Erdafitinib for the treatment of urothelial cancer.

: Fibroblast growth-factor receptor (FGFR) inhibition is a promising strategy of treatment in urothelial cancer (UC). mutations or fusions (mut/fus) are common in luminal-1 UC subtype, which exhibits poor responses to immunotherapy. Erdafitinib is a potent and selective pan-FGFR tyrosine kinase inhibitor. Based on the results of the phase 2 BLC2001 trial (NCT02365597), in which erdafitinib showed an overall response rate of 40% in metastatic UC with mut/fus, it is the first approved targeted therapy in metastatic UC. : This review covers the preclinical and clinical evidence for erdafitinib, summarizes the results of other FGFR inhibitors tested in UC and explores future perspectives of FGFR inhibition in UC. : In the era of precision medicine, erdafitinib approval marks a step forward in UC. Erdafitinib qualifies as a compelling comparator in the salvage therapy setting. Special attention must be paid to typical adverse class-effects of FGFR inhibitors. In the near future, in order to achieve an optimal selection of molecularly-altered tumors, it will be important to assess the performance of different diagnostic tools and to investigate the role of liquid biopsy. Combinations with immunotherapy represent a novel therapeutic opportunity being tested in ongoing trials.