Journal Article

DKFZ-2026-01808

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Erdafitinib in Patients With Advanced Solid Tumors With FGFR Alterations (RAGNAR): Results of Tumor-Specific Analyses and Secondary Cohorts.

Carranza, O. ; Pant, S. ; Schuler, M. ; Witt, O.DKFZ* ; Geoerger, B. ; Loriot, Y. ; Farouk Sait, S. ; Arnold, D. ; Dal Molin, G. Z. ; Lugowska, I. ; Winter, H. ; Baldini, C. ; Corassa, M. ; Thomas, S. ; Crow, L. ; Xia, Y. ; Triantos, S. ; Sweiti, H. ; Reardon, D. A.

2026
American Society of Clinical Oncology Alexandria, VA

Abstract: Erdafitinib, a pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor, is approved for second-line treatment of locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 alterations. Primary histology-agnostic analysis from the phase II RAGNAR study (ClinicalTrials.gov identifier: NCT04083976) indicated a clinical benefit with erdafitinib in Broad Panel Cohort (BPC) patients. We present tumor-specific updated efficacy and safety results from the BPC and tumor-agnostic findings from the Exploratory and Pediatric Cohorts of RAGNAR.The Broad Panel, Exploratory, and Pediatric Cohorts enrolled patients with target (selected) FGFR alterations, nontarget FGFR alterations, and any FGFR alterations, respectively. Adults and pediatric patients (6 years and older) with advanced, unresectable, or metastatic disease who progressed after ≥1 prior line of systemic therapy and exhausted standard therapies were eligible. Erdafitinib 3-8 mg once daily (with possible uptitration) was administered orally until disease progression or intolerable toxicity.Objective response rates (ORRs) in the BPC were 10.0% (3 of 30) for high-grade glioma, 26.1% (6 of 23) for non-small cell lung cancer, 28.6% for (2 of 7) low-grade glioma, 31.3% (5 of 16) for breast cancer, 33.3% (5 of 15) for head and neck squamous cell carcinoma, 55.6% (10 of 18) for pancreatic cancer, and 100% (5 of 5) for salivary gland cancer. The ORR was 3.8% (2 of 53) in the Exploratory Cohort. In the Pediatric Cohort (n = 11), there were one responder and seven patients with durable stable disease. Treatment-related adverse events (TRAEs) were consistent with the known safety profile of erdafitinib. Pediatric-specific TRAEs included epiphysiolysis and limb fracture.Encouraging activity was observed for erdafitinib across tumor types in patients with advanced malignancies and selected FGFR alterations who have exhausted treatment options. More limited activity was observed for erdafitnib in the Exploratory Cohort.

Keyword(s): Humans (MeSH) ; Pyrazoles: therapeutic use (MeSH) ; Pyrazoles: adverse effects (MeSH) ; Female (MeSH) ; Neoplasms: drug therapy (MeSH) ; Neoplasms: genetics (MeSH) ; Neoplasms: pathology (MeSH) ; Quinoxalines: therapeutic use (MeSH) ; Quinoxalines: adverse effects (MeSH) ; Middle Aged (MeSH) ; Male (MeSH) ; Aged (MeSH) ; Adult (MeSH) ; Child (MeSH) ; Cohort Studies (MeSH) ; Receptors, Fibroblast Growth Factor: genetics (MeSH) ; Receptors, Fibroblast Growth Factor: antagonists & inhibitors (MeSH) ; Protein Kinase Inhibitors: therapeutic use (MeSH) ; Adolescent (MeSH) ; erdafitinib ; Pyrazoles ; Quinoxalines ; Receptors, Fibroblast Growth Factor ; Protein Kinase Inhibitors

Note: #NCTZFB26#

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Contributing Institute(s):

1. KKE Pädiatrische Onkologie (B310)
2. Koordinierungsstelle NCT Heidelberg (HD02)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2026

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Database coverage:
; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; Ebsco Academic Search ; Essential Science Indicators ; IF < 5 ; JCR ; PubMed Central ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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