Targeting FGFR Mutations in Advanced Urothelial Cancer

Although mutationally directed therapeutics has become a standard of care for patients with lung cancer and other neoplasms, and despite the presence of myriad proposed driver mutations, therapeutic options remain limited for those with advanced urothelial cancer whose disease has failed to respond to platinum-based chemotherapy or checkpoint inhibitors (CPIs).

Now, investigators have conducted an industry-sponsored, open-label, phase II study of erdafitinib, a tyrosine kinase inhibitor of FGFR1–4, in patients with advanced urothelial cancers expressing FGFR alternations. Eligible patients had received prior platinum-based chemotherapy and had at least one FGFR3 mutation or FGFR2/3 fusion; prior therapy with CPIs was permitted.

Among the 99 p…