At the moment, early phase clinical trials are conducted with FGFR3‐directed targeted therapies on patients with FGFR3‐aberrated glioblastoma multiforme, transitional cell carcinoma, multiple myeloma, and other advanced solid malignancies (ClinicalTrials.gov Identifier: NCT01975701, NCT02278978, NCT02401542, NCT02052778). The gene discussed is FGFR3; the disease is transitional cell carcinoma.