Updated results from the phase 2 RAGNAR study found that erdafitinib, an oral selective FGFR tyrosine kinase inhibitor (TKI), demonstrated promising clinical activity among pre-treated patients with non-small cell lung cancer (NSCLC) harboring pre-specified FGFR alterations. 

These data were presented by Martin Schuler, MD, West German Cancer Center, University Hospital Essen, Essen, Germany, at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois.

According to Dr Schuler, “primary analysis results from the RAGNAR study demonstrated tumor agnostic efficacy in patients with advanced solid tumors with predefined FGFR alterations after failure of standard therapies.” These earlier results led to the US FDA approval of erdafitinib for treatment of locally advanced or metastatic urothelial carcinoma among patients with susceptible FGFR3 alterations who have progressed during or after ≥1 line of prior systemic therapy.

In this single-arm cohort, 23 patients with advanced or metastatic squamous (n = 14) or non-squamous (n = 9) NSCLC with pre-specified FGFR fusions (n = 13) or FGFR mutations (n = 10) who experienced disease progression after standard therapy were enrolled to receive oral erdafitinib until disease progression or unacceptable toxicity. The primary end point was objective response rate (ORR). Secondary end points included duration of response (DOR), disease control rate, progression-free survival (PFS), overall survival (OS), and safety. 

At a median follow-up of 23.7 months, ORR was 26% and disease control rate was 74%. Median time to response was 1.5 months. Response was observed in 21% of squamous patients, 33% of non-squamous patients, 29% of FGFR2 patients, 25% of patients with FGFR3 alterations, 20% of patients with FGFR mutations, and 31% with FGFR fusions. Median DOR was 4.6 months, PFS was 4.1 months, and OS was 10.5 months. The most common adverse events included diarrhea (65%), stomatitis (61%), dry mouth (44%), hyperphosphatemia (65%), dry skin (30%), and fatigue (22%). Serious adverse events were experienced by 6 patients and 2 patients discontinued treatment due to an adverse event. No treatment-related deaths were observed. 

Dr Schuler concluded, “erdafitinib demonstrated clinically meaningful activity in this rare lung cancer entity in heavily pretreated patients with non-small cell lung cancer harboring pre-specified FGFR alterations…and responses were observed in fusions and mutations and in squamous and non-squamous histologies.” 

Source: 

Schuler MH, Tabernero J, Carranza O, et al. Efficacy and safety of erdafitinib in adults with NSCLC and prespecified fibroblast growth factor receptor alterations in the phase 2 open-label, single-arm RAGNAR trial. Presented at the ASCO Annual Meeting. May 31-June 4, 2024; Chicago, IL. Abstract #8515