- A fibroblast growth factor receptor (FGFR) inhibitor shows 40% response in patients with locally advanced or metastatic urothelial carcinoma with FGFR alterations who progressed on previous treatment.
Why this matters
- FGFR alterations are common in urothelial cancer.
- These findings led to accelerated FDA approval of erdafitinib for these patients.
- Open-label, phase 2 BLC2001 study of continuous daily oral erdafitinib in 99 patients with locally advanced or metastatic urothelial carcinoma with FGFR alterations who progressed on chemotherapy.
- Funding: Janssen Research and Development.
- At data cutoff, median follow-up duration was 11.0 months.
- Median number of monthly cycles of erdafitinib was 5; median treatment duration was 5.3 months.
- The rate of confirmed response to erdafitinib therapy was 40% (complete response, 3%; partial response, 37%).
- Complete response rate was 59% in patients who progressed on immunotherapy (n=22).
- Median PFS was 5.5 (95% CI, 4.2-6.0) months, and median OS was 13.8 (95% CI, 9.8-not reached) months.
- Treatment-related grade ≥3 adverse event rate was 46%.
- 13 patients discontinued treatment because adverse event.
- Common grade ≥3 adverse events were hyponatremia (11%), stomatitis (10%), and asthenia (7%).
- Lack of an active comparator group.