The FDA has approved Lyrfigtu (lirafugratinib), a second-line treatment from Elevar Therapeutics to treat cholangiocarcinoma (CCA) with FGFR2 fusion or other rearrangement, a type of bile duct cancer.
The potent, selective, oral small molecule inhibitor of FGFR2 targets a receptor tyrosine kinase that is often mutated in certain cancers, including in oral bile duct cancer. The selective targeting of FGFR2 minimizes off-isoform toxicities.
In the phase 1/2 ReFocus trial, patients taking Lyrfigtu saw positive results, including an objective response rate (ORR) of 46% and a median duration of response of 11.8 months. The drug also led to a median progression-free survival of 11.3 months and had a 12-month rate of 49.2%. By using dose adjustments, it had a predictable and manageable safety profile.
Prior to its approval, Lyrfigtu had priority review designation from the FDA. It is now expected to be on the market by the end of the year.