Status:
Recruiting
Clinicaltrials.gov identifier:
Sponsor:
Janssen Research & Development, LLC
Enrollment:
210
Rationale:
JNJ-42756493 (Erdafitinib) is a selective and potent orally administered pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor with activity in patients with solid tumors with alterations in the FGFR pathway including urothelial carcinoma, indicating the potential to be a new therapeutic option for these patients. Recent advances in genomic profiling of urothelial carcinomas have identified potential therapeutic molecular targets in 69% of tumors (The Cancer Genome Atlas Project Nature 2014). Of the molecular alterations identified, FGFR signaling in particular is altered in a high proportion of bladder tumors in both muscle invasive (15–20%) and non-invasive tumors (70–80%).
Results:
The Phase 2 study BLC2001 presented at the 2018 ASCO Genitourinary Cancers Symposium showed an overall response rate of 42 percent in 59 patients with relapsed/refractory metastatic urothelial cancer whose tumors harbored actionable FGFR mutations (ASCO-GU abstract #411, ASCO abstract #4503)
A Breakthrough Therapy Designation in March 2018 was granted by the US FDA based on data from this multicenter, open-label Phase 2 clinical trial evaluating the efficacy and safety of erdafitinib in the treatment of adult patients with locally advanced or metastatic urothelial cancer, whose tumors have certain fibroblast growth factor receptor (FGFR) genetic alterations.
