Study Design

BALVERSA™ was studied in an open-label, single-arm phase 2 clinical trial of patients with locally advanced or metastatic UC that has susceptible FGFR3 or FGFR2 genetic alterations1

BALVERSA™ (erdafitinib) is a kinase inhibitor indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma that has

  • susceptible fibroblast growth factor receptor (FGFR)3 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.

Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA™.

Information on FDA-approved tests for the detection of FGFR genetic alterations in urothelial cancer is available at: http://www.fda.gov/CompanionDiagnostics.

This indication is approved under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

BALVERSA™ study design1

Study BLC2001 (NCT02365597) was a phase 2, multicenter, open-label, single-arm study to evaluate the efficacy and safety of BALVERSA™ in patients (N=87) with locally advanced or metastatic UC whose disease progressed on or after ≥1 line of prior platinum-based chemotherapy, and whose tumor tissue had at least 1 of the following genetic alterations: FGFR3 gene mutations (R248C, S249C, G370C, Y373C) or FGFR gene fusions (FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7), as determined by the CTA performed at a central laboratory.

Flow chart showing the clinical study design for BALVERSA

  • Dose increase based on serum phosphate levels: All patients received a starting dose of BALVERSA™ 8 mg once daily; the dose was increased to 9 mg once daily in patients whose serum phosphate levels between days 14 and 17 were below the target of 5.5 mg/dL; a dose increase occurred in 41% of patients. BALVERSA™ was administered until disease progression or unacceptable toxicity
  • Major efficacy outcome measures as determined by Blinded Independent Review Committee (BIRC) according to RECIST v1.1 were1:
    • ⁠objective response rate (ORR = complete response [CR] + partial response [PR])
    • ⁠duration of response (DOR)

66% of patients in the BALVERSA™ study had visceral metastases at baseline1

Baseline Patient Characteristics N=87
Median age (range), years 67 (36-87)
Male gender 79%
Caucasian ethnicity 74%
ECOG performance status 0 or 1 92%
Prior platinum-based regimen 97%
Prior cisplatin-based regimens only 56%
Prior carboplatin-based regimens only 29%
Both cisplatin- and carboplatin-based regimens 10%
Disease progression following prior platinum-containing neoadjuvant or adjuvant therapy only 3%
Prior anti-PD-L1/PD-1 therapy 24%
Visceral metastases 66%

CTA = clinical trial assay; ECOG = Eastern Cooperative Oncology Group; PD-L1/PD-1 = programmed death-ligand 1/programmed cell death protein; RECIST = Response Evaluation Criteria In Solid Tumors; UC = urothelial carcinoma.

Reference

1. BALVERSA™ [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc.