Erdafitinib (pan-FGFR TKI) drives 56% ORR in FGFR-altered pancreatic cancer in RAGNAR

Erdafitinib is an oral pan-FGFR1–4 tyrosine kinase inhibitor (TKI) tested in the Phase 2, histology-agnostic RAGNAR trial (NCT04083976) in patients with advanced/unresectable/metastatic solid tumors and FGFR alterations after ≥1 prior systemic line and exhaustion of standard options. In the Broad Panel Cohort (selected “target” FGFR alterations), ORR varied widely by histology: pancreatic cancer 55.6% (10/18) and salivary gland cancer 100% (5/5) stood out, while NSCLC was 26.1% (6/23), breast cancer 31.3% (5/16), HNSCC 33.3% (5/15), high-grade glioma 10.0% (3/30), and low-grade glioma 28.6% (2/7); activity was minimal in the Exploratory Cohort (nontarget FGFR alterations) with ORR 3.8% (2/53), and in the Pediatric Cohort (n=11) there was 1 response plus 7 durable stable disease. TRAEs were consistent with known erdafitinib toxicities; pediatric-specific signals included epiphysiolysis and limb fracture—important given the growth-plate risk in younger patients (vs later-line metastatic pancreatic SOC chemo regimens that typically deliver modest response rates and short survivals, e.g., ~6-month OS reported for nal-IRI/5-FU/LV after gemcitabine-based therapy).