The pan-FGFR tyrosine kinase inhibitor erdafitinib, (Balversa), was found to be active and safe in patients suffering from locally advanced or metastatic urothelial cancer. This was based on long-term follow up of a phase II trial.
At a median follow-up of 24 months, the investigator-assessed objective response rate (ORR) was 40% among 101 patients with prespecified FGFR alterations treated with erdafitinib, reported Arlene Siefker-Radtke, MD, of the University of Texas MD Anderson Cancer Center in Houston, and colleagues.
Of these patients, 39 had a partial response, and one had a complete response. Another 41 patients had a best response of stable disease, resulting in an overall disease control rate of 80%, the authors noted in Lancet Oncology.
Median progression-free survival (PFS) was 5.5 months, with a 12-month PFS rate of 21%, while the median overall survival (OS) was 11.3 months, with 12- and 24-month OS rates of 49% and 31%.
“Our results show that erdafitinib continues to have consistent clinical benefits in patients with metastatic or locally advanced urothelial carcinomas who have tumors that are FGFR alterations. Furthermore, the safety profile of erdafitinib is manageable,” Siefker–Radtke wrote.
All patients had at least one treatment-emergent adverse event (TEAE), with 59% experiencing a TEAE leading to dose reduction. Grade 3/4 TEAEs of any cause occurred in 71% of patients, the most common of which were stomatitis (14%) and hyponatremia (11%). Half of patients suffered from grade 3 TEAEs related to erdafitinib 8, while none of the grade 4 TEAEs were related to the drug.
Median duration of response was 6.0 months, and 31% of patients had a response duration of at least 12 months.
Prespecified subgroup analyses showed similar ORRs irrespective of the presence or absence of visceral metastases:
- 33% with lymph node-only disease
- 35% with liver metastases
- 40% with lung metastases
- 35% with bone metastases
- 40% with both liver and lung metastases
- 50% with other metastatic disease
Siefker-Radtke and colleagues also found that patients with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0-1 had longer median PFS (5.6 months vs 3.2 months for those with a score of 2) and longer median OS (13.8 months vs 5.1 months, respectively).
This open-label, non-comparator study was conducted at 126 medical centers in 14 countries across Asia, Europe, and North America, and included patients ages 18 and older with locally advanced