FDA Approval Summary: Erdafitinib for FGFR3-Altered Locally Advanced or Metastatic Urothelial Carcinoma.
William F Maguire, Elaine Chang, Flora Mulkey et al.
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In 30 seconds
This FDA approval summary describes evidence supporting erdafitinib for patients with FGFR3-altered locally advanced or metastatic urothelial carcinoma that progressed during or after at least one systemic therapy. The evidence came from Cohort 1 of BLC3001 (THOR), an open-label randomized trial of 266 patients comparing erdafitinib with docetaxel or vinflunine. Erdafitinib improved overall survival, progression-free survival, and objective response rate versus chemotherapy. Median overall survival was 12.1 months with erdafitinib and 7.8 months with chemotherapy (HR, 0.64; 95% CI, 0.47-0.88; P = 0.0050). The approval excludes patients eligible for and not previously treated with a PD-(L)1 inhibitor.
Key findings
- In randomized BLC3001 Cohort 1, median overall survival was 12.1 months with erdafitinib versus 7.8 months with chemotherapy (HR, 0.64; 95% CI, 0.47-0.88; P = 0.0050).
- Erdafitinib also significantly improved progression-free survival and objective response rate compared with chemotherapy.
- Cohort 2 showed no overall-survival benefit versus pembrolizumab (HR, 1.18; 95% CI, 0.92-1.51), informing a limitation of use.
Why it matters
The findings support a biomarker-defined treatment option for previously treated locally advanced or metastatic urothelial carcinoma and show an overall-survival advantage over the chemotherapy comparators studied. The approval’s restriction also underscores that these results do not support erdafitinib in patients eligible for, but not yet treated with, PD-(L)1 inhibitor therapy.
What to keep in mind
The efficacy comparison was from an open-label trial and applies to patients with susceptible FGFR3 alterations who had received prior systemic therapy; the abstract does not provide detailed safety results.