The U.S. Food and Drug Administration (FDA) has granted approval for Balversa (erdafitinib) for the treatment of adults with locally advanced or metastatic urothelial carcinoma who possess specific FGFR3 genetic mutations and have experienced disease progression after one line of systemic therapy. This decision expands upon the accelerated approval initially granted by the FDA in April 2019, which was specifically for patients with metastatic urothelial carcinoma featuring susceptible FGFR2 or FGFR3 alterations after prior treatment with platinum-containing chemotherapy.
Balversa, classified as a fibroblast growth factor (FGFR) inhibitor, is not recommended for patients eligible for prior treatment with a PD-1 or PD-L1 inhibitor and who have not previously received it.
The Crucial Role of FDA’s Decision on Balversa Approval for Urothelial Carcinoma
The FDA’s decision was informed by data from Study BLC3001 cohort 1, which analyzed information from 266 patients with metastatic urothelial carcinoma harboring selected FGFR3 alterations, all of whom had previously undergone one or two systemic treatments, including a PD-1 or PD-L1 inhibitor. In this open-label trial, patients were randomly assigned in a 1:1 ratio to receive either Balversa or chemotherapy as chosen by the investigator, which consisted of vinflunine or docetaxel. The randomization process was stratified based on factors such as performance status, region, and the presence of visceral or bone metastases.
FGFR3 alterations were identified in 75% of patients using the therascreen FGFR RGQ RT-PCR kit, while the remaining patients were identified through local next-generation sequencing assays. It’s important to note that the FGFR RGQ RT-PCR kit was approved as a companion diagnostic alongside Balversa in April 2019. The primary endpoint of the study was overall survival (OS), with additional outcome measures including objective response rate (ORR) and investigator-assessed progression-free survival (PFS).
Analysis of the data revealed that patients treated with Balversa achieved statistically significant improvements in OS, PFS, and ORR when compared to those who received chemotherapy. Median OS was notably longer in the Balversa group (12.1 months) than in the chemotherapy group (7.8 months). Moreover, median PFS in the Balversa cohort was significantly extended (5.6 months) compared to the chemotherapy cohort (2.7 months). The confirmed ORR was also markedly higher in the Balversa group (35.3%) compared to the chemotherapy group (8.5%).
Balversa for Urothelial Carcinoma: Safety Profile and Dosage Guidelines
Regarding safety, common adverse events reported by at least 20% of patients included nail disorders, increased phosphate levels, stomatitis, diarrhea, decreased hemoglobin, and several others. The recommended starting dose of Balversa is 8 mg taken orally once daily, with the option to increase the dose to 9 mg once daily based on patient tolerability. Patients can continue the Balversa regimen until they experience unacceptable toxicity or disease progression.
Urothelial carcinoma is the most prevalent form of bladder cancer, characterized by genetic mutations present in the bladder or the entire lining of the lower urinary tract. It ranks as the sixth most common cancer in the United States, predominantly affecting older adults with a median age of 73 years, with men being more susceptible than women. FGFR alterations are present in roughly one out of five patients with recurrent and refractory bladder cancer.
Resource: Pharmexec, January 19, 2024
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