FDA Grants Full Approval for Balversa to Treat Advanced or Metastatic Bladder Cancer

The FDA has fully approved Balversa for treating advanced bladder cancer with specific genetic alterations. This targeted therapy, particularly for patients with FGFR3 genetic alterations in urothelial carcinoma, has shown a 36% reduction in death risk compared to chemotherapy.

Johnson & Johnson announced that the US Food and Drug Administration (FDA) has granted full approval to Balversa® (erdafitinib), marking a landmark development in targeted therapy for advanced bladder cancer. This approval establishes Balversa® as the first and only treatment for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) and susceptible FGFR3 genetic alterations.

The Phase 3 THOR study demonstrated that BALVERSA® reduces the risk of death by 36% compared to chemotherapy, marking a significant advancement for patients with limited options. These pivotal results were presented at the 2023 American Society of Clinical Oncology Annual Meeting and published in the New England Journal of Medicine, underscoring their medical significance.

BALVERSA® is specifically designed for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) characterized by susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations. This condition represents a particularly aggressive form of bladder cancer, often associated with a poor prognosis and limited treatment options. Notably, BALVERSA® is not advised for patients who are eligible for but have not yet received PD-1 or PD-L1 inhibitor therapy.

The FDA's approval, transitioning from an accelerated approval in April 2019 to a full approval, is based on the impressive results of the Phase 3 THOR study. This study demonstrated a significant 36 percent reduction in the risk of death with BALVERSA® compared to chemotherapy in patients previously treated with a PD-1 or PD-(L)1 inhibitor. Patients in the BALVERSA® arm of the study lived a median of over four months longer than those receiving chemotherapy, a substantial improvement in this context.

Approximately 20 percent of mUC patients exhibit FGFR3 genetic alterations. For these patients, the prognosis following systemic therapy, including a checkpoint inhibitor, has historically been grim. BALVERSA®'s approval offers a much-needed alternative, confirmed by Cohort 1 of the THOR study, which showed clear benefits in extending overall survival compared to chemotherapy in the second-line setting.

Kiran Patel, M.D., Vice President of Clinical Development for Solid Tumors at Johnson & Johnson, emphasizes the significance of this milestone, highlighting the promise of targeted therapies in treating advanced bladder cancer. This approval underscores the importance of precision therapies in oncology and the ongoing quest for effective treatments for such diseases.

The THOR study, a Phase 3 randomized, open-label, multicenter trial, evaluated the efficacy and safety of BALVERSA®. It compared BALVERSA® to standard chemotherapy or pembrolizumab in patients who had progressed during or after one or two prior lines of treatment. The primary endpoint was overall survival, with secondary endpoints including progression-free survival, objective response rate, duration of response, patient-reported outcomes, safety, and pharmacokinetics.