Pemigatinib for previously treated metastatic or unresectable central nervous system tumors with fibroblast growth factor receptor mutations or rearrangements: FIGHT-207 results
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.
Authors
- Amit Mahipal (ORCID: https://orcid.org/0000-0002-1111-8333)
- María Vieito (ORCID: https://orcid.org/0000-0001-7531-343X)
- Jordi Rodón (ORCID: https://orcid.org/0000-0001-6467-3632)
- Marc Matrana (ORCID: https://orcid.org/0000-0002-7301-170X)
- Alice Hervieu
- Lipika Goyal (ORCID: https://orcid.org/0000-0002-9777-6221)
- Iben Spanggaard (ORCID: https://orcid.org/0000-0001-5925-2630)
- Caio Rocha Lima
- Santosh Kesari (ORCID: https://orcid.org/0000-0002-8221-8614)
- Xin Li (ORCID: https://orcid.org/0000-0003-0958-1078)
- Natália Cristina de Oliveira (ORCID: https://orcid.org/0000-0002-0747-9478)
- Maria Luisa Veronese
- Michael Schäffer
Institutions
- Hebron University (PS)
- Mayo Clinic (US)
- The University of Texas MD Anderson Cancer Center (US)
- Harvard University (US)
- Ochsner Medical Center (US)
- Copenhagen University Hospital (DK)
- Rigshospitalet (DK)
- Massachusetts General Hospital (US)
- Centre Georges François Leclerc (FR)
- Saint John's Health Center (US)
- Hôpital de Morges (CH)
- University Hospitals Seidman Cancer Center (US)
- Mayo Clinic in Arizona (US)
- Vall d'Hebron Hospital Universitari (ES)
- Stanford Medicine (US)
- Mayo Clinic in Florida (US)
- Center for Cancer Research (US)
- Wake Forest University (US)
- Incyte (United States) (US)
- Stanford University (US)
Publication Details
- Journal
- The Oncologist
- Published
- 2025-10-16
- DOI
- https://doi.org/10.1093/oncolo/oyaf272
- Primary Topic
- Fibroblast Growth Factor Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Bristol-Myers Squibb
- Eli Lilly and Company
- Pfizer
- AstraZeneca
- Incyte
- Puma Biotechnology
- Symphogen
- Bavarian Nordic
- Genentech