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PMID
Authors Mitesh J. Borad, Peter Lu, Xianzhang Meng, Kristin Ryan, Laura Alexander, Jia Liu, Do-Youn Oh, and Richard D. Kim
Title Efficacy and safety of lirafugratinib in FGFRi-naïve cholangiocarcinoma (CCA) patients harboring FGFR2 fusions/rearrangements (FGFR2 f/r).
Journal Vol Issue Date Pages Journal Short Title
Journal of Clinical Oncology 44 2_suppl January 12, 2026 J Clin Oncol
URL https://ascopubs.org/doi/10.1200/JCO.2026.44.2_suppl.476
Abstract Text Background: Outcomes remain poor for patients with advanced/metastatic CCA treated with first-line gemcitabine-cisplatin +/- anti-PD(L)1. Lirafugratinib is the first highly selective, irreversible FGFR2 inhibitor designed to target oncogenic FGFR2 driver alterations and resistance mutations. ReFocus (NCT04526106) is an open-label, multicenter phase 1/2 study in patients with advanced/metastatic CCA and other solid tumors with FGFRok2 alterations. Methods: Pivotal cohort of patients (n=116) with advanced/metastatic CCA harboring FGFR2 f/r previously treated with ≥1 systemic therapy and FGFRi-naive were treated with oral lirafugratinib 70 mg once daily until disease progression or unacceptable toxicity. The primary endpoint was confirmed ORR per RECIST v1.1 by independent review committee (IRC). Key secondary endpoint was duration of response (DOR); other secondary endpoints: disease control rate (DCR), progression-free survival (PFS), overall survival (OS), safety, and quality of life per EORTC QLC-C30. Results: As of 27SEP2024, the primary efficacy analysis of IRC-assessed data (n=114) and the secondary analysis of investigator-assessed data (n=116) set was done. The primary efficacy analysis excluded 2 patients as not available. Median age 57 years with 61.4% female. IRC-assessed ORR was 47%, and median DOR was 11.8 months (mos) (95% CI, 7.5-13.0), where 76.2% of responses lasted >6 mos. Median PFS was 11.3 mos (95% CI, 9.2, 14.8), with 12-month rate of 49.2%. Median OS was 22.8 mos (95% CI, 17.3-27.2), with 12-month rate of 74.6%. DCR was 96.5%. Common grade ≥3 on-target, off-tumor treatment-related adverse events (TRAEs) included palmar-plantar erythrodysesthesia (32.8%) and stomatitis (12.1%). TRAEs led to dose reductions of 75.9%, dose interruption of 82.8%, and treatment discontinuation of 4.3%. QoL was maintained. Conclusions: Lirafugratinib demonstrated clinically meaningful anti-tumor activity (ORR, DOR, and PFS), manageable and tolerable safety in CCA FGFR2 f/r patients. Clinical trial information: NCT04526106.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR2 rearrange cholangiocarcinoma sensitive Lirafugratinib FDA approved Actionable In a Phase I/II trial (ReFocus) that supported FDA approval, Lyrfigtu (lirafugratinib) treatment was safe and resulted in an objetive response rate of 47% (53/114, 3 complete and 50 partial responses), a median duration of response of 11.8 mo, a meidan progression-free survival of 11.3 mo, and median overall survial of 22.8 mo in FGFR inhibitor-naive cholangiocarcinoma patients harboring FGFR2 fusions or rearrangements (J Clin Oncol 44, 2026 (suppl 2; abstr 476); NCT04526106). detail...
FGFR2 fusion cholangiocarcinoma predicted - sensitive Lirafugratinib FDA approved Actionable In a Phase I/II trial (ReFocus) that supported FDA approval, Lyrfigtu (lirafugratinib) treatment was safe and resulted in an objetive response rate of 47% (53/114, 3 complete and 50 partial responses), a median duration of response of 11.8 mo, a meidan progression-free survival of 11.3 mo, and median overall survial of 22.8 mo in FGFR inhibitor-naive cholangiocarcinoma patients harboring FGFR2 fusions or rearrangements (J Clin Oncol 44, 2026 (suppl 2; abstr 476); NCT04526106). detail...