The growing interest in FGFR inhibitors as targeted therapy forFGFR2-fusion positive iCCA is fueled by exceptionally encouraging results fromclinical phase II trials in pre-treated patients, which resulted in the recentUnited States Food and Drug Administration (FDA) approval of pemigatinib for thetreatment of advanced iCCA patients with FGFR2 fusions. Here, FGFR2 is linked to infantile convulsions and choreoathetosis.