FDA Approves First FGFR Kinase Inhibitor for Advanced Bladder Cancer

The FDA granted accelerated approval to erdafitinib (Balversa, Janssen) for the treatment of adults with locally advanced or metastatic urothelial cancer with susceptible fibroblast growth factor receptor (FGFR) alterations that has progressed during or after platinum-containing chemotherapy. Patients should be selected for therapy using an FDA-approved companion diagnostic device.

FGFR alterations are present in approximately one in five patients with recurrent and refractory urothelial cancer. The approval was based on the phase 2, multicenter, open-label, single-arm BLC2001trial (ClinicalTrials.gov Identifier: NCT02365597) evaluating 87 patients who had locally advanced or metastatic urothelial cancer that had progressed during or after at least one prior chemotherapy and had at least one FGFR3 gene mutation or FGFR2 gene fusions, as determined by a clinical trial assay performed at a central laboratory.

The investigators found that erdafitinib yielded an objective response rate of 32.2% (95% CI, 22.4%-42.0%), with a complete response of 2.3% and a partial response of 29.9%.

The FDA simultaneously approved a companion diagnostic for use with erdafitinib, the therascreen FGFR RGQ Reverse-transcription (RT)-polymerase chain reaction (PCR) Kit (Qiagen), the first PCR-based companion diagnostic approved to detect FGFR alterations.