marked.bio

FGFR2-selective inhibitor ยท small molecule

lirafugratinib

1 active trials, targeting FGFR2. 1 active approval for a biomarker on marked.bio.

Also known as RLY-4008.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
lirafugratinib
FDA, 2026
previously treated, unresectable locally advanced or metastatic, FGFR2 fusion or other rearrangement FDA approval 23/09/2026; the FDA announcement does not say whether the approval is accelerated. Source
Lyrfigtu 		FGFR2 fusion or rearrangement cholangiocarcinoma Approved 2026 Not approved

REFOCUS single-arm, 116 without prior FGFR inhibitor
Response 46% (36-55)Lasting 11.8 months

Serious side effects 32%Stopped for side effects 5%Fatal 0.9%

Trial details and side effects
Patients
previously treated FGFR2 fusion/rearrangement cholangiocarcinoma (NCT04526106)
Most common side effects
nail toxicity 89%, hand-foot syndrome 82%, stomatitis 80%, alopecia 66%, dry eye 53%, dry mouth 50%, fatigue 43%, retinal pigment epithelial detachment 38% (116 patients)
Notes
FDA approval summary (2026). FGFR2-selective inhibitor. Warnings: eye toxicity, high phosphate. Side effects: Lyrfigtu US label, section 6 (REFOCUS).
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
FGFR2 fusion or rearrangement 1 2 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of lirafugratinib.

  1. Elevar Therapeutics 1

Trials

1 of 2 trials shown

  1. A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement

    Recruiting Phase 2 Elevar Therapeutics Started 2026Primary completion 2027-09 NCT07359820

    lirafugratinib FDA

    Documented FGFR2 gene fusion or rearrangement per local testing of blood and/or tumor.

    FGFR2 fusion or rearrangementFGFR2 Gene Fusion/Rearrangement, Other Solid Tumors, Adult