A phase I/II study (NCT01752920), including patients (n = 29) with cholangiocarcinoma with FGFR2 fusions, demonstrated an ORR of 20.7%, DCR of 82.8%, and a mPFS of 5.7 months (95% CI, 4.0–9.2) [49]. The gene discussed is FGFR2; the disease is cholangiocarcinoma.