The FDA has approved Elevar Therapeutics’ Lyrfigtu, or lirafugratinib, for patients with cholangiocarcinoma whose tumors have an FGFR2 fusion or other rearrangement.
Cholangiocarcinoma is a rare bile duct cancer. Elevar described Lyrfigtu as a second-line treatment option and said it expects the medicine to be available in the United States by the fourth quarter of 2026.
The approval follows results from the Phase 1/2 ReFocus trial. Among patients with the relevant FGFR2 alteration, the confirmed objective response rate was 46%, and median duration of response was 11.8 months, Elevar said. Median progression-free survival was 11.3 months.
Lyrfigtu is an oral drug designed to inhibit FGFR2, a protein that can be altered in certain cancers. Elevar is also evaluating it in other previously treated advanced or metastatic solid tumors with FGFR2 fusions or rearrangements.
The treatment carries warnings for eye toxicity, elevated blood phosphate levels and potential harm during pregnancy. In safety data covering 385 patients, retinal pigment epithelial detachment occurred in 31%, while elevated phosphate levels occurred in 21%. Serious adverse reactions occurred in 32% of patients, and one fatal hemorrhage was reported.
Patients receiving Lyrfigtu require eye examinations before treatment and at intervals during treatment.
Elevar is majority-owned by HLB and holds an exclusive worldwide commercialization license for lirafugratinib from Relay Therapeutics.
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