Efficacy of BGJ398, a fibroblast growth factor receptor 1–3 inhibitor, in patients with previously treated advanced urothelial carcinoma with FGFR3 alterations

Sumanta K. Pal, Jonathan E. Rosenberg, Jean H. Hoffman-Censits, Raanan Berger, David I. Quinn, Matthew D. Galsky, Juergen Wolf, Christian Dittrich, Bhumsuk Keam, Jean-Pierre Delord, Jan H. M. Schellens, Gwenaelle Gravis, Jacques Medioni, Pablo Maroto, Virote Sriuranpong, Chaiyut Charoentum, Howard A. Burris, Viktor Grünwald, Daniel Petrylak, Ulka VaishampayanEliahu Gez, Ugo de Giorgi, Jae-Lyun Lee, Jens Voortman, Sumati Gupta, Sunil Sharma, Amir Mortazavi, David J. Vaughn, Randi Isaacs, Katie Parker, Xueying Chen, Kun Yu, Dale Porter, Diana Graus Porta, Dean F. Bajorin

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193 Citations (Scopus)


BGJ398, a potent and selective pan-FGFR antagonist, was prospectively evaluated in patients with metastatic urothelial carcinoma bearing a diverse array of FGFR3 alterations. Patients (N = 67) who were unable to receive platinum chemotherapy were enrolled. The majority (70.1%) had received two or more prior antineoplastic therapies. BGJ398 was administered orally at 125 mg/day on a 3 weeks on, 1 week off schedule until unacceptable toxicity or progression. The primary endpoint was the response rate. Among 67 patients treated, an overall response rate of 25.4% was observed and an additional 38.8% of patients had disease stabilization, translating to a disease control rate of 64.2%. The most common treatment-emergent toxicities were hyperphosphatemia, elevated creatinine, fatigue, constipation, and decreased appetite. Further examination of BGJ398 in this disease setting is warranted. SIGnIFICAnCE: BJG398 is active in patients with alterations in FGFR3, resulting in both reductions in tumor volume and stabilization of disease. Our data highlight putative mechanisms of resistance to the agent, which may be useful in following disease status.
Original languageEnglish
Pages (from-to)812-821
JournalCancer discovery
Issue number7
Publication statusPublished - 2018

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