Erdafitinib demonstrated durable activity in patients with locally advanced or metastatic urothelial carcinoma with specific FGFR alterations, according to updated results from the BLC2001 trial published in The Lancet Oncology.1

The phase 2 BLC2001 trial ( Identifier: NCT02365597) was designed to investigate erdafitinib in patients with locally advanced and unresectable or metastatic urothelial carcinoma who had at least 1 FGFR3 mutation or FGFR2/3 fusion. 

The patients had to have progressed during or after 1 or more lines of previous systemic chemotherapy or within 12 months after neoadjuvant or adjuvant chemotherapy, or they had to be ineligible for cisplatin and chemotherapy-naive.

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In the previously published primary analysis of this trial, at a median follow-up of 11 months, erdafitinib was found to be active and well tolerated.2 

The current analysis1 includes 101 patients who were treated with the regimen selected in the initial part of the study. This consisted of continuous oral erdafitinib at 8 mg per day in 28-day cycles, with provision for pharmacodynamically guided uptitration to 9 mg per day. Ultimately, 60 patients received the 8 mg dose, and 41 patients were uptitrated to 9 mg.

Among the 101 patients, 69% had FGFR mutations only, 25% had fusions only, and 6% had both mutations and fusions. The most common mutations were FGFR3 S249C (46%), FGFR3 R248C (13%), and FGFR3 Y373C (12%), and the most common fusion was FGFR3 TACC3v1 (11%).

The median treatment duration was 5.4 months. Before the first disease evaluation, 2 patients had died from progressive disease. 

At a median follow-up of 24.0 months, erdafitinib produced an objective response rate of 40%. The partial response rate was 36%, and the complete response rate was 4%.

The median duration of response was 6.0 months, and 31% of patients maintained a response for at least 12 months. The disease control rate was 80% (81/101). 

The median progression-free survival (PFS) was 5.5 months, and the 12-month PFS rate was 21%. The median overall survival (OS) was 11.3 months. The 1-year OS rate was 49%, and the 2-year OS rate was 31%.

Grade 3-4 treatment-emergent adverse events of any cause were observed in 71% of patients. The most common of these were stomatitis (14%) and hyponatremia (11%). The researchers noted that there were no new safety signals or treatment-related deaths with longer follow-up.

“Erdafitinib remains an important treatment option for patients with locally advanced or metastatic urothelial carcinoma who progressed during or after one or more lines of previous platinum-based chemotherapy and who have specific FGFR alterations,” the researchers concluded.

Disclosures: This research was supported by Janssen Research & Development. Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


1. Siefker-Radtke AO, Necchi A, Park SH, et al. Efficacy and safety of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma: Long-term follow-up of a phase 2 study. Lancet Oncol. Published online January 11, 2022. doi:10.1016/S1470-2045(21)00660-4
2. Loriot Y, Necchi A, Park SH, et al. Erdafitinib in locally advanced or metastatic urothelial carcinoma. N Engl J Med. 2019;381(4):338-348. doi:10.1056/NEJMoa1817323