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News
A new targeted therapy just got the green light for a tough-to-treat cancer. The FDA approved lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or rearrangement. The drug's selective FGFR2 targeting sets it apart from existing pan-FGFR inhibitors, with a 46% objective response rate seen in clinical trials.
A new option for bile duct cancer patients
The FDA has approved lirafugratinib (Lyrfigtu, Elevar Therapeutics), a selective FGFR2 inhibitor, for adults with previously treated unresectable, locally advanced, or metastatic cholangiocarcinoma carrying an FGFR2 gene fusion or rearrangement. It joins two existing FGFR2-targeted therapies — pemigatinib and futibatinib — on the US market, though lirafugratinib's high selectivity for FGFR2 (versus broader pan-FGFR inhibition) may translate to a more favorable side effect profile.
Approval was based on the phase 1/2 ReFocus trial in 116 patients who had received prior chemotherapy or chemoimmunotherapy but no prior FGFR inhibitor. The drug is taken as a 70 mg oral dose once daily and is expected to launch in Q4 2026.
By the Numbers
Why it matters: Cholangiocarcinoma is aggressive and difficult to treat, with limited second-line options. Lirafugratinib's selective FGFR2 targeting offers clinicians a new tool with potentially fewer off-target side effects, expanding the treatment landscape for this rare but deadly cancer.
