• Jones T Nauseef, Dario M Villamar, Justin Lebenthal, Panagiotis J Vlachostergios, and Scott T Tagawa.
• Department of Medicine, Division of Hematology & Oncology, Weill Cornell Medicine , New York, NY, USA.
• Expert Opin Pharmacother. 2020 Jun 1; 21 (8): 863-870.

IntroductionTreatment of unresectable or metastatic urothelial carcinoma (UC) has historically relied upon platinum-based chemotherapy and, more recently, immune checkpoint inhibitors. When tumors progress despite those therapies, remaining effective options are limited.Areas CoveredIn this review, the authors review the advancement in genomic targets in UC, most notably fibroblast growth factor receptor (FGFR). FGFR has been identified as a target in UC as it is commonly genomically altered (activating mutations or fusions), and may be enriched in UC subtypes that are relatively resistant to immune checkpoint blockade. Erdafitinib, a potent and selective inhibitor of FGFRs, represents the first targeted therapy approved for the treatment of UC by virtue of a confirmed response rate of 40% in an open-label, single-armed phase II trial in molecularly selected tumors. The authors provide their expert opinion of its approval and place it in the context of the current and forthcoming treatment strategies for metastatic UC.Expert OpinionThe approval of erdafitinib provides clinicians with an important new treatment option for patients with metastatic UC and projects forward into an era of enhanced molecular precision in identifying effective therapies in UC.

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