Erdafitinib: A novel therapy for FGFR-mutated urothelial cancer.

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.

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.

Erdafitinib is the first small-molecule FGFR inhibitor approved for use in advanced bladder cancer.

American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists. 2020 Feb 19 [Epub]

Kiera Roubal, Zin W Myint, Jill M Kolesar

School of Pharmacy, University of Wisconsin, Madison, WI., Division of Medical Oncology, Department of Internal Medicine, University of Kentucky, Lexington, KY., University of Kentucky College of Pharmacy, Lexington, KY.