FDA approved Elevar Therapeutics’ Lyrfigtu (lirafugratinib) as a second-line treatment for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements. The decision follows results from the multicenter, open-label, single-group REFOCUS trial. In REFOCUS, lirafugratinib achieved a 46% objective response rate, with a median duration of response of 11.8 months, alongside reported progression-free and overall survival outcomes. The FDA label includes warnings aligned with the known FGFR-inhibitor safety profile, including ocular toxicity and hyperphosphatemia. The approval is a meaningful validation for the targeted FGFR2 space in a tumor type where options remain limited for biomarker-selected patients, and it provides a new standard-setting data point for competitors developing next-generation FGFR inhibitors.