Urothelial carcinoma (UC) is a common type of bladder cancer, accounting for approximately 90% of all bladder cancers. About one-fifth of mUC patients have FGFR gene alterations. FGFRs are a family of receptor tyrosine kinases that can be activated by gene alterations in various types of tumors, potentially leading to increased tumor cell growth and survival.
Janssen Pharmaceuticals, a company under the US pharmaceutical giant Johnson & Johnson (JNJ), recently received FDA approval for its Balversa (erdafitinib) for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) harboring specific fibroblast growth factor receptor (FGFR) gene alterations. Specifically, this includes adult patients with susceptible FGFR3 or FGFR2 gene alterations who have experienced disease progression during or after at least one platinum-based chemotherapy treatment (including within 12 months of neoadjuvant or adjuvant platinum-based chemotherapy).
Balversa is specifically approved for the treatment of mUC patients with tumors harboring FGFR3 or FGFR2 gene alterations. It is an oral FGFR kinase inhibitor and the first FGFR kinase inhibitor approved by the FDA in the United States. Previously, the FDA granted Balversa Breakthrough Therapy Designation (BTD) in March of last year and Priority Therapy Designation in September. This accelerated approval is based on the results of the Phase II clinical trial BLC2001 (NCT02365597).
The BLC2001 (NCT02365597) study was a multicenter, open-label, single-arm trial that enrolled 87 patients who had received at least one chemotherapy session or whose disease had progressed after chemotherapy, and who carried at least one of the following gene alterations: FGFR3 gene mutations (R248C, S249C, G370C, Y373C) or FGFR gene fusions (FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7). All participants received Balversa orally once daily during the study.
Results showed that the objective response rate (ORR) of Balversa treatment was 32.2% (95% CI: 22.4–42.0), and responders included patients who had previously failed to respond to anti-PD-1/PD-L1 therapy. In this study, the ORR was evaluated using RECIST v1.1, with a complete response rate of 2.3% and a partial response rate of 29.9%. The results also showed that the median duration of response (DoR) in patients treated with Balversa was 5.4 months (95% CI: 4.3–6.9).
