Erdafitinib in FGFR-Altered Advanced Urothelial Carcinoma


Key Points

  • The objective response rate was 40%.
  • Median duration of response was 5.6 months.

As reported in The New England Journal of Medicine by Loriot et al, the fibroblast growth factor receptor 1-4 tyrosine kinase inhibitor erdafitinib showed activity in FGFR-altered advanced urothelial carcinoma.

Study Details

The study enrolled patients from sites in 14 countries between May 2015 and March 2018. Enrolled patients had locally advanced nonresectable or metastatic disease with at least one FGFR3 mutation or FGFR2/3 fusion and a history of disease progression during or after at least one course of chemotherapy or within 12 months after neoadjuvant or adjuvant chemotherapy. Prior immunotherapy was permitted. 

The current report provides data on 99 patients who received continuous oral erdafitinib at a starting dose of 8 mg/day (selected-regimen group) after a randomized dose-selection phase.  The primary endpoint was objective response rate.  

Response Rates

Median follow-up was 11.2 months. Confirmed response was observed in 40 patients (40%, with complete response observed in 3%). Among 22 patients who had received prior immunotherapy, response was achieved in 13 (59%). Median duration of response was 5.6 months. Median progression-free survival was 5.5 months, with a 12-month rate of 19%. Median overall survival was 13.8 months, with a 12-month rate of 55%.

Adverse Events

Adverse events of grade ≥ 3 were observed in 67% of patients (considered treatment-related in 46%), with the most common being hyponatremia (11%), stomatitis (10%), and asthenia (7%). Serious adverse events occurred in 39% of patients. Adverse events led to treatment discontinuation in 13 patients (13%), with causes including detachment of the retinal pigment epithelium, hand-foot syndrome, dry mouth, and skin or nail events. No treatment-related deaths were reported.

The investigators concluded, “The use of erdafitinib was associated with an objective tumor response in 40% of previously treated patients who had locally advanced and unresectable or metastatic urothelial carcinoma with FGFR alterations. Treatment-related grade 3 or higher adverse events were reported in nearly half the patients.”

Disclosure: The study was funded by Janssen Research and Development. For full disclosures of the study authors, visit


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