Erdafitinib: A novel therapy for FGFR-mutated urothelial cancer

Kiera Roubal, Zin W. Myint, Jill M. Kolesar

Research output: Contribution to journalReview articlepeer-review

36 Scopus citations


Purpose: To provide an overview of fibroblast growth factor receptor (FGFR) gene alterations and the pharmacology, clinical effectiveness, dosage and administration, cost, and place in therapy of erdafitinib in bladder cancer. Summary: Erdafitinib (Balversa, Janssen Pharmaceuticals) is a novel pan-FGFR inhibitor recently approved for the treatment of patients with advanced urothelial cancer with specific FGFR genetic alterations who have received at least one prior platinum-containing regimen. Erdafitinib binding to the FGFR2 and FGFR3 receptors inhibits FGF activity, resulting in cell death. Erdafitinib is available in tablet form, and the current recommended daily dosing is 8 mg, with dose escalation to 9 mg after 14 to 21 days of therapy if tolerated. A phase 2 clinical trial demonstrated that patients who received erdafitinib experienced on average 5.5 months of progression-free survival (95% confidence interval [CI], 4.2-6.0 months). In addition, 40% (95% CI, 31-50%) of patients responded to erdafitinib therapy. Patients receiving erdafitinib therapy should be monitored specifically for elevations in serum phosphate levels and changes in vision. Other adverse effects include anemia, thrombocytopenia, and electrolyte abnormalities. Conclusion: Erdafitinib is the first small-molecule FGFR inhibitor approved for use in advanced bladder cancer.

Original languageEnglish
Pages (from-to)346-351
Number of pages6
JournalAmerican Journal of Health-System Pharmacy
Issue number5
StatePublished - Mar 1 2020

Bibliographical note

Publisher Copyright:
© 2020 American Society of Health-System Pharmacists 2020. All rights reserved.


  • FGFR inhibitor
  • FGFR mutation
  • bladder cancer
  • erdafitinib
  • fibroblast growth factor
  • urothelial carcinoma

ASJC Scopus subject areas

  • Pharmacy
  • Pharmacology
  • Health Policy


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