Erdafitinib in Patients With Advanced Solid Tumors With FGFR Alterations (RAGNAR): Results of Tumor-Specific Analyses and Secondary Cohorts
Year: 2026
Type: article
Abstract: PURPOSE 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. METHODS 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. RESULTS 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. CONCLUSION 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.
Source: JCO Precision Oncology
DOI: 10.1200/po-25-01094
Authors: Omar Carranza, Shubham Pant, Martin Schuler, Olaf Witt, Birgit Geoerger, Yohann Loriot, Sameer Farouk Sait, Dirk Arnold, Graziela Z. Dal Molin, Iwona Lugowska, Helen Winter, Capucine Baldini, Marcelo Corassa, Shibu Thomas, Lauren Crow, Yichuan Xia, Spyros Triantos, Hussein Sweiti, David A. Reardon
Institutions: Hospital Privado de Comunidad, The University of Texas MD Anderson Cancer Center, Essen University Hospital, University of Duisburg-Essen, German Cancer Research Center, University Hospital Heidelberg, National Center for Tumor Diseases, Hopp Children's Cancer Center Heidelberg, Inserm, Université Paris-Saclay, Institut Gustave Roussy, Memorial Sloan Kettering Cancer Center, Asklepios Klinik Altona, Asklepios, Asklepios Kliniken Hamburg, Beneficência Portuguesa de São Paulo, The Maria Sklodowska-Curie National Research Institute of Oncology, Fondation Gustave Roussy, AC Camargo Hospital, Johnson & Johnson (United States), Harvard University, Center for Neuro-Oncology, Dana-Farber Cancer Institute, Dana-Farber/Harvard Cancer Center
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
Funder: National Cancer Institute
Award: Cancer Center Support Grant
OpenAlex ID: W7168359282