The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for erdafitinib in the treatment of urothelial cancer. Urothelial cancer, most frequently in the bladder, is the sixth most common type of cancer in the U.S.¹ 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.2 The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.²

“For patients diagnosed with urothelial cancer, outcomes are unfortunately disheartening due to the aggressiveness of the disease,” said Peter Lebowitz, MD, PhD, Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Through the continued development of erdafitinib, and working closely with the FDA, we look forward to bringing a potential new treatment option to patients.”

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 adult patients with locally advanced or metastatic urothelial cancer, whose tumors have certain fibroblast growth factor receptor (FGFR) genetic alterations. 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).³

About Urothelial Cancer
Urothelial cancer, also known as transitional cell cancer, is the sixth most common type of cancer in the U.S.¹ These cancers start in the urothelial cells that line the inside of the bladder.⁴ In 2018, an estimated 81,190 new cases of bladder cancer are expected, resulting in 17,240 deaths.⁴ 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 five percent.¹

About Erdafitinib
Erdafitinib is an oral pan-fibroblast growth factor receptor (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 which may be upregulated in various tumor cell types and may be involved in tumor cell differentiation and proliferation, tumor angiogenesis, and tumor cell survival.⁵ In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Therapeutics Ltd. to develop and commercialize erdafitinib.