First Bladder Cancer Targeted Small Molecule Receives Full FDA Approval

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First Bladder Cancer Targeted Small Molecule Receives Full FDA Approval

First Bladder Cancer Targeted Small Molecule Receives Full FDA Approval

In a recent announcement, Johnson & Johnson revealed that its small molecule inhibitor, Balversa (erdafitinib), has received comprehensive approval from the U.S. Food and Drug Administration (FDA) for the treatment of locally advanced or metastatic urothelial carcinoma (mUC) in adult patients with genetic variations in the FGFR3 gene, and whose disease has progressed following at least one line of systemic therapy.

Balversa is the first orally administered fibroblast growth factor receptor (FGFR) kinase inhibitor to receive approval and marks the inaugural targeted therapy for mUC patients with FGFR mutations.

This approval is based on the positive outcomes of the Phase 3 clinical trial (THOR). The study was a randomized, open-label, multicenter research initiative that included mUC patients with specific FGFR genetic variations, experiencing disease progression during or after first- or second-line treatment.

Results from the THOR trial indicate a significant improvement in overall survival and a 36% reduction in the risk of death when comparing Balversa to chemotherapy in patients who had previously received PD-1 or PD-L1 inhibitors during second-line treatment. Patients treated with Balversa had a median survival period 4 months longer than those receiving chemotherapy.

Common adverse reactions related to Balversa include elevated phosphate levels, nail disorders, oral mucositis, and diarrhea.

Balversa is a once-daily oral FGFR kinase inhibitor. Approximately 20% of mUC patients carry FGFR gene mutations, and Balversa achieves therapeutic effects by inhibiting FGFR. The therapy received Breakthrough Therapy Designation (BTD) from the FDA in 2018 and accelerated approval in April 2019.

First Bladder Cancer Targeted Small Molecule Receives Full FDA Approval