BLC2001: Long-Term Results of Erdafitinib in Patients With Urothelial Carcinoma

By Shane Whitney - Last Updated: January 17, 2022

A study, published in The Lancet Oncology, examined the long-term characteristics of treatment with erdafitinib, a pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor, in participants with advanced urothelial carcinoma and prespecified FGFR alterations from the BLC2001 study. Arlene O Siefker-Radtke, MD, and colleagues reported that with a longer follow-up after the initial study phase, erdafitinib treatment appeared to be consistently active and manageably safe in patients with locally advanced or metastatic urothelial carcinoma.

This final phase of the BLC2001 study was conducted at 126 centers in 14 countries across Asia, Europe, and North America. All participants were aged 18 years or older, with unresectable tumors, an Eastern Cooperative Oncology Group (ECOG) performance status of zero to two, and progressive disease. The study’s treatment regimen was once daily 8 mg oral erdafitinib in 28-day cycles, and the primary outcome was investigator-assessed confirmed objective response rate based on the Response Evaluation Criteria In Solid Tumors (RECIST).

A total of 101 patients were ultimately treated with the selected erdafitinib regimen and followed up for efficacy and safety analysis. Over a median follow-up of 24.0 months (interquartile range [IQR]: 22.7–26.6), the objective response rate for treated patients was 40% (40 of 101; 95% confidence interval [CI], 30–49). According to the investigators, the safety profile was consistent with that observed in the initial data analysis, and no new safety concerns were identified. Grade III to IV treatment-emergent adverse events occurred in 71% (72 of 101), of which the most common were stomatitis in 14 patients and hyponatremia in 11 patients. There were no reported treatment-related deaths.

After the longer follow-up period and analysis, the study’s authors found favorable and persistent results, consistent with the evidence from the earlier analysis of the study cohort. The team concluded that erdafitinib appears to be a safe and effective treatment option for patients locally advanced or metastatic urothelial carcinoma and prespecified FGFR alterations.