Table 2.
| Therapy | Target | OR (HR) | Prevalence in iCCA | Note | Clinical Phases [49] |
|---|---|---|---|---|---|
| Ramucirumab | VEGFR2 | 0.710 (0·531–0·949) | AFP > 400 ng/mL, 2nd line, REACH-2 | NCT01140347 [76] | |
| Pemigatinib | FGFR2 fusion (EGFR G179A. exon18) | 11–15% | FIGHT-302, Comparator (Gemcitabine + cisplatin) | NCT03656536 [75] | |
| Fisogatinib (BLU-554), Ivosidenib | IDH1 mutation | 5–13% | NCT02989857, NCT02273739 [40,41] | ||
| MET amplification | 2–6% | ||||
| 2–3% | MSI high TMB high | ||||
| FGFR2 V564F, N564 | |||||
| Dabrafenib (BRAFi) + Trametinib (MEFi) | BRAF600E gain of function mutations |
5% (BRAF) | |||
| Larotrectinib | TRK Fusion | TRK fusion–positive cancer | ClinicalTrials.gov numbers, NCT02122913, NCT02637687, and NCT02576431 [74] | ||
| Futibatinib (TAS-120) | irreversible FGFR1–4 inhibitor |
Comparator (Gemcitabine + cisplatin) | III, NCT04093362 [77]; I NCT02052778 | ||
| Infigratinib (BGJ398) | FGFR1–3 | Comparator (Gemcitabine + cisplatin) | III, NCT03773302 | ||
| Derazantinib (ARQ-087) | pan-FGFR | III, NCT03230318 NCT04087876 |