Submission Based on Confirmatory Data from Cohort 1 of the Phase 3 THOR Study, Which Showed a 36 Percent Reduction in the Risk of Death in Patients Treated with BALVERSA® Versus Chemotherapy 

RARITAN, NJ, USA I August 28, 2023 I The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking full approval of BALVERSA® (erdafitinib), a kinase inhibitor, for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible fibroblast growth factor receptor (FGFR)3 genetic alterations, and progressed during or following at least one line of a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the locally advanced or metastatic setting or within 12 months of neoadjuvant or adjuvant therapy.1

BALVERSA® received Breakthrough Therapy Designation from the U.S. FDA in 2018 and received accelerated approval in 2019 for the treatment of adults with locally advanced or mUC which has susceptible FGFR3 or FGFR2 genetic alterations and who have 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 sNDA submission for BALVERSA® is intended to satisfy the regulatory requirements to confirm the clinical benefit of BALVERSA® based on the randomized data from Cohort 1 of the Phase 3 THOR study.2 

“BALVERSA continues to generate promising clinical findings for patients with FGFR-altered metastatic urothelial cancer, who often face poor disease outcomes,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Through the ongoing development of this targeted therapy, we are committed to transforming bladder cancer treatment to positively impact the lives of patients.”

The sNDA is based upon data from Cohort 1 of the randomized, controlled, open-label, multicenter Phase 3 THOR (NCT03390504) study evaluating the efficacy and safety of BALVERSA®. The study met its primary endpoint of overall survival (OS), with patients who received BALVERSA® achieving a median OS of over one year at the prespecified interim analysis data cutoff.2

As the interim results met the predefined criteria for superiority of treatment with BALVERSA® over chemotherapy, the independent data safety monitoring committee recommended that the study be stopped, and that patients randomized to chemotherapy be offered the opportunity to cross over to BALVERSA®. The safety profile of BALVERSA® observed in THOR was consistent with the known safety profile of BALVERSA® in mUC. Results from Cohort 1 were presented in a Late-Breaking Presentation Session (Abstract # LBA4619) at the 2023 American Society of Clinical Oncology Annual Meeting.3  

The current full prescribing information is available at

About THOR
THOR (NCT03390504) is a Phase 3 randomized, open-label, multicenter study evaluating the efficacy and safety of BALVERSA®. All patients included in the study had metastatic or unresectable UC, with selected FGFR genetic alterations, and showed disease progression during or after one or two prior lines of treatment. The study compared BALVERSA® in two cohorts; BALVERSA® versus standard of care chemotherapy (investigators choice of docetaxel or vinflunine) after at least one line of treatment including an anti-programmed death (ligand) 1 (PD-[L]1) agent (Cohort 1); and BALVERSA® compared to pembrolizumab after one prior treatment not containing an anti-PD-(L)1 agent (Cohort 2). The trial consists of screening, a treatment phase (from randomization until disease progression, intolerable toxicity, withdrawal of consent or decision by investigator to discontinue treatment) and a post-treatment follow-up (from end-of-treatment to participant’s death, withdraws consent, lost to follow-up study completion for the respective cohort, whichever comes first). A long-term extension period is planned for after the clinical cutoff date is achieved for the final analysis of each cohort for patients who continue to benefit from the study intervention. The primary endpoint of the study is OS; progression free survival (PFS), objective response rate (ORR), duration of response (DOR), patient-reported outcomes, safety and pharmacokinetics (PK) are secondary endpoints. Results from Cohort 1 were presented at the 2023 ASCO Annual Meeting earlier this year; findings from Cohort 2 will be presented at an upcoming medical meeting.2,3

BALVERSA® (erdafitinib) is a once-daily, oral FGFR kinase inhibitor that is approved by the U.S. FDA for the treatment of adults with locally advanced or mUC that have susceptible FGFR3 or FGFR2 genetic alterations and have progressed during or following at least one line of platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Patients are selected 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: 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.1,4

In addition to the Phase 3 THOR study, BALVERSA® is being studied in the Phase 2 THOR–2/BLC2003 study (NCT04172675) study examining BALVERSA® versus investigator choice of intravesical chemotherapy in participants who received Bacillus Calmette-Guérin and recurred with high risk non-muscle-invasive bladder cancer and the Phase 1 study (NCT05316155) investigating BALVERSA® in patients with  non-muscle invasive or muscle invasive bladder cancer with select FGFR alterations, given via the TARIS intravesical drug delivery system (TAR-210), which is designed to release BALVERSA® in the bladder to treat localized bladder cancer, while reducing systemic toxicities.1,2,5  

In 2008, Janssen Pharmaceutica NV entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA®.

For more information, visit

About Urothelial Carcinoma
Urothelial carcinoma, also known as transitional cell carcinoma, starts in the innermost lining of the bladder.6 It is the most common and frequent form of bladder cancer, representing more than 90 percent of all bladder cancers.7 Metastatic or unresectable disease is identified in approximately 20 percent of patients presenting with urothelial cancer, or an estimated five to eight percent of all bladder cancers. Approximately one in five patients (20 percent) diagnosed with mUC have an FGFR genetic alteration.8,9 Fibroblast growth factor receptors are a family of receptor tyrosine kinases that can be activated by genetic alterations in a variety of tumor types, and these alterations may lead to increased tumor cell growth and survival.6,10,11,12,13 Select FGFR genetic alterations can be detected through an FDA-approved companion diagnostic. The five-year survival rate for patients with Stage IV metastatic bladder cancer that has spread to distant parts of the body is currently eight percent.14

Please see the full Prescribing Information for BALVERSA®.

About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.

Learn more at Follow us at @JanssenGlobal and @JanssenUS. Janssen Research & Development, LLC, and Janssen Biotech, Inc., are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

1 BALVERSA Prescribing Information.
2 A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations. Accessed May 2023.
3 ASCO Publications. Phase 3 THOR study: Results of erdafitinib (erda) versus chemotherapy (chemo) in patients (pts) with advanced or metastatic urothelial cancer (mUC) with select fibroblast growth factor receptor alterations (FGFRalt). Available at:
4 U.S. Food & Drug Administration. FDA grants accelerated approval to erdafitinib for metastatic urothelial carcinoma. Available at: Accessed September 2021.
5 A Study of Erdafitinib in Participants with Metastatic or Locally Advanced Urothelial Cancer. Accessed May 2023.
6 American Cancer Society. “What is Bladder Cancer.” Available at Accessed May 2023.
7 National Cancer Institute. “Bladder Cancer Treatment (PDQ®)–Health Professional Version”. Available at: Accessed May 2023.
8 Tomlinson et al. FGFR3 protein expression and its relationship to mutation status and prognostic variables in bladder cancer. J Pathol. 2007;213(1):91-98.
9 De Santis M, et al. J Clin Oncol. 2011;30:191-199.
10 Helsten et al. The FGFR Landscape in Cancer: Analysis of 4,853 Tumors by Next-Generation Sequencing. Clin Cancer Res. 2015;22(1):259-267.
11 Eisenhauer E.A. et al. New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1). European Journal of Cancer. 2009. 45: 228 – 247
12 Janssen Pharmaceuticals, Inc. Data on file.
13 U.S. and World Population Clock. Accessed May 2023.
14 Bladder Cancer: Statistics. Available at: Accessed May 2023.

SOURCE: Janssen