drug target or alteration is in this cancer. Population-level and approximate; see the target page for detail. Full matrix.

History

15top
  1. 1965Klatskin describes perihilar cholangiocarcinoma

    Distinct clinicopathologic entity at the hepatic duct confluence.

  2. 1993Mayo Clinic begins neoadjuvant chemoradiation and transplant for perihilar cholangiocarcinoma
    Liver transplantation for cancer (Milan criteria and beyond)
  3. 2010ABC-02: gemcitabine-cisplatin becomes standard
    ABC-02Gemcitabine + cisplatin
  4. 2013FGFR2 fusions and IDH1 mutations characterised as drivers of intrahepatic cholangiocarcinoma
    FGFR2 fusions and rearrangementsIDH1 / IDH2
  5. 2017BILCAP: adjuvant capecitabine adopted
    BILCAP
  6. 2020Pemigatinib: first FGFR2 inhibitor
  7. 2020Pemigatinib: first targeted approval in biliary cancer
    PemigatinibFIGHT-202
  8. 2021Ivosidenib approved (ClarIDHy); infigratinib approved then withdrawn (2022)
    IvosidenibClarIDHy
  9. 2022TOPAZ-1: immunotherapy first line
    Durvalumab
  10. 2022TOPAZ-1: first immunotherapy approval; futibatinib approved
    TOPAZ-1DurvalumabFutibatinibFOENIX-CCA2
  11. 2023KEYNOTE-966: pembrolizumab confirms chemo-IO
    KEYNOTE-966
  12. 2024Zanidatamab for HER2+ BTC
    Zanidatamab
  13. 2024Zanidatamab: first HER2 approval in biliary cancer; T-DXd tumour-agnostic HER2 IHC3+; NALIRICC negative
    ZanidatamabTrastuzumab deruxtecanNALIRICC (AIO)
  14. 2025TOPAZ-1 three-year survival update; FIRST-308 doses first US patient
    TOPAZ-1FIRST-308
  15. 2026Zenocutuzumab approved for NRG1-fusion cholangiocarcinoma; HERIZON-BTC-302 enrolling
    ZenocutuzumabHERIZON-BTC-302

Pipeline

12top
ApprovedBispecific antibody (HER2×HER3)
Zenocutuzumab · Bizengri
Phase 3Small-molecule multi-kinase inhibitor (FGFR1-3, VEGFR, Aurora, JAK)
Tinengotinib
ApprovedBiparatopic bispecific antibody (HER2)
Zanidatamab · Ziihera
ApprovedADC
Trastuzumab deruxtecan · Enhertu
Phase 3ADC
Sonesitatug vedotin
HERIZON-BTC-302FIRST-308ctDNA-guided switching among FGFR inhibitorsLiquid biopsy (ctDNA)Liver transplantation for cancer (Milan criteria and beyond)Radioembolisation (TARE / SIRT, yttrium-90)FAPI PET

Open problems

  • FGFR inhibitor resistance.
  • Late diagnosis.
  • Median survival in advanced disease is still barely a year; the immunotherapy benefit is a small tail with no predictive biomarker.
  • FGFR inhibitor resistance (polyclonal kinase-domain mutations) limits benefit to ~7-9 months; sequencing next-generation agents is unproven.
  • Second-line chemotherapy is weak (FOLFOX) and evidence for liposomal irinotecan is contradictory.
  • Gallbladder cancer is under-represented in trials despite distinct biology and high HER2 prevalence.
  • Early detection in primary sclerosing cholangitis and fluke-endemic regions is unsolved; CA 19-9 is non-specific.
  • Molecular testing is slow and tissue is scarce from brushings; liquid biopsy adoption lags.
  • Adjuvant evidence rests on a technically negative trial (BILCAP); immunotherapy adjuvant trials are ongoing.

Trials

top

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Biliary tract cancer (cholangiocarcinoma)
condition: cholangiocarcinoma
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Landmark trials in OnCo

ABC-02BILCAPClarIDHyFIGHT-202FIRST-308FOENIX-CCA2HERIZON-BTC-302KEYNOTE-966NALIRICC (AIO)TOPAZ-1

Expert centres

top
Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

top
Bring to your appointment

Questions to ask your oncologist about Biliary tract cancer

Generated from this cancer's standard of care, biomarkers, and pipeline · 32 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. Which biomarkers have been tested on my tumour (for example FGFR2 fusions, IDH1, HER2, NRG1, MSI), and what were the results?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include Intrahepatic cholangiocarcinoma, Perihilarcholangiocarcinoma, Distal extrahepatic cholangiocarcinoma.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Resectable

  1. For my situation (resectable), which of the standard options do you recommend and why?
    Why: Guideline options include: Surgery + adjuvant capecitabine.
  2. For my situation (resectable), which of the standard options do you recommend and why?
    Why: Guideline options include: Margin-negative resection (hepatectomy, Whipple, or radical cholecystectomy) with lymphadenectomy; adjuvant capecitabine 6 months (BILCAP).
  3. How do the results of BILCAP apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced

  1. For my situation (advanced), which of the standard options do you recommend and why?
    Why: Guideline options include: Gem-cis + PD-(L)1; targeted therapy by genotype second line.
  2. Am I a candidate for Durvalumab, Pembrolizumab, Zanidatamab or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Diagnosis and staging

  1. For my situation (diagnosis and staging), which of the standard options do you recommend and why?
    Why: Guideline options include: Contrast CT/MRI with MRCP; ERCP or EUS-guided biopsy; molecular profiling (DNA + RNA NGS) for all advanced disease; biliary drainage if jaundiced.

Unresectable perihilar in selected patients

  1. For my situation (unresectable perihilar in selected patients), which of the standard options do you recommend and why?
    Why: Guideline options include: Neoadjuvant chemoradiation then liver transplantation (Mayo protocol) at experienced centres.

Advanced, first line

  1. For my situation (advanced, first line), which of the standard options do you recommend and why?
    Why: Guideline options include: Gemcitabine-cisplatin + durvalumab (TOPAZ-1) or + pembrolizumab (KEYNOTE-966); zanidatamab added in HER2+ disease within HERIZON-BTC-302.
  2. Am I a candidate for Gemcitabine + cisplatin, Durvalumab, Pembrolizumab, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of TOPAZ-1 and KEYNOTE-966 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced, FGFR2 fusion after chemotherapy

  1. For my situation (advanced, fgfr2 fusion after chemotherapy), which of the standard options do you recommend and why?
    Why: Guideline options include: Pemigatinib or futibatinib; tinengotinib in FIRST-308 after progression.
  2. Am I a candidate for Pemigatinib, Futibatinib, Tinengotinib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of FIGHT-202 and FOENIX-CCA2 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced, IDH1 mutation after chemotherapy

  1. For my situation (advanced, idh1 mutation after chemotherapy), which of the standard options do you recommend and why?
    Why: Guideline options include: Ivosidenib (ClarIDHy).
  2. Am I a candidate for Ivosidenib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of ClarIDHy apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced, HER2-positive after chemotherapy

  1. For my situation (advanced, her2-positive after chemotherapy), which of the standard options do you recommend and why?
    Why: Guideline options include: Zanidatamab (IHC 3+ or amplified); trastuzumab deruxtecan (IHC 3+, tumour-agnostic).
  2. Am I a candidate for Zanidatamab, Trastuzumab deruxtecan, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Advanced, other alterations

  1. For my situation (advanced, other alterations), which of the standard options do you recommend and why?
    Why: Guideline options include: Zenocutuzumab (NRG1 fusion, approved 2026); dabrafenib-trametinib (BRAF V600E); pembrolizumab or dostarlimab (MSI-H/dMMR); larotrectinib/entrectinib (NTRK).
  2. Am I a candidate for Zenocutuzumab, Dabrafenib + trametinib, Pembrolizumab or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Second-line, no target

  1. For my situation (second-line, no target), which of the standard options do you recommend and why?
    Why: Guideline options include: FOLFOX (ABC-06, modest benefit); liposomal irinotecan/5-FU had conflicting results (NIFTY positive, NALIRICC negative); clinical trials preferred.
  2. How do the results of NALIRICC (AIO) apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Locoregional (intrahepatic, liver-confined)

  1. For my situation (locoregional (intrahepatic, liver-confined)), which of the standard options do you recommend and why?
    Why: Guideline options include: Y-90 radioembolisation, hepatic arterial infusion pump chemotherapy, or SBRT at specialised centres; no phase 3 proof of survival benefit.

Any stage

  1. Are there clinical trials I could join, for example of Zenocutuzumab, HERIZON-BTC-302, FIRST-308, Tinengotinib?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. Would a second opinion at a high-volume centre change anything, and can you help arrange it?
    Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
  3. What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
    Why: Supportive care improves quality of life and helps patients complete treatment.
  4. I read that “FGFR inhibitor resistance”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Late diagnosis”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.

Everything relevant

101top

Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

20
Antibody-drug conjugate (ADC)Biliary stenting and drainageBispecific antibodiesComprehensive genomic profilingCytotoxic chemotherapyEpigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)FAPI PETImmune checkpoint inhibitorsIntravesical therapy (BCG, chemotherapy, devices, gene and viral therapy)Liquid biopsy (ctDNA)Liver transplantation for cancer (Milan criteria and beyond)MRIPercutaneous hepatic perfusion (chemosaturation)Platinum agentsRadioembolisation (TARE / SIRT, yttrium-90)RNA sequencing & expression profilingRobotic & minimally invasive surgerySBRT / SABR (stereotactic radiotherapy)Small-molecule kinase inhibitorsTopoisomerase-I inhibitors (and ADC payloads)

targets

10
BRAFClaudin 18.2FGFR2HER2HER3IDH1 / IDH2NTRKPD-1PD-L1PRMT5 (MTAP-deleted cancers)

drugs

14
ApprovedSmall-molecule kinase inhibitors (BRAF + MEK)
Dabrafenib + trametinib · Tafinlar + Mekinist
ApprovedMonoclonal antibody (anti-PD-1)
Dostarlimab · Jemperli
ApprovedMonoclonal antibody (anti-PD-L1)
Durvalumab · Imfinzi
ApprovedSmall-molecule irreversible kinase inhibitor (FGFR1-4)
Futibatinib · Lytgobi
Not mapped hereNucleoside analogue (deoxycytidine)
Gemcitabine · Gemzar / Infugem
Not mapped hereCytotoxic regimen
Gemcitabine + cisplatin
ApprovedSmall-molecule IDH1 inhibitor
Ivosidenib · Tibsovo
ApprovedMonoclonal antibody (anti-PD-1)
Pembrolizumab · Keytruda / Keytruda Qlex (SC)
ApprovedSmall-molecule kinase inhibitor (FGFR1-3)
Pemigatinib · Pemazyre
Phase 3ADC
Sonesitatug vedotin
Phase 3Small-molecule multi-kinase inhibitor (FGFR1-3, VEGFR, Aurora, JAK)
Tinengotinib
ApprovedADC
Trastuzumab deruxtecan · Enhertu
ApprovedBiparatopic bispecific antibody (HER2)
Zanidatamab · Ziihera
ApprovedBispecific antibody (HER2×HER3)
Zenocutuzumab · Bizengri

companies

14
AstraZenecaBeOne Medicines (formerly BeiGene)Daiichi SankyoEli Lilly (incl. Loxo)GSKIncyteJazz PharmaceuticalsMerck & Co. (MSD)MerusNovartisServierTaiho Pharmaceutical (Otsuka)TransThera SciencesZymeworks

institutions

13
All India Institute of Medical Sciences, New DelhiAsan Medical CenterCancer Research UKFundación Arturo López PérezIRCCS Humanitas Research HospitalMayo ClinicMemorial Sloan Kettering Cancer CenterNational Cancer Centre SingaporePostgraduate Institute of Medical Education and Research, ChandigarhRamathibodi Hospital, Mahidol UniversitySiriraj Hospital, Mahidol UniversityThe Royal MarsdenZhongshan Hospital, Fudan University

terms

5
CA 19-9FGFR2 fusions and rearrangementsGene fusionIntrahepatic, perihilar, distal and gallbladder cancerTumour-agnostic (tissue-agnostic) approval

trials

10
ABC-02BILCAPClarIDHyFIGHT-202FIRST-308FOENIX-CCA2HERIZON-BTC-302KEYNOTE-966NALIRICC (AIO)TOPAZ-1

pairings

1
Gemcitabine-cisplatin + PD-(L)1 blockade in biliary cancer

ideas

8
A non-profit pharmaceutical company for the cancers markets ignoreAn open model bank for the rare tumours nobody has models forAn open organoid bank for cancers too rare to have modelsctDNA-guided switching among FGFR inhibitorsOne global rare cancer network with n-of-1 and Bayesian trial frameworksOne standing umbrella trial for all rare cancers in a countryTest drugs on the patient's own cancer cells when there is no trial to joinTrial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancer

collections

1
Japanese oncology guidelines (JSMO, JSCO and organ societies)

people

4
Do-Youn OhEileen M. O'ReillyTanios Bekaii-SaabTheodore S. Hong

bottlenecks

1
Rare and paediatric cancers without markets

Latest papers

top
Literature trend1,873 papers in the last 12 months0% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Biliary tract cancer" OR ABSTRACT:"Biliary tract cancer" OR TITLE:"cholangiocarcinoma" OR ABSTRACT:"cholangiocarcinoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Biliary tract cancer (cholangiocarcinoma), not a curated reading list.

Connected

91top

technologies

15
Biliary stenting and drainageBispecific antibodiesComprehensive genomic profilingCytotoxic chemotherapyFAPI PETImmune checkpoint inhibitorsLiquid biopsy (ctDNA)Liver transplantation for cancer (Milan criteria and beyond)MRIPercutaneous hepatic perfusion (chemosaturation)Radioembolisation (TARE / SIRT, yttrium-90)RNA sequencing & expression profilingRobotic & minimally invasive surgerySBRT / SABR (stereotactic radiotherapy)Small-molecule kinase inhibitors

targets

10
BRAFClaudin 18.2FGFR2HER2HER3IDH1 / IDH2NTRKPD-1PD-L1PRMT5 (MTAP-deleted cancers)

drugs

14
ApprovedSmall-molecule kinase inhibitors (BRAF + MEK)
Dabrafenib + trametinib · Tafinlar + Mekinist
ApprovedMonoclonal antibody (anti-PD-1)
Dostarlimab · Jemperli
ApprovedMonoclonal antibody (anti-PD-L1)
Durvalumab · Imfinzi
ApprovedSmall-molecule irreversible kinase inhibitor (FGFR1-4)
Futibatinib · Lytgobi
Not mapped hereNucleoside analogue (deoxycytidine)
Gemcitabine · Gemzar / Infugem
Not mapped hereCytotoxic regimen
Gemcitabine + cisplatin
ApprovedSmall-molecule IDH1 inhibitor
Ivosidenib · Tibsovo
ApprovedMonoclonal antibody (anti-PD-1)
Pembrolizumab · Keytruda / Keytruda Qlex (SC)
ApprovedSmall-molecule kinase inhibitor (FGFR1-3)
Pemigatinib · Pemazyre
Phase 3ADC
Sonesitatug vedotin
Phase 3Small-molecule multi-kinase inhibitor (FGFR1-3, VEGFR, Aurora, JAK)
Tinengotinib
ApprovedADC
Trastuzumab deruxtecan · Enhertu
ApprovedBiparatopic bispecific antibody (HER2)
Zanidatamab · Ziihera
ApprovedBispecific antibody (HER2×HER3)
Zenocutuzumab · Bizengri

companies

9
AstraZenecaIncyteJazz PharmaceuticalsMerck & Co. (MSD)MerusServierTaiho Pharmaceutical (Otsuka)TransThera SciencesZymeworks

institutions

13
All India Institute of Medical Sciences, New DelhiAsan Medical CenterCancer Research UKFundación Arturo López PérezIRCCS Humanitas Research HospitalMayo ClinicMemorial Sloan Kettering Cancer CenterNational Cancer Centre SingaporePostgraduate Institute of Medical Education and Research, ChandigarhRamathibodi Hospital, Mahidol UniversitySiriraj Hospital, Mahidol UniversityThe Royal MarsdenZhongshan Hospital, Fudan University

terms

5
CA 19-9FGFR2 fusions and rearrangementsGene fusionIntrahepatic, perihilar, distal and gallbladder cancerTumour-agnostic (tissue-agnostic) approval

trials

10
ABC-02BILCAPClarIDHyFIGHT-202FIRST-308FOENIX-CCA2HERIZON-BTC-302KEYNOTE-966NALIRICC (AIO)TOPAZ-1

pairings

1
Gemcitabine-cisplatin + PD-(L)1 blockade in biliary cancer

ideas

8
A non-profit pharmaceutical company for the cancers markets ignoreAn open model bank for the rare tumours nobody has models forAn open organoid bank for cancers too rare to have modelsctDNA-guided switching among FGFR inhibitorsOne global rare cancer network with n-of-1 and Bayesian trial frameworksOne standing umbrella trial for all rare cancers in a countryTest drugs on the patient's own cancer cells when there is no trial to joinTrial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancer

collections

1
Japanese oncology guidelines (JSMO, JSCO and organ societies)

people

4
Do-Youn OhEileen M. O'ReillyTanios Bekaii-SaabTheodore S. Hong

bottlenecks

1
Rare and paediatric cancers without markets