FDA approves lirafugratinib for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma
Regulatory approval: On September 23, 2026, the FDA approved lirafugratinib (Lyrfigtu; Elevar Therapeutics), a kinase inhibitor, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement. Approval was supported by the single-arm REFOCUS trial (NCT04526106; n=116), which reported an ORR of 46% (95% CI, 36–55) and a median DOR of 11.8 months (95% CI, 7.5–13.0).
FDA approval (Sept 23, 2026)
The US Food and Drug Administration (FDA) approved lirafugratinib (Lyrfigtu; Elevar Therapeutics, Inc.), a kinase inhibitor, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement.
Full prescribing information is expected to be posted on Drugs@FDA.
Supporting evidence
Efficacy and safety were evaluated in REFOCUS (NCT04526106), a multicenter, open-label, single-arm trial enrolling 116 patients with unresectable or metastatic cholangiocarcinoma who were FGFR inhibitor–naïve and had received prior chemotherapy or chemoimmunotherapy. Patients received lirafugratinib 70 mg orally once daily until disease progression or unacceptable toxicity.
Primary efficacy endpoints were objective response rate (ORR) and duration of response (DOR) per independent review committee (RECIST v1.1). Reported outcomes:
- ORR: 46% (95% CI, 36–55)
- Median DOR: 11.8 months (95% CI, 7.5–13.0)
Safety and dosing highlights
The label includes warnings/precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity. The recommended dose is 70 mg once daily until progression or unacceptable toxicity.
Regulatory pathway
The review used the RTOR pilot program and Assessment Aid; the application received priority review and had breakthrough therapy and orphan drug designations.