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|Molecular Profile||Indication/Tumor Type||Response Type||Relevant Treatment Approaches||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|FGFR3 over exp||multiple myeloma||sensitive||FGFR3 Antibody||Vofatamab||Phase I||Actionable||In a Phase I trial, Vofatamab (B-701) demonstrated safety and resulted in stable disease in 42% (6/14) of multiple myeloma patients overexpressing FGFR3 (Blood (ASH Annual Meeting Abstracts) 2012 120: Abstract 4029).||detail...|
|FGFR3 over exp||glioblastoma||sensitive||U0126||Preclinical||Actionable||In a preclinical study, U0126 inhibited downstream signaling and growth of glioblastoma cells over-expressing Fgfr3 in culture (PMID: 23298836).||23298836|
|FGFR3 over exp||transitional cell carcinoma||sensitive||FGFR3 Inhibitor||AZD4547||Preclinical||Actionable||In a preclinical study, AZD4547 inhibited proliferation of urothelial cancer cells with over expression of FGFR3 in culture (PMID: 22869148).||22869148|
|FGFR3 over exp||transitional cell carcinoma||sensitive||FGFR3 Inhibitor||AZ8010||Preclinical||Actionable||In a preclinical study, AZ8010 inhibited proliferation of urothelial cancer cells with over expression of FGFR3 in culture (PMID: 22869148).||22869148|
|FGFR3 over exp||Advanced Solid Tumor||sensitive||FGFR Inhibitor (Pan)||LY2874455||Preclinical||Actionable||In a preclinical study, LY2874455 induced tumor regression in FGFR3-over expressing bladder and multiple myeloma xenograft models (PMID: 21900693).||21900693|
|FGFR3 over exp||urinary bladder cancer||sensitive||FGFR3 Inhibitor||SU5402||Preclinical||Actionable||In a preclinical study, SU5402 inhibited growth of bladder cancer cells over expressing wild-type FGFR3 in culture (PMID: 21119661).||21119661|
|FGFR3 over exp||head and neck squamous cell carcinoma||sensitive||FGFR Inhibitor (Pan)||Rogaratinib||Preclinical||Actionable||In a preclinical study, Rogaratinib (BAY 1163877) inhibited tumor growth in a head and neck squamous cell carcinoma xenograft model with FGFR3 overexpression (Cancer Res August 1, 2015 75:772).||detail...|
|FGFR3 over exp||urinary bladder cancer||sensitive||FGFR Inhibitor (Pan)||Dovitinib||Phase II||Actionable||In a Phase II trial, Dovitinib (TKI258) treatment resulted in complete response for 6 months in 8% (1/13) of non-muscle invasive bladder cancer patients over expressing FGFR3 (J Clin Oncol 34, 2016 (suppl; abstr 4526)).||detail...|
|FGFR3 over exp||urinary bladder cancer||sensitive||FGFR Inhibitor (Pan)||Infigratinib||Preclinical - Pdx||Actionable||In a preclinical study, treatment with Truseltiq (infigratinib) led to improved progression-free survival in a patient-derived xenograft (PDX) model of bladder cancer with FGFR3 over expression (PMID: 26270481).||26270481|
|FGFR3 over exp||urinary bladder cancer||sensitive||Dactolisib + Sorafenib||Preclinical - Pdx||Actionable||In a preclinical study, the combination of BEZ235 and Nexavar (sorafenib) resulted in improved progression-free survival in an FGFR3-over expressing patient-derived xenograft (PDX) model of bladder cancer with secondary resistance to BGJ398 due to reactivation of downstream signaling, as evidenced by increased activation of Akt and Erk (PMID: 26270481).||26270481|
|FGFR3 over exp||Advanced Solid Tumor||sensitive||FGFR Inhibitor (Pan)||PRN1371||Preclinical - Cell culture||Actionable||In a preclinical study, PRN1371 inhibited proliferation in transformed cells overexpressing wild-type FGFR3 in culture (PMID: 28978721).||28978721|
|FGFR3 over exp||Advanced Solid Tumor||predicted - sensitive||FGFR Inhibitor (Pan)||Rogaratinib||Phase I||Actionable||In a Phase I trial, treatment with Rogaratinib (BAY 1163877) was well-tolerated and resulted in objective response rate (ORR) of 15% (15/100) in patients with FGFR1, FGFR2, or FGFR3-overexpressing advanced solid tumors, including urothelial cancer, head and neck squamous cell carcinoma, and non-small cell lung cancer, and led to an ORR of 67% (10/15) in patients with FGFR overexpression, but without an FGFR genetic aberration (PMID: 31405822; NCT01976741).||31405822|
|FGFR3 over exp||lung squamous cell carcinoma||no benefit||FGFR Inhibitor (Pan)||Rogaratinib||Phase II||Actionable||In a Phase II trial (SAKK 19/18), Rogaratinib (BAY 1163877) treatment did not meet its primary endpoint for 6-month progression-free survival (PFS) in patients with advanced lung squamous cell carcinoma with overexpression of FGFR1, FGFR2, or FGFR3, and resulted in only 6.7% (1/15) of patients achieving 6-month PFS, with no objective responses, a median PFS of 1.6 months, and a median overall survival of 3.5 months (PMID: 36099710; NCT03762122).||36099710|
|FGFR3 over exp||transitional cell carcinoma||no benefit||FGFR Inhibitor (Pan)||Rogaratinib||Phase II||Actionable||In a Phase II trial (FORT-1), Rogaratinib (BAY 1163877) treatment did not result in improved outcomes compared to chemotherapy in advanced or metastatic urothelial carcinoma patients with FGFR1 or FGFR3 overexpression, with similar median overall survival (8.3 mo vs 9.8 mo), median progression-free survival (2.7 mo vs. 3.2 mo), and objective response rate (20.7% (18/87) vs 19.3% (17/88)), failing to meet the predetermined criteria for continuation to Phase III (PMID: 36240478; NCT03410693).||36240478|