Elevar Therapeutics has asked the FDA to approve lirafugratinib (RLY-4008) as a second-line treatment for cholangiocarcinoma, a rare bile duct cancer, in patients with FGFR2 gene changes. The request is based on the ReFocus trial, which included 116 patients who had already had chemotherapy but not another FGFR-targeted drug. The drug showed a 46.5% overall response rate, a 96.5% disease control rate, and responses lasting nearly a year on average. Median overall survival was about 23 months.
Lirafugratinib is highly selective for FGFR2, which helps avoid side effects linked to other FGFR drugs and may work even when resistance mutations appear. Common severe side effects included hand-foot syndrome, nail issues, and mouth sores, but most patients managed these with dose adjustments, and very few stopped treatment entirely.
If approved for cholangiocarcinoma, the company plans to seek FDA approval to use lirafugratinib in other cancers with the same FGFR2 alterations, moving toward more precise, targeted therapies.