with disease recurrence after bacillus CalmetteeGuérin (BCG) treatment and who are ineligible for/refuse radical
cystectomy. FGFR alterations are commonly detected in NMIBC. We evaluated the activity of oral erdafitinib, a
selective pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor, versus intravesical chemotherapy in
patients with high-risk NMIBC and select FGFR3/2 alterations following recurrence after BCG treatment.
Patients and methods: Patients aged 18 years with recurrent, BCG-treated, papillary-only high-risk NMIBC (high-grade
Ta/T1) and select FGFR alterations refusing or ineligible for radical cystectomy were randomized to 6 mg daily oral
erdafitinib or investigator’s choice of intravesical chemotherapy (mitomycin C or gemcitabine). The primary endpoint
was recurrence-free survival (RFS). The key secondary endpoint was safety.
Results: Study enrollment was discontinued due to slow accrual. Seventy-three patients were randomized 2 : 1 to
erdafitinib (n ¼ 49) and chemotherapy (n ¼ 24). Median follow-up for RFS was 13.4 months for both groups.
Median RFS was not reached for erdafitinib [95% confidence interval (CI) 16.9 months-not estimable] and was 11.6
months (95% CI 6.4-20.1 months) for chemotherapy, with an estimated hazard ratio of 0.28 (95% CI 0.1-0.6;
nominal P value ¼ 0.0008). In this population, safety results were generally consistent with known profiles for
erdafitinib and chemotherapy.
Conclusions: Erdafitinib prolonged RFS compared with intravesical chemotherapy in patients with papillary-only, highrisk NMIBC harboring FGFR alterations who had disease recurrence after BCG therapy and refused or were ineligible for
Key words: erdafitinib, FGFR, intravesical chemotherapy, non-muscle-invasive bladder cancer, recurrence-free survival,
The standard of care for patients with high-risk, papillary, n