Erdafitinib nets 40% response rate in advanced urothelial cancer

  • Loriot Y & al.
  • N Engl J Med
  • 25 Jul 2019

  • curated by Deepa Koli
  • Univadis Clinical Summaries
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Takeaway

  • 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.

Study design

  • 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.

Key results

  • 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%).

Limitations

  • Lack of an active comparator group.