# Biliary tract cancer

The EU has approved 7 indications of 6 drugs in biliary tract cancer. 5 of them need a biomarker result: FGFR (2), HER2 (1), IDH1/2 (1) and MMR/MSI (1). 6 tumour-agnostic approvals also apply when the tumour carries the alteration.

## Approved indications

### dMMR / MSI-H

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Pembrolizumab](https://oncolabels.com/drugs/pembrolizumab) (Keytruda) | MMR/MSI deficient | Monotherapy | 2nd line, advanced | Disease progression on or following at least one prior therapy |

### FGFR2 fusion

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Futibatinib](https://oncolabels.com/drugs/futibatinib) (Lytgobi) | FGFR2 fusion | Monotherapy | 2nd line, advanced | Cholangiocarcinoma only; Progressed after at least one prior line of systemic therapy |
| [Pemigatinib](https://oncolabels.com/drugs/pemigatinib) (Pemazyre) | FGFR2 fusion | Monotherapy | 2nd line, advanced | Cholangiocarcinoma only; After at least one prior line of systemic therapy |

### HER2

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Zanidatamab](https://oncolabels.com/drugs/zanidatamab) (Ziihera) | HER2-positive (IHC 3+) | Monotherapy | 2nd line, advanced | After at least one prior line of systemic therapy |

### IDH1 mutation

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Ivosidenib](https://oncolabels.com/drugs/ivosidenib) (Tibsovo) | IDH1 R132 | Monotherapy | 2nd line, advanced | Cholangiocarcinoma only; After at least one prior line of systemic therapy |

### No biomarker required

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Durvalumab](https://oncolabels.com/drugs/durvalumab) (Imfinzi) | None | + gemcitabine and cisplatin | 1st line, advanced | - |
| [Pembrolizumab](https://oncolabels.com/drugs/pembrolizumab) (Keytruda) | None | + gemcitabine and cisplatin | 1st line, advanced | - |

## Tumour-agnostic approvals

Approved for any solid tumour that carries the alteration, wherever the tumour started.

### EpCAM

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Catumaxomab](https://oncolabels.com/drugs/catumaxomab) (Korjuny) | EpCAM-positive | Monotherapy | 2nd line | Malignant ascites; not eligible for further systemic anticancer therapy Ambiguous in the label (tumour site, line of therapy): check the SmPC. |

### HER2

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Trastuzumab deruxtecan](https://oncolabels.com/drugs/trastuzumab-deruxtecan) (Enhertu) | HER2-positive (IHC 3+) | Monotherapy | 2nd line, advanced | After prior treatment; No satisfactory treatment options |

### NTRK fusion

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Entrectinib](https://oncolabels.com/drugs/entrectinib) (Rozlytrek) | NTRK fusion | Monotherapy | Any line | No prior NTRK inhibitor; Older than 1 month; disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity; no satisfactory treatment options |
| [Larotrectinib](https://oncolabels.com/drugs/larotrectinib) (Vitrakvi) | NTRK fusion | Monotherapy | Any line | Locally advanced, metastatic or where surgical resection is likely to result in severe morbidity; no satisfactory treatment options |
| [Repotrectinib](https://oncolabels.com/drugs/repotrectinib) (Augtyro) | NTRK fusion | Monotherapy | Advanced | Prior NTRK inhibitor, or no prior NTRK inhibitor and treatment options not targeting NTRK provide limited clinical benefit or have been exhausted; Aged 12 years and older |

### RET fusion

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Selpercatinib](https://oncolabels.com/drugs/selpercatinib) (Retsevmo) | RET fusion | Monotherapy | Advanced | Treatment options not targeting RET provide limited clinical benefit, or have been exhausted; Aged 2 years and older |

## Label wording

- **Pembrolizumab** (Keytruda), Biliary tract cancer, 2nd line, advanced: “unresectable or metastatic gastric, small intestine, or biliary cancer, who have disease progression on or following at least one prior therapy.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/keytruda-epar-product-information_en.pdf)
- **Pembrolizumab** (Keytruda), Biliary tract cancer, 1st line, advanced: “KEYTRUDA, in combination with gemcitabine and cisplatin, is indicated for the first-line treatment of locally advanced unresectable or metastatic biliary tract carcinoma in adults.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/keytruda-epar-product-information_en.pdf)
- **Durvalumab** (Imfinzi), Biliary tract cancer, 1st line, advanced: “IMFINZI in combination with gemcitabine and cisplatin is indicated for the first-line treatment of adults with unresectable or metastatic biliary tract cancer (BTC).” [SmPC](https://www.ema.europa.eu/en/documents/product-information/imfinzi-epar-product-information_en.pdf)
- **Zanidatamab** (Ziihera), Biliary tract cancer, 2nd line, advanced: “Ziihera as monotherapy is indicated for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC3+) biliary tract cancer (BTC) previously treated with at least one prior line of systemic therapy (for biomarker-based patient selection, see section 4.2).” [SmPC](https://www.ema.europa.eu/en/documents/product-information/ziihera-epar-product-information_en.pdf)
- **Pemigatinib** (Pemazyre), Biliary tract cancer, 2nd line, advanced: “Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/pemazyre-epar-product-information_en.pdf)
- **Ivosidenib** (Tibsovo), Biliary tract cancer, 2nd line, advanced: “Tibsovo monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (see section 5.1).” [SmPC](https://www.ema.europa.eu/en/documents/product-information/tibsovo-epar-product-information_en.pdf)
- **Futibatinib** (Lytgobi), Biliary tract cancer, 2nd line, advanced: “Lytgobi monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/lytgobi-epar-product-information_en.pdf)

Source: section 4.1 of each summary of product characteristics (SmPC), EMA data as of 5 Nov 2026. AI models structured these entries and no person has checked them yet. oncolabels is a non-commercial reference for education and research and gives no medical advice; the current SmPC takes precedence.

Data under CC BY 4.0: credit oncolabels and the European Medicines Agency. Page: https://oncolabels.com/tumours/biliary-tract-cancer