Cholangiocarcinoma
Recent clinical, regulatory, research and industry developments relating to this disease.
Tumor immune microenvironment and the current immunotherapy of cholangiocarcinoma (Review).
NCCN Guidelines® Insights: Biliary Tract Cancers, Version 2.2023.
Futibatinib for <i>FGFR2</i>-Rearranged Intrahepatic Cholangiocarcinoma.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 3 clinical trials expected to report results, the earliest in Q4 2027.
- Q4 2027An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma
- Q4 2028HYPERION CCA: a Phase 2 Trial of Systemic Therapy With or Without Liver-directed Radiation Therapy for Patients With Advanced Intrahepatic Cholangiocarcinoma
- Q1 2029Efficacy and Safety of Postoperative Concurrent Chemoradiotherapy for Extrahepatic Cholangiocarcinoma and Gallbladder Carcinoma: A Multicenter Prospective Phase II Study
Clinical MilestonesViewHide
- 2026-06-25Phase II Study of Intravenous and Intraperitoneal Paclitaxel and Oral Nilotinib for Peritoneal Carcinomatosis From Colorectal, Appendiceal, Small Bowel, Gastric, Cholangiocarcinoma, Breast, Ovarian, or Other Gynecologic Primary CancerResults posted
- 2025-10-28Contrast-Enhanced Ultrasound for Diagnosis and Therapy of CholangiocarcinomaResults posted
- 2026-07-31An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic CholangiocarcinomaResults expected Q4 2027
- 2026-06-24Efficacy and Safety of Postoperative Concurrent Chemoradiotherapy for Extrahepatic Cholangiocarcinoma and Gallbladder Carcinoma: A Multicenter Prospective Phase II StudyResults expected Q1 2029
- 2026-03-11HYPERION CCA: a Phase 2 Trial of Systemic Therapy With or Without Liver-directed Radiation Therapy for Patients With Advanced Intrahepatic CholangiocarcinomaResults expected Q4 2028
- 2025-11-03A Multicenter, Open-Label, Phase 2 Trial of E7090 in Subjects With Unresectable Advanced or Metastatic Cholangiocarcinoma With FGFR 2 Gene FusionCompleted
- 2025-10-20Prospective Multicentre Trial of Biliary Drainage Plus Hepatic Arterial Infusion Chemotherapy Versus Biliary Drainage Plus Best Supportive Care in Locally Advanced Perihilar Cholangiocarcinomas.Completed
- 2025-10-01Hepatic Arterial Infusion Chemotherapy (HAIC) Combined With Donafenib and Sintilimab in First-line Treatment of Unresectable Intrahepatic Cholangiocarcinoma (ICC): a Prospective, Open-label, Phase II StudyCompleted
- 2026-07-31ClinicalAn Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic CholangiocarcinomaResults expected Q4 2027
- 2026-06-25ClinicalPhase II Study of Intravenous and Intraperitoneal Paclitaxel and Oral Nilotinib for Peritoneal Carcinomatosis From Colorectal, Appendiceal, Small Bowel, Gastric, Cholangiocarcinoma, Breast, Ovarian, or Other Gynecologic Primary CancerResults posted
- 2026-06-24ClinicalEfficacy and Safety of Postoperative Concurrent Chemoradiotherapy for Extrahepatic Cholangiocarcinoma and Gallbladder Carcinoma: A Multicenter Prospective Phase II StudyResults expected Q1 2029
- 2026-03-11ClinicalHYPERION CCA: a Phase 2 Trial of Systemic Therapy With or Without Liver-directed Radiation Therapy for Patients With Advanced Intrahepatic CholangiocarcinomaResults expected Q4 2028
- 2025-11-03ClinicalA Multicenter, Open-Label, Phase 2 Trial of E7090 in Subjects With Unresectable Advanced or Metastatic Cholangiocarcinoma With FGFR 2 Gene FusionCompleted
- 2025-10-28ClinicalContrast-Enhanced Ultrasound for Diagnosis and Therapy of CholangiocarcinomaResults posted
- 2025-10-20ClinicalProspective Multicentre Trial of Biliary Drainage Plus Hepatic Arterial Infusion Chemotherapy Versus Biliary Drainage Plus Best Supportive Care in Locally Advanced Perihilar Cholangiocarcinomas.Completed
- 2025-10-01ClinicalHepatic Arterial Infusion Chemotherapy (HAIC) Combined With Donafenib and Sintilimab in First-line Treatment of Unresectable Intrahepatic Cholangiocarcinoma (ICC): a Prospective, Open-label, Phase II StudyCompleted
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Lytgobi monotherapy is indicated for the treatment of adult patients with locally advance… (2023)
Approval — Tibsovo in combination with azacitidine is indicated for the treatment of adult patients… (2023)
Approval — Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or me… (2021)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Cholangiocarcinoma7
- Bile Duct Neoplasms5
- Liver Neoplasms3
- Carcinoma, Hepatocellular2
- Antineoplastic Agents1
- Antineoplastic Combined Chemotherapy Protocols1
- Bile Ducts, Intrahepatic1
- Biliary Tract Neoplasms1
Leading journals6
- Cancer control : journal of the Moffitt Cancer Center1
- ESMO open1
- Gut and liver1
- International journal of oncology1
- JAMA oncology1
- Journal of hepatology1
Leading researchers8
- Valle JW4
- Kelley RK3
- Adeva J2
- Alvaro D2
- Andersen JB2
- Banales JM2
- Braconi C2
- Bridgewater JA2
Affiliations (unnormalised)6
- Biotech Research and Innovation Centre (BRIC)2
- Hospital Universitario 12 de Octubre2
- Institute of Cancer Sciences2
- Institute of Pathology2
- Mayo Clinic2
- Mayo Clinic College of Medicine and Science2
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Cholangiocarcinoma is a malignant tumor that arises from the epithelium of the bile ducts. It is a heterogeneous biliary cancer that can develop anywhere along the biliary tree and is often diagnosed at an advanced stage because early presentation is frequently silent.
The literature describes cholangiocarcinoma as a heterogeneous disease arising from a complex interaction between host genetic background and multiple risk factors. The supplied grounding does not support a single definitive cause or a complete aetiologic pathway.
Cholangiocarcinoma shows marked heterogeneity at the genomic, epigenetic, and molecular levels, which contributes to its aggressive behavior and limits treatment efficacy. The tumor microenvironment and chronic inflammatory signaling are implicated in carcinogenesis, including processes such as epithelial-mesenchymal transition, angiogenesis, and metastasis.
Reported risk factors vary geographically and reflect both environmental and genetic determinants. The grounding supports infectious, lifestyle, metabolic, and heritable risk factors, and notes that some are potentially preventable or treatable, such as alcohol and tobacco use and hepatitis infection.
Surgery is the only potentially curative treatment described in the grounding. For advanced disease, chemotherapy has limited effectiveness, and the literature emphasizes increasing use of molecularly informed approaches, including next-generation sequencing to guide targeted therapies in metastatic cancer.
AI-generated summary grounded in MeSH and 6 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
A malignant tumor arising from the epithelium of the BILE DUCTS.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.