An evaluation of the efficacy and safety of erdafitinib for the treatment of bladder cancer

References

• Antoni S, Ferlay J, Soerjomataram I, et al. Bladder cancer incidence and mortality: a global overview and recent trends. Eur Urol. 2017;71(1):96–108.
   PubMed Web of Science ®Google Scholar
• Siegel RL, Miller KD, Jemal A. Cancer statistics, 2019. CA Cancer J Clin. 2019;69(1):7–34.
   PubMed Web of Science ®Google Scholar
• Chang SS, Bochner BH, Chou R, et al. Treatment of nonmetastatic muscle-invasive bladder cancer: American Urological Association/American Society of Clinical Oncology/American Society for Radiation Oncology/Society of urologic oncology clinical practice guideline summary. J Oncol Pract. 2017;13(9):621–625.
   PubMed Web of Science ®Google Scholar
• von der Maase H, Hansen SW, Roberts JT, et al. Gemcitabine and cisplatin versus methotrexate, vinblastine, doxorubicin, and cisplatin in advanced or metastatic bladder cancer: results of a large, randomized, multinational, multicenter, phase III study. J Clin Oncol. 2000;18(17):3068–3077.
   PubMed Web of Science ®Google Scholar
• Sternberg CN, de Mulder P, Schornagel JH, et al. Seven year update of an EORTC phase III trial of high-dose intensity M-VAC chemotherapy and G-CSF versus classic M-VAC in advanced urothelial tract tumours. Eur J Cancer. 2006;42(1):50–54.
   PubMed Web of Science ®Google Scholar
• Bellmunt J, Theodore C, Demkov T, et al. Phase III trial of vinflunine plus best supportive care compared with best supportive care alone after a platinum-containing regimen in patients with advanced transitional cell carcinoma of the urothelial tract. J Clin Oncol. 2009;27(27):4454–4461.
   PubMed Web of Science ®Google Scholar
• McCaffrey JA, Hilton S, Mazumdar M, et al. Phase II trial of docetaxel in patients with advanced or metastatic transitional-cell carcinoma. J Clin Oncol. 1997;15(5):1853–1857.
   PubMed Web of Science ®Google Scholar
• Vaughn DJ, Broome CM, Hussain M, et al. Phase II trial of weekly paclitaxel in patients with previously treated advanced urothelial cancer. J Clin Oncol. 2002;20(4):937–940.
   PubMed Web of Science ®Google Scholar
• Petrylak DP, de Wit R, Chi KN, et al. Ramucirumab plus docetaxel versus placebo plus docetaxel in patients with locally advanced or metastatic urothelial carcinoma after platinum-based therapy (RANGE): Overall survival and updated results of a randomised, double-bline, phase 3 trial. Lancet Oncol. 2020;21(1):105–120.
   PubMedGoogle Scholar
• Bellmunt J, de Wit R, Vaughn DJ, et al. Pembrolizumab as second-line therapy for advanced urothelial carcinoma. N Engl J Med. 2017;376(11):1015–1026.
   PubMed Web of Science ®Google Scholar
• Robertson AG, Kim J, Al-Ahmadie H, et al. Comprehensive molecular characterization of muscle-invasive bladder cancer. Cell. 2017;171(3):540–556. e25.
   PubMed Web of Science ®Google Scholar
• Cancer Genome Atlas Research, N. Comprehensive molecular characterization of urothelial bladder carcinoma. Nature. 2014;507(7492):315–322.
   PubMed Web of Science ®Google Scholar
• Choi W, Porten S, Kim S, et al. Identification of distinct basal and luminal subtypes of muscle-invasive bladder cancer with different sensitivities to frontline chemotherapy. Cancer Cell. 2014;25(2):152–165.
   PubMed Web of Science ®Google Scholar
• Helsten T, Elkin S, Arthur E, et al. The FGFR landscape in cancer: analysis of 4,853 tumors by next-generation sequencing. Clin Cancer Res. 2016;22(1):259–267.
   PubMed Web of Science ®Google Scholar
• Robinson BD, Vlachostergios PJ, Bhinder B, et al. Upper tract urothelial carcinoma has a luminal-papillary T-cell depleted contexture and activated FGFR3 signaling. Nat Commun. 2019;10(1):2977.
   PubMed Web of Science ®Google Scholar
• Kim YS, Kim K, Kwon GY, et al. Fibroblast growth factor receptor 3 (FGFR3) aberrations in muscle-invasive urothelial carcinoma. BMC Urol. 2018;18(1):68.
   PubMedGoogle Scholar
• Wu YM, Su F, Kalyana-Sundaram S, et al. Identification of targetable FGFR gene fusions in diverse cancers. Cancer Discov. 2013;3(6):636–647.
   PubMed Web of Science ®Google Scholar
• Williams SV, Hurst CD, Knowles MA. Oncogenic FGFR3 gene fusions in bladder cancer. Hum Mol Genet. 2013;22(4):795–803.
   PubMed Web of Science ®Google Scholar
• Milowsky MI, Dittrich C, Duran I, et al. Phase 2 trial of dovitinib in patients with progressive FGFR3-mutated or FGFR3 wild-type advanced urothelial carcinoma. Eur J Cancer. 2014;50(18):3145–3152.
   PubMed Web of Science ®Google Scholar
• Pal SK, Rosenberg JE, Hoffman-Censits JH, et al. Efficacy of BGJ398, a fibroblast growth factor receptor 1-3 inhibitor, in patients with previously treated advanced urothelial carcinoma with FGFR3 alterations. Cancer Discov. 2018;8(7):812–821.
   PubMed Web of Science ®Google Scholar
• Dizman N, Rosenberg JE, Hoffman-Censits JH, et al. Infigratinib in upper tract urothelial carcinoma vs urothelial carcinoma of the bladder and association with comprehensive genomic profiling/cell-free DNA results. Liver. 2019;37(15_suppl):4510.
   Google Scholar
• Kompier LC, Lurkin I, van der Aa MN, et al. FGFR3, HRAS, KRAS, NRAS and PIK3CA mutations in bladder cancer and their potential as biomarkers for surveillance and therapy. PLoS One. 2010;5(11):e13821.
   PubMed Web of Science ®Google Scholar
• Joerger M, Cassier PA, Penel N, et al. Rogaratinib in patients with advanced urothelial carcinomas prescreened for tumor FGFR mRNA expression and effects of mutations in the FGFR signaling pathway. J Clin Oncol. 2018;36(15_suppl):4513.
   Google Scholar
• Sternberg CN, Bellmunt J, Nishiyama H, et al. 930TiPPhase II/III study of rogaratinib versus chemotherapy in patients with locally advanced or metastatic urothelial carcinoma selected based on FGFR1/3 mRNA expression. Ann Oncol. 2018;29(suppl_8):viii331.
   Google Scholar
• Necchi A, Castellano DE, Mellado B, et al. Fierce-21: phase II study of vofatmab (B-701), a selective inhibitor of FGFR3, as salvage therapy in metastatic urothelial carcinoma (mUC). J Clin Oncol. 2019;37(7_suppl):409.
   Google Scholar
• Siefker-Radtke AO, Currie G, Abella E, et al. FIERCE-22: clinical activity of vofatamab (V) a FGFR3 selective inhibitor in combination with pembrolizumab (P) in WT metastatic urothelial carcinoma, preliminary analysis. Vaccine. 2019;37(15_suppl):4511.
   PubMedGoogle Scholar
• Rosenberg JE, O’Donnell PH, Balar AV, et al. Pivotal trial of enfortumab vedotin in urothelial carcinoma after platinum and anti-programmed death 1/programmed death ligand 1 therapy. J Clin Oncol. 2019;37(29):2592–2600.
   PubMed Web of Science ®Google Scholar
• BALVERSATM (erdafitinib): US prescribing information. 2019 [cited 2019 Oct 17]. Available from: https://www.accessdata.fda.gov/drugs atfda_docs/label/2019/21201 8s000 lbl.pdf
   Google Scholar
• Katoh M, Nakagama H. FGF receptors: cancer biology and therapeutics. Med Res Rev. 2014;34(2):280–300.
   PubMed Web of Science ®Google Scholar
• Dienstmann R, Rodon J, Prat A, et al. Genomic aberrations in the FGFR pathway: opportunities for targeted therapies in solid tumors. Ann Oncol. 2014;25(3):552–563.
   PubMed Web of Science ®Google Scholar
• Perera TPS, Jovcheva E, Mevellec L, et al. Discovery and Pharmacological characterization of JNJ-42756493 (Erdafitinib), a functionally selective small-molecule FGFR family inhibitor. Mol Cancer Ther. 2017;16(6):1010–1020.
   PubMed Web of Science ®Google Scholar
• Dai S, Zhou Z, Chen Z, et al. Fibroblast growth factor receptors (FGFRs): structures and small molecule inhibitors. Cells. 2019;8:6.
   Web of Science ®Google Scholar
• Razzaque MS. The FGF23-klotho axis: endocrine regulation of phosphate homeostasis. Nat Rev Endocrinol. 2009;5(11):611–619.
   PubMed Web of Science ®Google Scholar
• Wohrle S, Bonny O, Beluch N, et al. FGF receptors control vitamin D and phosphate homeostasis by mediating renal FGF-23 signaling and regulating FGF-23 expression in bone. J Bone Miner Res. 2011;26(10):2486–2497.
   PubMed Web of Science ®Google Scholar
• Tabernero J, Bahleda R, Dienstmann R, et al. Phase I dose-escalation study of JNJ-42756493, an oral pan-fibroblast growth factor receptor inhibitor, in patients with advanced solid tumors. J Clin Oncol. 2015;33(30):3401–3408.
   PubMed Web of Science ®Google Scholar
• Bahleda R, Italiano A, Hierro C, et al. Multicenter phase I study of erdafitinib (JNJ-42756493), oral pan-fibroblast growth factor receptor inhibitor, in patients with advanced or refractory solid tumors. Clin Cancer Res. 2019;25(16):4888–4897.
   Web of Science ®Google Scholar
• Tabernero J, Infante JR, Mita A, et al. Pharmacokinetics (PK) of the pan-FGFR inhibitor erdafitinib in urothelial carcinoma. Ann Oncol. 2016;27(suppl_6):vi273.
   Google Scholar
• Nishina T, Takahashi S, Iwasawa R, et al. Safety, pharmacokinetic, and pharmacodynamics of erdafitinib, a pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor, in patients with advanced or refractory solid tumors. Invest New Drugs. 2018;36(3):424–434.
   PubMed Web of Science ®Google Scholar
• Loriot Y, Necchi A, Park SH, et al. Erdafitinib in locally advanced or metastatic urothelial carcinoma. N Engl J Med. 2019;381(4):338–348.
   PubMed Web of Science ®Google Scholar
• FDA grants accelerated approval to erdafitinib for metastatic urothelial carcinoma. 2019 [cited 2019 Dec 4]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-erdafitinib-metastatic-urothelial-carcinoma
   Google Scholar
• Burki TK. Erdafitinib for advanced urothelial carcinoma. Lancet Oncol. 2019;20(9):e469.
   PubMed Web of Science ®Google Scholar
• Wang L, Gong Y, Saci A, et al. Fibroblast growth factor receptor 3 alterations and response to PD-1/PD-L1 blockade in patients with metastatic urothelial cancer. Eur Urol. 2019;76(5):599–603.
   PubMed Web of Science ®Google Scholar
• Palakurthi S, Kuraguchi M, Zacharek SJ, et al. The combined effect of FGFR inhibition and PD-1 blockade promotes tumor-intrinsic induction of antitumor immunity. Cancer Immunol Res. 2019;7(9):1457–1471.
   PubMed Web of Science ®Google Scholar
• Moreno V, Loriot A, Begona PV, et al. Does escalation results from phase Ib/II norse study of erdafitinib (ERDA) + PD-1 inhibitor JNJ-63723283 (Cetrelimab [CET]) in patients (pts) with metastatic or locally advanced urothelial carcinoma (mUC) and selected fibroblast growth factor receptor (FGFR) gene alterations. J Clin Oncol. 2020;38(6_suppl):511.
   Web of Science ®Google Scholar
• Park SH, Loriot Y, Zhong B, et al. Erdafitinib in high-risk patients (pts) with advanced urothelial carcinoma (UC). Vaccine. 2019;37(15_suppl):4543.
   PubMedGoogle Scholar
• Shohdy KS, Vlachostergios PJ, Abdel-Malek RR, et al. Rationale for co-targeting CDK4/6 and FGFR pathways in urothelial carcinoma. Expert Opin Ther Targets. 2019;23(2):83–86.
   PubMedGoogle Scholar
• Wang L, Sustic T, Leite de Oliveira R, et al. A functional genetic screen identifies the phosphoinositide 3-kinase pathway as a determinant of resistance to fibroblast growth factor receptor inhibitors in FGFR mutant urothelial cell carcinoma. Eur Urol. 2017;71(6):858–862.
   PubMed Web of Science ®Google Scholar