For patients with advanced cholangiocarcinoma, a phase IIa study demonstrated a partial response of 46.7% and stable disease of 33.3% in patients (n = 17) receiving prior treatment for cholangiocarcinoma exhibiting FGFR alterations (10 FGFR2 fusions, 4 FGFR3 mutations, 1 FGFR3 fusion, and two FGFR3 mutations). This evidence concerns the gene FGFR3 and cholangiocarcinoma.