The FDA has granted a breakthrough therapy designation for erdafitinib (Janssen Pharmaceuticals) in the treatment of urothelial cancer, an often-aggressive disease with a grim prognosis that is seen most frequently in the bladder. It’s the sixth most common type of cancer in the U.S.
The breakthrough therapy designation is based on data from a multicenter, open-label phase 2 clinical trial evaluating the efficacy and safety of erdafitinib in the treatment of adults with locally advanced or metastatic urothelial cancer whose tumors have certain fibroblast growth factor receptor (FGFR) genetic alterations. The study, BLC2001, presented at the 2018 ASCO Genitourinary Cancers Symposium, showed an overall response rate of 42% in 59 patients with relapsed/refractory metastatic urothelial cancer whose tumors harbored actionable FGFR mutations.
A breakthrough therapy designation is granted to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition. The criteria require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.
Urothelial cancer, also known as transitional cell cancer, starts in the urothelial cells that line the inside of the bladder. In 2018, an estimated 81,190 new cases of bladder cancer are expected, resulting in 17,240 deaths. For patients with metastatic disease, outcomes can be dire due to the often-rapid progression of the tumor and the lack of efficacious treatments, especially in relapsed or refractory disease. The relative five-year survival rate for patients with metastatic disease is 5%.
Erdafitinib is an oral pan-FGFR tyrosine kinase inhibitor being evaluated by Janssen in phase 2 and 3 clinical trials in patients with advanced urothelial cancer. FGFRs are a family of receptor tyrosine kinases that may be upregulated in various tumor cell types and may be involved in tumor cell differentiation and proliferation, tumor angiogenesis, and tumor cell survival. In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Therapeutics Ltd. to develop and commercialize erdafitinib.
Source: Janssen; March 15, 2018.