Curie Brief
Turn on cookies to sign in
Signing in saves your progress to your Curie account. We can only do that with cookies on — turn them on to continue.
News
The FDA has approved lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable, locally advanced, or metastatic cholangiocarcinoma harboring FGFR2 gene fusions or rearrangements. The oral, once-daily pill showed a 46% objective response rate and a median overall survival of nearly two years in the phase 1/2 ReFocus trial. It joins two other FGFR2-targeted therapies already on the U.S. market.
The FDA has approved lirafugratinib (Lyrfigtu, Elevar Therapeutics) for adults with previously treated, unresectable, locally advanced, or metastatic cholangiocarcinoma (bile duct cancer) harboring FGFR2 gene fusions or other rearrangements. The oral, once-daily drug is a highly selective, irreversible FGFR2 inhibitor — meaning it locks onto its target permanently, which helps it overcome resistance mutations that can develop with earlier FGFR inhibitors.
Approval was based on the phase 1/2 ReFocus trial, which enrolled 116 patients who had received prior chemotherapy or chemoimmunotherapy but had never taken an FGFR inhibitor. Lirafugratinib joins pemigatinib and futibatinib as the third FGFR2-targeted option in the U.S. for this indication, though its selective FGFR2 targeting is designed to reduce off-target side effects common with pan-FGFR inhibitors. The drug is expected to be commercially available in Q4 2026.
By the Numbers
Why it matters: Cholangiocarcinoma is a rare and aggressive cancer with historically limited treatment options. Lirafugratinib's durable responses and next-generation mechanism offer a meaningful new precision medicine option — and reinforce the importance of routine molecular testing at diagnosis to match patients with the therapies most likely to help them.
