The FDA approved Elevar Therapeutics’ Lyrfigtu (lirafugratinib), positioning the oral FGFR2 inhibitor as a second-line option for patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or other rearrangements. The approval follows results from the REFOCUS multicenter, open-label, single-group trial. In REFOCUS, lirafugratinib delivered an objective response rate of 46% with a median duration of response of 11.8 months, with additional survival and progression-free survival metrics reported in conference presentations earlier this year. The label includes class-typical warnings including ocular toxicity, hyperphosphatemia and embryo-fetal toxicity. For the biotech sector, the approval underscores continued regulatory momentum for targeted small molecules in biomarker-defined biliary tract cancers. It also adds pressure on competing FGFR2-pathway programs as clinicians look for effective sequencing options after first-line chemotherapy or chemoimmunotherapy.