# Pemigatinib for previously treated metastatic or unresectable central nervous system tumors with fibroblast growth factor receptor mutations or rearrangements: FIGHT-207 results

**Authors:** Iben Spanggaard, Marc Matrana, Caio Rocha Lima, Amit Mahipal, Maria Vieito, Alice Hervieu, Lipika Goyal, Jordi Rodón, Maria Luisa Veronese, Natalia Oliveira, Xin Li, Michael Schaffer, Santosh Kesari

PMC · DOI: 10.1093/oncolo/oyaf272 · The Oncologist · 2025-10-16

## TL;DR

This study shows pemigatinib, an FGFR inhibitor, has some effectiveness and manageable safety in treating brain tumors with FGFR gene changes.

## Contribution

The study is the first to evaluate pemigatinib in CNS tumors with FGFR alterations.

## Key findings

- Three out of 13 patients with CNS tumors showed objective responses to pemigatinib.
- Safety profile of pemigatinib was consistent with prior findings in other tumor types.
- Patients with FGFR3-TACC3 fusions were the most common subgroup in the study.

## Abstract

Central nervous system (CNS) tumors often harbor alterations in genes regulating key cellular pathways, including fibroblast growth factor receptor (FGFR) genes. Here, we report the efficacy and safety of treatment with pemigatinib, an oral, potent, selective FGFR1-3 inhibitor, in patients with advanced FGFR-altered CNS tumors. FIGHT-207 was a single-arm, open-label, phase 2 study of pemigatinib in patients with advanced solid tumors harboring FGFR fusions/rearrangements or other mutations. Patients received pemigatinib 13.5 mg once daily until disease progression or unacceptable toxicity. Endpoints included tumor response and safety. Of the 13 patients with CNS tumors in FIGHT-207, 10 had glioblastoma. Fibroblast growth factor receptor alterations were FGFR3-TACC3 fusions (n = 9), FGFR1 K656E mutations (n = 2), FGFR1 N546K mutation (n = 1), and FGFR1-MITF fusion (n = 1). Three patients (23%) displayed objective responses (1 complete, 2 partial). Safety was consistent with the overall FIGHT-207 population. Pemigatinib had antitumor activity and a manageable safety profile in patients with CNS tumors.

## Linked entities

- **Genes:** FGFR (fibroblast growth factor receptor) [NCBI Gene 373310], FGFR1 (fibroblast growth factor receptor 1) [NCBI Gene 2260], FGFR3 (fibroblast growth factor receptor 3) [NCBI Gene 2261], TACC3 (transforming acidic coiled-coil containing protein 3) [NCBI Gene 10460], MITF (melanocyte inducing transcription factor) [NCBI Gene 4286]
- **Chemicals:** pemigatinib (PubChem CID 86705695)
- **Diseases:** glioblastoma (MONDO:0018177)

## Full-text entities

- **Genes:** FGFR1 (fibroblast growth factor receptor 1) [NCBI Gene 2260] {aka BFGFR, CD331, CEK, ECCL, FGFBR, FGFR-1}, MITF (melanocyte inducing transcription factor) [NCBI Gene 4286] {aka CMM8, COMMAD, MI, MITF-A, WS2, WS2A}, FGFR3 (fibroblast growth factor receptor 3) [NCBI Gene 2261] {aka ACH, CD333, CEK2, HSFGFR3EX, JTK4}, TACC3 (transforming acidic coiled-coil containing protein 3) [NCBI Gene 10460] {aka ERIC-1, ERIC1, Tacc4, maskin}
- **Diseases:** CNS tumors (MESH:D016543), solid tumors (MESH:D009369), toxicity (MESH:D064420), glioblastoma (MESH:D005909)
- **Chemicals:** Pemigatinib (MESH:C000705477), FIGHT-207 (-)
- **Species:** Homo sapiens (human, species) [taxon 9606]
- **Mutations:** K656E, N546K

## Full text

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## Figures

2 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12640119/full.md

## References

6 references — full list in the complete paper: https://tomesphere.com/paper/PMC12640119/full.md

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Source: https://tomesphere.com/paper/PMC12640119