FIGHT-202

NCT02924376 📎

Regimen

Experimental
Pemigatinib 13.5 mg PO QD (2 weeks on, 1 week off, 21-day cycle) until PD or intolerable toxicity
Control
single-arm / no comparator (historical 2L BTC benchmark ORR ~5-10% with chemotherapy)

Population

Previously treated unresectable or metastatic CCA; Cohort A (n=107) FGFR2 fusion or rearrangement; Cohort B (n=20) other FGFR alterations; Cohort C (n=18) no FGFR alterations. ICC-dominant (>95% of enrolled patients, consistent with FGFR2 fusion biology exclusive to ICC). Prior chemo median 1 line (range 1-6). FGFR2 status by local next-generation sequencing or FISH.

Key finding

First FDA-approved targeted therapy for any CCA molecular subset (approved April 2020, accelerated approval). Established FGFR2 fusions as a therapeutically actionable biomarker in ICC. Cohort A ORR 35.5% against a single-digit historical chemo benchmark validated the molecular-matching principle. Cohort B (non-fusion FGFR alterations, n=20) showed 0% ORR, confirming that fusion/rearrangement specificity is required. Major toxicities: hyperphosphatemia (94%), alopecia, dysgeusia — all on-target FGFR inhibition effects.

Source: PMID 32203698

Timeline

    Guideline citations

    • NCCN BTC (p.76)