Elevar Therapeutics announced US FDA approval of Lyrfigtu (lirafugratinib), an oral FGFR2 inhibitor for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. The approval gives patients with FGFR2-driven disease another targeted treatment option in a setting where Pemazyre (pemigatinib) and Lytgobi (futibatinib) are already approved.
The approval was supported by the Phase I/II ReFocus trial (NCT04526106). In 116 FGFR inhibitor-naïve patients with the approved indication, lirafugratinib produced a confirmed objective response rate of 46% and a median duration of response of 11.8 months, according to the FDA. Serious adverse reactions occurred in 32% of patients, while ocular toxicity, including retinal pigment epithelial detachment, is among the key monitored risks associated with treatment.
Lirafugratinib is a selective, irreversible FGFR2 inhibitor that forms a covalent bond within the receptor’s ATP-binding pocket, allowing sustained target engagement while limiting inhibition of FGFR1, FGFR3, and FGFR4. Preclinical studies have also shown activity against several FGFR2 resistance mutations that can emerge during treatment with earlier FGFR inhibitors.
Elevar, a majority-owned subsidiary of South Korea-based HLB Co., Ltd., holds worldwide development and commercialization rights to lirafugratinib under an exclusive agreement with Relay Therapeutics signed in December 2024. The company submitted its NDA in January 2026, and the FDA subsequently granted Priority Review. Elevar has said Lyrfigtu is expected to become available to US patients in Q4 2026.