Disease Briefing

Cholangiocarcinoma: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-12Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 192 studies · 8,116 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in cholangiocarcinoma — FGFR2, IDH1, KRAS, TP53, ERBB2, BRAF, BAP1, ARID1A, CDKN2A, PBRM1, SMAD4, CD274 — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

7
drugs carry an FDA label naming cholangiocarcinoma: Durvalumab, Futibatinib, Ivosidenib, Pembrolizumab, Pemigatinib, Zanidatamab and 1 more. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
4
of the 12 genes above carry a drug that is approved in cholangiocarcinoma itself — CD274, ERBB2, FGFR2, IDH1. Across all of them 126 drug entries reach these genes, 118 distinct once salt forms are merged
2
registered PD-1 cell-therapy trials in cholangiocarcinoma, 0 active. Counted from ClinicalTrials.gov across 3 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
6
targets carry an Open Targets tractability signal and have no clinical programme of any kind: KRAS, TP53, BAP1, ARID1A, PBRM1, SMAD4. ERBB2 has cell-therapy trials, so it is undrugged rather than untouched
406
human GEO series match the disease; 192 survive on-topic filtering, and only 9 are patient cohorts of 100+ samples
192
Europe PMC full-text papers name GSE107943 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$7.4M
NIH obligations in FY2025, up 92% since 2013 — while distinct core projects went 13 to 17. Both more projects (+31%) and larger awards, the latter carrying more of the growth; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-12 · weekly

12 genes recurrently implicated in cholangiocarcinoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 7 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Cholangiocarcinoma does have labelled therapy — 7 drugs, listed in the next section.

Two different questions

The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what cholangiocarcinoma is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell therapy here
FGFR2 across cancers → 26 10 approvedE-7090, Erdafitinib, Futibatinib, Infigratinib, Nintedanib, Nintedanib Esylate, Palifermin, Pemigatinib, Regorafenib, TraferminApproved in cholangiocarcinoma: Futibatinib, Pemigatinib.13 active of 38 cholangiocarcinoma trials antibody, other clinical modality, protein degrader, small molecule
IDH1 across cancers → 7 3 approvedIvosidenib, Olutasidenib, VorasidenibApproved in cholangiocarcinoma: Ivosidenib.7 active of 12 cholangiocarcinoma trials antibody, protein degrader, small molecule
KRAS across cancers → 3 2 approvedAdagrasib, SotorasibApproved in non-small cell lung carcinoma. No cholangiocarcinoma indication appears on these drugs’ labels.No cholangiocarcinoma trial of any of these drugs antibody, protein degrader, small molecule
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo cholangiocarcinoma trial of any of these drugs other clinical modality, protein degrader, small molecule
ERBB2 across cancers → 45 17 approvedAfatinib, Afatinib Dimaleate, Dacomitinib, Lapatinib, Lapatinib Ditosylate, Margetuximab, Masoprocol, Neratinib, Pertuzumab, Trastuzumab, Trastuzumab Deruxtecan, Trastuzumab Duocarmazine, Trastuzumab Emtansine, Tucatinib, Vandetanib, Zanidatamab, ZenocutuzumabApproved in cholangiocarcinoma: Zanidatamab, Zenocutuzumab.10 active of 33 cholangiocarcinoma trials antibody, other clinical modality, protein degrader, small molecule 1 active of 1 trial
BRAF across cancers → 16 6 approvedDabrafenib, Encorafenib, Regorafenib, Sorafenib, Tovorafenib, VemurafenibApproved in metastatic melanoma, low grade glioma, colorectal neoplasm and 4 other indications. No cholangiocarcinoma indication appears on these drugs’ labels.2 active of 13 cholangiocarcinoma trials antibody, protein degrader, small molecule
BAP1 across cancers → 0 No drug protein degrader
ARID1A across cancers → 0 No drug protein degrader
CDKN2A across cancers → 0 No drug
PBRM1 across cancers → 0 No drug protein degrader, small molecule
SMAD4 across cancers → 0 No drug protein degrader, small molecule
CD274 across cancers → 13 5 approvedAtezolizumab, Avelumab, Cosibelimab, Durvalumab, SugemalimabApproved in cholangiocarcinoma: Durvalumab.50 active of 74 cholangiocarcinoma trials antibody, other clinical modality, protein degrader, small molecule

Dataset evidence counts studies in the 192-study ranked set whose title or abstract names the gene; the bar is scaled to KRAS. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-12. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

What is actually approved here

openFDA drug labels · weekly

7 drugs carry an FDA label naming cholangiocarcinoma: Durvalumab, Futibatinib, Ivosidenib, Pembrolizumab, Pemigatinib, Zanidatamab, Zenocutuzumab. Separately, 36 of the drugs returned for the genes in the table above are approved only for other diseases and reach cholangiocarcinoma through trials, not through their labels.

7Labelled for cholangiocarcinomaFDA INDICATIONS AND USAGE names the disease
36Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
0Active PD-1 cell-therapy trialsof 2 registered

Every label that names cholangiocarcinoma

DrugRoleWhat the label says
DurvalumabIMFINZILabelled hereBiliary Tract Cancers IMFINZI, in combination with gemcitabine and cisplatin, is indicated for the treatment of adult patients with locally advanced or metastatic biliary tract cancer (BTC).
FutibatinibLYTGOBILabelled hereLYTGOBI is a kinase inhibitor indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.
IvosidenibTIBSOVOLabelled hereLocally Advanced or Metastatic Cholangiocarcinoma For the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated
PembrolizumabKEYTRUDA, KEYTRUDA QLEXLabelled hereBiliary Tract Cancer (BTC) in combination with gemcitabine and cisplatin, for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer.
PemigatinibPEMAZYRELabelled hereCholangiocarcinoma PEMAZYRE is indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test [see Dosage and Administration
ZanidatamabZIIHERALabelled hereBiliary Tract Cancer (BTC) • for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-authorized test.*
ZenocutuzumabBIZENGRILabelled hereAdults with advanced, unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 ( NRG1 ) gene fusion with disease progression on or after prior systemic therapy.
Indocyanine Green And WaterIndocyanine GreenNot a therapyVisual assessment of the major extrahepatic bile duct For visual assessment of the major extrahepatic bile ducts with OLYMPUS infrared imaging endoscopic imaging system.

6 labels match indications_and_usage:"cholangiocarcinoma" OR indications_and_usage:"biliary tract cancer" OR indications_and_usage:"bile duct"; they collapse to 8 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Diagnostic and contrast agents (Indocyanine Green And Water) are listed but are not treatments. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-12. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against PD-1

ClinicalTrials.gov · retrieved 2026-09-12 · weekly

PD-1 is the busiest cell-therapy antigen in cholangiocarcinoma: 2 registered trials, 0 still active. It has no gene entry of its own and is reached through PDCD1: a form or product of that gene.

TrialPhaseStatusTitleLast update
NCT027573911TerminatedCD8+ T Cell Therapy and Pembrolizumab in Treating Patients With Metastatic Gastrointestinal Tumors2020-10-30
NCT039378951/2CompletedAllogeneic NK Cell ("SMT-NK") in Combination With Pembrolizumab in Advanced Biliary Tract Cancer2022-03-29

2 of 2 shown, most recently active first. Other antigens searched: HER2 (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-12. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the cholangiocarcinoma literature, not a count, because the field itself grew: 2015–2018 (n=1,977) against 2021–2025 (n=3,717). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Tumor Microenvironment1.11%7.1%6.38×264 papers
Immunotherapy0.66%2.45%3.72×91 papers
B7-H1 Antigen0.46%1.67%3.66×62 papers
Antibodies, Monoclonal, Humanized0.3%1.1%3.63×41 papers
Precision Medicine0.35%1.26%3.57×47 papers
Programmed Cell Death 1 Receptor0.25%0.89%3.51×33 papers
CD8-Positive T-Lymphocytes0.3%1.05%3.46×39 papers
Lymphocytes, Tumor-Infiltrating0.3%1.02%3.37×38 papers
Pyrimidines0.61%1.96%3.24×73 papers
Phosphatidylinositol 3-Kinases0.51%1.61%3.19×60 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Time Factors6.27%0.91%0.15×34 papers
Survival Analysis5.36%0.83%0.16×31 papers
Proportional Hazards Models5.67%1.05%0.19×39 papers
Immunohistochemistry5.87%1.13%0.19×42 papers
Case-Control Studies2.83%0.54%0.19×20 papers
Japan1.72%0.35%0.2×13 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Bile Duct Neoplasms87.25%94.78%1.09×3523 papers
Bile Ducts, Intrahepatic28.78%48.91%1.7×1818 papers
Prognosis23.22%22.73%0.98×845 papers
Hepatectomy16.74%14.61%0.87×543 papers
Klatskin Tumor14.21%13.29%0.94×494 papers
Cell Proliferation12.44%11.54%0.93×429 papers
Treatment Outcome16.34%9.82%0.6×365 papers
Gene Expression Regulation, Neoplastic10.07%8.66%0.86×322 papers
Biomarkers, Tumor12.7%7.94%0.63×295 papers
Tumor Microenvironment1.11%7.1%6.38×264 papers

Publication mix

Type2015–182021–25
Clinical Trial0.1%0.0%
Randomized Controlled Trial0.6%0.6%
Review11.2%9.8%
Meta-Analysis2.3%1.9%
Case Reports0.0%2.9%

Query: Cholangiocarcinoma[MeSH Major Topic] NOT ("Carcinoma, Hepatocellular"[MeSH] OR "Gallbladder Neoplasms"[MeSH] OR "Pancreatic Neoplasms"[MeSH] OR "Common Bile Duct Neoplasms"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-12.

Disease burden

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

MeasureValueMatchWhat it counts
New cases each year8,000 cases/yeardirect
New cases each year42,340 cases/yearproxycounts liver and intrahepatic bile duct cancer, which is broader than this disease; 2026
Deaths each year30,980 deaths/yearproxycounts liver and intrahepatic bile duct cancer, which is broader than this disease; 2026
Incidence rate9.5 cases per 100,000 per yearproxycounts liver and intrahepatic bile duct cancer, which is broader than this disease; 2019-2023
Death rate6.6 deaths per 100,000 per yearproxycounts liver and intrahepatic bile duct cancer, which is broader than this disease; 2020-2024
People living with it116,514 people living with the diseaseproxycounts liver and intrahepatic bile duct cancer, which is broader than this disease; 2023
Deaths each yearnot publishedNot published for the disease alone; the liver and intrahepatic bile duct figure is mostly hepatocellular carcinoma.
Five-year relative survivalnot publishedThe American Cancer Society publishes five-year relative survival by stage and by site (intrahepatic, extrahepatic), not one figure; SEER has no page. An average across them would be a derivation the sources do not make.
Median age at diagnosisnot publishedThe American Cancer Society gives "in the 70s"; no median is published.

Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis is not recorded for this disease.

Funding

NIH RePORTER · quarterly

NIH obligations naming cholangiocarcinoma, after removing the 513 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

$7.4MNIH obligations, FY2025from $3.9M in FY2013 · +92%
17distinct projects funded13 in FY2013
$7.4Mpeak year was FY2025obligations, all institutes
90%of FY2025 awards from NCI18 of 20

NIH obligations by fiscal year

$2M$4M$6M$7M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$3.9M141315
FY2014$2.6M11919
FY2015$2.9M111021
FY2016$3.1M121124
FY2017$2.2M121123
FY2018$4.5M131236
FY2019$4.5M181348
FY2020$4.8M141340
FY2021$5.3M131254
FY2022$4.8M151461
FY2023$5.6M151460
FY2024$5.9M161554
FY2025$7.4M201758

Where FY2025 money went

InstitutionObligationsAwards
Mayo Clinic Rochester$3.0M9
University Of Pittsburgh At Pittsburgh$0.8M2
Sanford Burnham Prebys Medical Discovery Institute$0.7M1
Medical University Of South Carolina$0.7M1
Massachusetts General Hospital$0.7M1
Icahn School Of Medicine At Mount Sinai$0.6M1
Washington University$0.5M1
Fred Hutchinson Cancer Center$0.4M1
University Of Pennsylvania$0.0M1
Vanderbilt University$0.0M1

Text search cholangiocarcinoma over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-12.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

NIH institutes

NCI18 · 90%
NIDDK1 · 5%
VA1 · 5%

Projects by administering institute. The rows above are the top 3 and account for 20 of 20.

Award mechanisms

R019 · 45%
P504 · 20%
F312 · 10%
R031 · 5%
U011 · 5%
U2C1 · 5%

R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 8 and account for 20 of 20.

Where it lands

Mayo Clinic Rochester$3.0M · 39.9%
University Of Pittsburgh At Pittsburgh$0.8M · 11.3%
Sanford Burnham Prebys Medical Discovery$0.7M · 9.4%
Medical University Of South Carolina$0.7M · 9.0%
Massachusetts General Hospital$0.7M · 8.8%
Icahn School Of Medicine At Mount Sinai$0.6M · 8.2%

Share of $7.4M in FY2025. The top three hold 61%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

406 human GEO series match cholangiocarcinoma. Keyword relevance cannot tell a 1,403-sample patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Composition of the 192-study ranked set

unspecified 75patient 53cell line 39mixed 19xenograft 6
75 unspecified53 patient39 cell line19 mixed6 xenograft75 carry clinical annotation60 carry survival9 patient cohorts ≥100 GEO samples8,116 GEO samples totalin 5 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE26566Molecular classification of human cholangiocarcinoma2012 · array16917511.7
patient cohortAPT 0.95survivalstagemolecularBRAFHER2KRAS475 cites
GSE76311Human hepatocellular carcinoma (HCC) and Cholangiocarcinoma (CCA) from Thailand Initiative in Genomics and Expression Research for Liver Cancer (TIGER-LC)2017 · array6081759.9
APT 0.95351 cites
GSE33327Integrative Molecular Analysis of Intrahepatic Cholangiocarcinoma Reveals 2 Classes That Have Different Outcomes2013 · array2836312.0
patient cohortAPT 0.95survivalmolecularBRAFKRAS456 cites
GSE132305Molecular classification and therapeutic targets in extrahepatic cholangiocarcinoma2020 · array2204911.2
patient cohortAPT 0.95271 cites
GSE89749Integrative genomic and epigenetic analysis in cholangiocarcinoma2017 · array1207624.3
APT 0.95777 cites
GSE59856MicroRNA markers for the diagnosis of pancreatic and biliary tract cancers2015 · array571463.9
patient cohortAPT 0.75113 cites
GSE107943Integrative molecular and clinical analysis of intrahepatic cholangiocarcinoma reveals two prognostic subclassees2018 · sequencing571922.2
APT 0.5survival64 cites
GSE49656Distinct Mutational Patterns of Infection and Non-Infection-Related Bile Duct Cancers Revealed by Exome Sequencing2013 · methylation401510.6
APT 0.95molecularARID1ABAP1IDH1419 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE179443Molecular and radiopathologic spectrum between HCC and intrahepatic cholangiocarcinoma2022 · sequencing13743.9
patient cohortAPT 0.5survivalmolecularKRAS39 cites
GSE244807Self-supervised learning for predicting transcriptomic groups on whole slides images in intrahepatic cholangiocarcinoma2023 · sequencing246110.8
patient cohortAPT 0.5survival7 cites
GSE255058Clinical and biomarker analyses of hepatic arterial infusion chemotherapy plus lenvatinib and PD-1 inhibitor for patients with advanced intrahepatic cholangiocarcinoma2024 · sequencing1822.5
patient cohortAPT 0.95survivalmolecularPD-118 cites
GSE201241Integrative Analysis Defines Distinct Prognostic Subgroups of Intrahepatic Cholangiocarcinoma2022 · methylation64123.1
APT 0.75survival88 cites
GSE201425Single-cell atlas of diverse immune populations in the advanced biliary tract cancer microenvironment2022 · single-cell32141.2
cell lineAPT 0.524 cites
GSE210067Circulating monocytes associated with anti-PD-1 resistance in human biliary cancer induce T cell paralysis2022 · sequencing2732.2
APT 0.75molecularPD-141 cites

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. SubSeries are collapsed to one row per study by linked PMID. Of 406 series retrieved, 45 were dropped by the profile’s exclusion rules and 117 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Where the gaps are

Derived from the sections above · recomputed on every refresh

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

KRAS

2 approved drugs (Adagrasib, Sotorasib) and no registered trial in cholangiocarcinoma. The molecules exist; nobody has tested them here.

BAP1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

ARID1A

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

CDKN2A

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

PBRM1

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

SMAD4

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

How a machine reads this page

Static

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

Fully server-rendered

Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.

Every number carries a source

Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-12), ClinicalTrials.gov (2026-09-12), openFDA (2026-09-12), NCBI GEO (2026-09-12), PubMed (2026-09-12), NIH RePORTER (2026-09-12).

Negatives stated explicitly

Where nothing exists for cholangiocarcinoma — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated negative is the finding.

Denominators, not just percentages

Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.

The filters are published

5 exclusion patterns are applied to free text before anything is ranked, because RBE is used as a model system in none of consequence for the line; "RBE" the term is radiobiology. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Cholangiocarcinoma",
  "mesh": "Cholangiocarcinoma",
  "facts": "https://usebiotransfer.org/disease/cholangiocarcinoma.json",
  "methods": "https://usebiotransfer.org/methods/",
  "all_diseases": "https://usebiotransfer.org/disease/api.json",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}