BALVERSA is a kinase inhibitor indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma that has...susceptible FGFR3 or FGFR2 genetic alterations and...progressed during or following at least one line of prior platinum-containing chemotherapy including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

...the NCCN Panel recommends pembrolizumab, atezolizumab, nivolumab, durvalumab, avelumab, or erdafitinib as preferred second-line systemic therapy options after platinum-based therapy….In addition to chemotherapy options, erdafitinib is also recommended for second-line systemic therapy following a first-line checkpoint inhibitor and as a third- or subsequent-line therapy option for patients who have already received both a platinum-containing therapy and a checkpoint inhibitor, if eligible on the basis of FGFR3 or FGFR2 genetic alterations.

We conducted a global phase 3 trial of erdafitinib as compared with chemotherapy in patients with metastatic urothelial carcinoma with susceptible FGFR3/2 alterations who had progression after one or two previous treatments that included an anti–PD-1 or anti–PD-L1....Erdafitinib therapy resulted in significantly longer overall survival than chemotherapy among patients with metastatic urothelial carcinoma and FGFR alterations after previous anti–PD-1 or anti–PD-L1 treatment.

Pts (≥18 y) with unresectable advanced/mUC and select FGFR3/2alt (mutations/fusions)...Primary end point was met: mOS 12.1 vs 7.8 mo in erda vs chemo (HR=0.64; 95% CI, 0.47-0.88). OS benefit was seen across subgroups (Table). Erda improved mPFS (6 vs 3 mo) and ORR (46% vs 12%) vs chemo...In pts with FGFRalt advanced/mUC after prior anti-PD-(L)1 tx, erda significantly improved OS vs chemo. Clinically relevant subgroups showed a consistent OS benefit for erda. No new safety signals were observed.

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for erdafitinib in the treatment of urothelial cancer...Breakthrough Therapy Designation is based on data from a multicenter, open-label Phase 2 clinical trial evaluating the efficacy and safety of erdafitinib in the treatment of adult patients with locally advanced or metastatic urothelial cancer, whose tumors have certain fibroblast growth factor receptor (FGFR) genetic alterations.

...age ≥18 y, with histologically confirmed, BCG-unresponsive HR-NMIBC with FGFR3/2alt...In this cohort, pts received continuous oral erda 6 mg once daily without uptitration in 28-d cycles...Of 10 enrolled pts, the CR rates at first evaluation (C3D1) and second evaluation (C6D1) were 100% (9/9 evaluable pts) and 75% (6/8 evaluable pts), respectively. The median duration of response was 3.0 mos.

...age ≥18 y, with histologically confirmed NMIBC with FGFR3/2alt...Pts received continuous oral erda 6 mg once daily without uptitration in 28-d cycles…. Pts received erda for a median duration of 2.9 mos (range 1.1-8.4). Efficacy (n=8 evaluable): 6 pts had CR (CR rate, 75.0%; 95% CI, 34.9-96.8%), and 1 had PR. Of 7 pts with CR or PR, median observed duration of response was 2.8 mos....Data from Cohort 3 of THOR-2 demonstrate efficacy in adult pts with IR-NMIBC with FGFRalt.

With longer follow-up, treatment with the selected regimen of erdafitinib showed consistent activity and a manageable safety profile in patients with locally advanced or metastatic urothelial carcinoma and prespecified FGFR alterations.

The open-label, phase II BLC2001 study enrolled pts with measurable mUC, prespecified FGFRa...Median follow-up for 101 pts treated with ERDA 8 mg/d UpT was 24.0 mos; median treatment duration was 5.4 mos….ERDA had measurable benefit in pts with advanced UC with FGFRa.

The patients were required to have at least one FGFR3 mutation or FGFR2/3 fusion...The response rate among the 74 patients with FGFR mutations in the selected-regimen group was 49% (Table 2).