Relevant targetable aberrations in BTCs

Molecular alterationIncidence (%)Anatomical locationInvestigated agents
FGFR2 gene fusions14–23iCCAPemigatinib
Infigratinib
Futibatinib
Derazantinib
Debio 1347
Erdafitinib
IDH mutations7–20 (IDH1)
3 (IDH2)
iCCAIvosidenib
Enasidenib
Dasatinib
FT-2102
HER2 overexpression/amplification15GBC, eCCATrastuzumab
Trastuzumab-pertuzumab
Trastuzumab deruxtecan
T-DM1
Zanidatamab
HER2 mutations1–2GBC, eCCA, iCCANeratinib
7AVC
BRAF V600E mutation< 5iCCADabrafenib-trametinib
NTRK fusions3.5iCCALarotrectinib
Entrectinib
MSI-H1eCCA, iCCA, GBCPembrolizumab

T-DM1: trastuzumab-emtansine; FT-2102: olutasidenib