Erdafitinib for the treatment of metastatic bladder cancer

Expert Rev Clin Pharmacol. 2020 Jan;13(1):1-6. doi: 10.1080/17512433.2020.1702025. Epub 2019 Dec 22.


Introduction: Since the approval of immune checkpoint inhibitors (ICIs), there has been continuing and significant progress in urothelial cancer (UC) treatment. However, only about one fifth of UC patients respond to ICI. Recently, erdafitinib was developed for treating locally advanced or metastatic UC (mUC) with FGFR3 or FGFR2 alterations, accounting for 15-20% of patients. Erdafitinib is the first targeted therapy ever approved for mUC.Areas covered: This review summarizes the preclinical and clinical data on erdafitinib for UC. PubMed search and relevant articles presented at international conferences were used for the literature search.Expert opinion: The FDA approval of erdafitinib provided a new treatment option for FGFR-altered UC progressing on platinum-based chemotherapy. It is not clear whether FGFR inhibitor is a preferred second-line treatment choice to ICI. Compared to ICI, erdafitinib has a better response rate in patients with visceral metastases. However, a shorter duration of response and toxicity profile of erdafitinib, particularly ocular toxicity, is an important consideration. Regular eye exams are recommended by the FDA. Tumor profiling during upfront therapy may help identify those who benefit at the time of progression. In summary, a high unmet need remains for new drugs in chemotherapy- and ICI-refractory UC.

Keywords: Bladder cancer; FGFR; FGFR3-TACC3 translocation; erdafitinib; mutation; urothelial carcinoma.

Publication types

  • Review

MeSH terms

  • Animals
  • Carcinoma, Transitional Cell / drug therapy
  • Carcinoma, Transitional Cell / pathology
  • Humans
  • Neoplasm Metastasis
  • Protein Kinase Inhibitors / administration & dosage*
  • Protein Kinase Inhibitors / adverse effects
  • Protein Kinase Inhibitors / pharmacology
  • Pyrazoles / administration & dosage*
  • Pyrazoles / adverse effects
  • Pyrazoles / pharmacology
  • Quinoxalines / administration & dosage*
  • Quinoxalines / adverse effects
  • Quinoxalines / pharmacology
  • Receptor, Fibroblast Growth Factor, Type 2 / antagonists & inhibitors
  • Receptor, Fibroblast Growth Factor, Type 3 / antagonists & inhibitors
  • Urinary Bladder Neoplasms / drug therapy*
  • Urinary Bladder Neoplasms / pathology


  • Protein Kinase Inhibitors
  • Pyrazoles
  • Quinoxalines
  • erdafitinib
  • FGFR2 protein, human
  • FGFR3 protein, human
  • Receptor, Fibroblast Growth Factor, Type 2
  • Receptor, Fibroblast Growth Factor, Type 3