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Gene Symbol FGFR3
Synonyms ACH | CD333 | CEK2 | HSFGFR3EX | JTK4
Gene Description FGFR3, fibroblast growth factor receptor 3, is a receptor tyrosine kinase activated upon binding of the FGF ligand, which activates RAS-MAPK and PI3K-AKT pathways (PMID: 22508544). Altered function of FGFR3 in cancer may lead to increased cell proliferation and decreased apoptosis (PMID: 22508544) and mutations and fusions are commonly observed in bladder cancer (PMID: 30975452, PMID: 23175443), while FGFR3 overexpression only in bladder cancer may have different treatment implications (PMID: 32682615).
NCBI Gene ID Chromosome Map Location Canonical Transcript Gene Role
2261 4 4p16.3 NM_000142 Oncogene (PMID: 30606230)

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR3 wild-type urinary bladder cancer resistant Ponatinib Preclinical Actionable In a preclinical study, bladder cancer cells with wild-type FGFR3 were resistant to growth inhibition by Iclusig (ponatinib) in cell culture (PMID: 22238366). 22238366
FGFR3 wild-type myeloid neoplasm sensitive SSR128129E Preclinical Actionable In a preclinical study, SSR128129E inhibited proliferation of myeloma cells expressing wild-type FGFR3 in culture (PMID: 23597562). 23597562
FGFR3 wild-type bladder carcinoma sensitive Vofatamab Preclinical - Cell line xenograft Actionable In a preclinical study, Vofatamab (B-701) decreased wild-type FGFR3 activation, thereby inhibited cell proliferation of FGFR3 wild-type human bladder cancer cells in culture and in cell line xenograft models (PMID: 19381019). 19381019
FGFR3 wild-type myeloid neoplasm sensitive SU5402 Preclinical - Cell culture Actionable In a preclinical study, SU5402 inhibited proliferation of myeloma cells expressing wild-type FGFR3 in culture (PMID: 23597562). 23597562
FGFR3 dec exp FGFR3 wild-type HRAS G12V transitional cell carcinoma resistant Fexagratinib Preclinical Actionable In a preclinical study, urothelial cancer cells with low expression of wild-type FGFR3 that harbored HRAS G12V demonstrated resistance to AZD4547 in culture (PMID: 22869148). 22869148
FGFR3 dec exp FGFR3 wild-type HRAS G12V transitional cell carcinoma resistant PD173074 Preclinical Actionable In a preclinical study, urothelial cancer cells with low expression of wild-type FGFR3 that harbored HRAS G12V demonstrated resistance to PD173074 in culture (PMID: 22869148). 22869148
FGFR3 dec exp FGFR3 wild-type HRAS G12V transitional cell carcinoma decreased response AZ8010 Preclinical Actionable In a preclinical study, urothelial cancer cells with low expression of wild-type FGFR3 that harbored HRAS G12V demonstrated resistance to AZ8010 in culture (PMID: 22869148). 22869148
FGFR3 K652E Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) decreased viability of a cell line expressing FGFR3 K652E in culture (PMID: 32370101). 32370101
FGFR3 K652E urinary bladder cancer sensitive Derazantinib Preclinical - Cell culture Actionable In a preclinical study, Derazantinib (ARQ 087) inhibited growth of bladder cancer cells harboring FGFR3 K652E in culture (PMID: 27627808). 27627808
FGFR3 K652E Advanced Solid Tumor sensitive Vofatamab Preclinical - Cell culture Actionable In a preclinical study, Vofatamab (B-701) inhibited ligand-dependent cell proliferation in cells expressing FGFR3 K652E in culture (PMID: 19381019). 19381019
FGFR3 K652E urinary bladder cancer resistant PD173074 Preclinical - Cell line xenograft Actionable In a preclinical study, bladder cancer cells harboring FGFR3 K652E were resistant to PD173074 in culture and in cell line xenograft models (PMID: 23558953). 23558953
FGFR3 K652E Advanced Solid Tumor sensitive Dasatinib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Sprycel (dasatinib) treatment inhibited viability of a cell line expressing FGFR3 K652E in culture (PMID: 32370101). 32370101
FGFR3 K652E PIK3CA P124L urinary bladder cancer sensitive Alpelisib + Nintedanib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with Ofev (nintedanib) and Piqray (alpelisib) led to synergistic inhibition of growth in a bladder cancer cell line harboring FGFR3 K652E and PIK3CA P124L in culture (PMID: 36805958). 36805958
FGFR3 K652E PIK3CA P124L urinary bladder cancer sensitive Nintedanib + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with Ofev (nintedanib) and Pictilisib (GDC-0941) inhibited cell proliferation in a bladder cancer cell line harboring FGFR3 K652E PIK3CA P124L in culture (PMID: 36805958). 36805958
FGFR3 act mut Advanced Solid Tumor predicted - sensitive Fexagratinib Phase II Actionable In a Phase II (MATCH) trial, AZD4547 treatment resulted in an overall response rate of 10.5% (2/19) in patients with advanced solid tumors harboring FGFR2 or 3 activating single nucleotide variants and a 6-month progression-free survival rate of 6%, with a partial response in 1 and stable disease in 2 of 7 patients with FGFR3 activating mutations (PMID: 32463741; NCT02465060). 32463741
FGFR3 act mut Advanced Solid Tumor sensitive Dovitinib Preclinical Actionable In a preclinical study, Dovitinib (TKI258) inhibited receptor phosphorylation and cell proliferation in transformed cells expressing constitutively active FGFR3 in culture (PMID: 22238366). 22238366
FGFR3 act mut Advanced Solid Tumor decreased response Nintedanib Preclinical Actionable In a preclinical study, transformed cells expressing constitutively active FGFR3 demonstrated reduced sensitivity to Ofev (Nintedanib) in culture, when compared to other tyrosine kinase inhibitors (PMID: 22238366). 22238366
FGFR3 act mut Advanced Solid Tumor sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited receptor phosphorylation and cell growth in transformed cells expressing constitutively active FGFR3 in culture (PMID: 22238366). 22238366
FGFR3 act mut Advanced Solid Tumor no benefit Brivanib Preclinical Actionable In a preclinical study, Brivanib (BMS-540215) did not inhibit receptor phosphorylation and cell proliferation in transformed cells expressing constitutively active FGFR3 in culture (PMID: 22238366). 22238366
FGFR3 act mut Advanced Solid Tumor decreased response Cediranib Preclinical Actionable In a preclinical study, transformed cells expressing constitutively active FGFR3 demonstrated reduced sensitivity to inhibition of receptor phosphorylation and cell proliferation by Cediranib (AZD-2171) in culture, when compared to other tyrosine kinase inhibitors (PMID: 22238366). 22238366
FGFR3 act mut Advanced Solid Tumor sensitive Zoligratinib Phase I Actionable In a Phase I trial, Debio 1347 (CH5183284) dosing regimen has been determined in solid tumor patients with activating FGFR3 alterations (JCO, Vol 33, No 15_suppl (May 20 Supplement), 2015: 2540). detail...
FGFR3 act mut bladder urothelial carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in the guidelines for patients with advanced or metastatic urothelial carcinoma harboring susceptible Fgfr3 alterations after progression on platinum-based regimens (NCCN.org). detail...
FGFR3 act mut Advanced Solid Tumor predicted - sensitive Erdafitinib Phase I Actionable In a Phase I trial, Balversa (erdafitinib) treatment resulted in stable disease in 70% (16/23) and partial response in 22% (5/23) of patients with advanced solid tumors harboring FGFR 1-4 activating mutations (including amplifications, mutations and translocations), while no antitumor activity was observed in patients with unknown or no known changes in FGFR (PMID: 26324363; NCT01703481). 26324363
FGFR3 act mut Advanced Solid Tumor predicted - sensitive Erdafitinib Phase II Actionable In a Phase II trial (RAGNAR), Balversa (erdafitinib) treatment resulted in an objective response rate of 29.5% (64/217, 6 complete and 58 partial responses), a disease control rate of 74%, clinical benefit rate of 46%, a median duration of response of 6.9 months, median progression-free survival of 4.2 months, and median overall survival of 10.7 months in patients with advanced solid tumors harboring FGFR1, FGFR2, or FGFR3 mutations or fusions (PMID: 37541273; NCT04083976). 37541273
FGFR3 act mut urinary bladder cancer predicted - sensitive S-49076 Preclinical Actionable In a preclinical study, S-49076 inhibited autophosphorylation of FGFR3 and downstream signaling in bladder cancer cells over expressing constitutively active FGFR3 in culture (PMID: 23804704). 23804704
FGFR3 act mut transitional cell carcinoma no benefit Derazantinib Phase Ib/II Actionable In a Phase I/II trial (FIDES-02), Derazantinib (ARQ 087) treatment was well tolerated but demonstrated limited efficacy in patients with urothelial carcinoma harboring FGFR1 (n=4), FGFR2 (n=7), or FGFR3 (n=35) mutations or FGFR3 fusions (n=6), with an objective response rate of 8.2% (4/49, all partial responses) and disease control rate of 30.6% (15/49) by independent central review (PMID: 38627238; NCT04045613). 38627238
FGFR3 act mut transitional cell carcinoma sensitive Pemigatinib Phase II Actionable In a Phase II trial (FIGHT-201), Pemazyre (pemigatinib) treatment resulted in an objective response rate (ORR) of 17.8%, median duration of response (mDOR) of 6.2 mo, median progression-free survival (mPFS) of 4.0 mo, and median overall survival (mOS) of 6.8 mo with continuous dosing, and an ORR of 23.3%, mDOR of 6.2 mo, mPFS of 4.3 mo, and mOS of 8.9 mo with intermittent dosing in patients with urothelial carcinoma harboring FGFR3 mutations or fusions/rearrangements (PMID: 37956738; NCT02872714). 37956738
FGFR3 act mut transitional cell carcinoma no benefit Atezolizumab + Derazantinib Phase Ib/II Actionable In a Phase I/II trial (FIDES-02), combination treatment with Derazantinib (ARQ 087) and Tecentriq (atezolizumab) was well tolerated but demonstrated limited efficacy in patients with urothelial carcinoma harboring FGFR3 mutations (n=8) or FGFR3 fusions (n=2), with an objective response rate (ORR) of 0% (0/10) and disease control rate of 20.0% (2/10) by independent central review (PMID: 38627238; NCT04045613). 38627238
FGFR3 act mut Advanced Solid Tumor predicted - sensitive KIN-3248 Phase I Actionable In a Phase I trial, KIN-3248 treatment demonstrated safety and preliminary activity in patients with advanced solid tumors harboring alterations in FGFR2 or FGFR3, resulting in an objective response rate of 9.25% (5/54, 5 partial responses) and disease control rate of 46% (25/54), with stable disease in 20 patients (PMID: 38602417; NCT05242822). 38602417
FGFR3 act mut FGFR3 over exp transitional cell carcinoma predicted - sensitive Rogaratinib Phase II Actionable In a Phase II trial (FORT-1), Rogaratinib (BAY 1163877) treatment resulted in a higher objective response rate of 52.4% (11/21) compared to 26.7% (4/15) with chemotherapy in advanced or metastatic urothelial carcinoma patients with FGFR3 overexpression and an FGFR3 alteration (including FGFR3 G370C, FGFR3 R248C, FGFR3 S249C, FGFR3 Y373C, FGFR3-TACC3v1, and FGFR3-TACC3v3) (PMID: 36240478; NCT03410693). 36240478
FGFR3 mutant urinary bladder cancer sensitive Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, AZD4547 inhibited survival of bladder cancer cells harboring FGFR3 mutation in culture (PMID: 27550940). 27550940
FGFR3 mutant transitional cell carcinoma sensitive Infigratinib Clinical Study Actionable In a clinical study, Truseltiq (infigratinib) treatment in patients with FGFR3 alterations led to 25.4% (17/67) confirmed responses and a disease control rate of 64% (43/67), which included complete responses, partial responses, and stable disease, and resulted in a median progression-free survival of 3.75 months and a median overall survival of 7.75 months (PMID: 29848605). 29848605
FGFR3 mutant transitional cell carcinoma sensitive Infigratinib Phase I Actionable In a Phase I trial, Truseltiq (infigratinib) treatment resulted in complete response in 4% (1/25) and partial response in 32% (8/25) of urothelial carcinoma patients harboring FGFR3 mutations or fusions (J Clin Oncol 34, 2016 (suppl; abstr 4517); NCT01004224). detail...
FGFR3 mutant bladder urothelial carcinoma sensitive Infigratinib Phase I Actionable In a Phase I trial, patients with bladder urothelial carcinoma harboring an FGFR3 mutation demonstrated a disease control rate of 75% (6/8) when treated with Truseltiq (infigratinib), including 3 patients with a partial response and 3 with stable disease (PMID: 27870574; NCT01004224). 27870574
FGFR3 mutant Advanced Solid Tumor sensitive Infigratinib Preclinical Actionable In a preclinical study, tumor cell lines with FGFR3 mutations demonstrated sensitivity to Truseltiq (infigratinib) in culture (PMID: 23002168). 23002168
FGFR3 mutant bladder urothelial carcinoma sensitive Dovitinib Phase II Actionable In a Phase II trial, Dovitinib (TKI258) treatment resulted in complete response in 33% (1/3) of patients with BCG-unresponsive, non-muscle-invasive, urothelial carcinoma of the bladder harboring FGFR3 mutations (PMID: 27932416). 27932416
FGFR3 mutant Advanced Solid Tumor predicted - sensitive Zoligratinib Phase I Actionable In a Phase I trial, Debio 1347 treatment resulted in partial response in 10.5% (6/57) and stable disease in 28.1% (16/57) of patients with advanced solid tumors harboring genomic alterations of FGFR1/2/3, including amplifications, fusions, and mutations (PMID: 30745300; NCT01948297). 30745300
FGFR3 mutant transitional cell carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in the guidelines for patients with advanced or metastatic urothelial carcinoma harboring Fgfr3 alterations after progression on platinum-based regimens (NCCN.org). detail...
FGFR3 mutant transitional cell carcinoma sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase I trial, Balversa (erdafitinib) treatment resulted in 25% tumor shrinkage at week 8 in an urothelial cancer patient harboring FGFR3 mutations (PMID: 26324363; NCT01703481). 26324363
FGFR3 mutant transitional cell carcinoma sensitive Erdafitinib Phase I Actionable In a Phase I trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 46% (12/26) in patients with urothelial carcinoma harboring FGFR genomic alterations, including 17 with FGFR3 mutations, and 11 with FGFR2 and/or FGFR3 fusions (PMID: 31088831; NCT01703481). 31088831
FGFR3 mutant transitional cell carcinoma sensitive Erdafitinib FDA approved - Has Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 mutant bladder urothelial carcinoma sensitive Erdafitinib FDA approved - Has Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 mutant cholangiocarcinoma predicted - sensitive Erdafitinib Phase I Actionable In a Phase I trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 27% (3/11) in patients with cholangiocarcinoma harboring FGFR genomic alterations, including 1 with FGFR2 mutation, 2 with FGFR3 mutations, and 8 with FGFR2 fusions (PMID: 31088831; NCT01703481). 31088831
FGFR3 mutant Advanced Solid Tumor predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (MATCH), Balversa (erdafitinib) treatment resulted in an objective response rate of 16% (4/25), median progression-free survival of 3.6 months, and median overall survival of 11.0 months in patients with advanced solid tumors harboring FGFR1, FGFR2, or FGFR3 mutations or fusions (PMID: 38603650; NCT02465060). 38603650
FGFR3 mutant transitional cell carcinoma no benefit Atezolizumab Phase II Actionable In a Phase II trial (IMVigor 210, CheckMate 275), Tecentriq (atezolizumab) (n=119) treatment resulted in similar response rate (24% vs 21%, p=0.8) in patients with FGFR3 mutant or wild-type metastatic transitional cell carcinoma (PMID: 31272788). 31272788
FGFR3 mutant transitional cell carcinoma no benefit Nivolumab Phase II Actionable In a Phase II trial (CheckMate 275), Opdivo (nivolumab) (n=270) treatment resulted in similar response rate (20% vs 21%, p=0.2) in patients with FGFR3 mutant or wild-type metastatic transitional cell carcinoma (PMID: 31272788). 31272788
FGFR3 mutant Advanced Solid Tumor sensitive Pemigatinib Preclinical - Cell line xenograft Actionable In a preclinical study, a variety of cancer cell lines harboring mutations in FGFR1, FGFR2, and/or FGFR3 demonstrated sensitivity to Pemazyre (pemigatinib) in culture and in cell line xenograft models, resulting in inhibition of tumor growth (Cancer Res 2015;75(15 Suppl):Abstract nr 771). detail...
FGFR3 mutant transitional cell carcinoma predicted - sensitive Cetrelimab + Erdafitinib Phase II Actionable In a Phase II trial, the combination of Balversa (erdafitinib) and Cetrelimab (JNJ-63723283) treatment resulted in an overall response rate of 54.5% (6 complete responses), disease control rate of 79.5%, median duration of response of 11.10 months, median progression-free survival of 10.97 months, and a 12-month overall survival of 68% in patients with metastatic urothelial carcinoma harboring FGFR mutations (J Clin Oncol 41, 2023 (suppl 16; abstr 4504); NCT03473743). detail...
FGFR3 mutant transitional cell carcinoma predicted - sensitive Docetaxel + Vofatamab Phase Ib/II Actionable In a Phase I/II trial, Vofatamab (B-701) in combination with Taxotere (docetaxel) demonstrated safety and preliminary efficacy, resulted in enhanced activity in patients with locally advanced or metastatic urothelial carcinoma harboring FGFR3 mutations or fusions comparing to wild-type patients (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 4534-4534; NCT02401542). detail...
FGFR3 mutant Advanced Solid Tumor predicted - sensitive ICP-192 Phase I Actionable In a Phase I trial, ICP-192 (gunagratinib) was well-tolerated, and resulted in an overall response rate or 33.3% (4/12, 1 complete response, 3 partial response) and a disease control rate of 91.7% (11/12) in patients with advanced solid tumors harboring FGF/FGFR gene aberrations (J Clin Oncol 39, 2021 (suppl 15; abstr 4092); NCT03758664). detail...
FGFR3 mut FGFR3 over exp urinary bladder cancer sensitive Dovitinib Phase II Actionable In a Phase II trial, Dovitinib (TKI258) treatment resulted in complete response for 6 months in 33% (1/3) of non-muscle invasive bladder cancer patients harboring both FGFR3 mutations and FGFR3 over expression (J Clin Oncol 34, 2016 (suppl; abstr 4526)). detail...
FGFR3 fusion FGFR3 mut multiple myeloma sensitive 3D185 Preclinical - Cell culture Actionable In a preclinical study, 3D185 inhibited proliferation of a multiple myeloma cell line harboring an FGFR3 fusion and an FGFR3 mutation in culture (PMID: 31438996). 31438996
FGFR3 rearrange myeloid neoplasm sensitive Fexagratinib Preclinical - Cell line xenograft Actionable In a preclinical study, AZD4547 inhibited survival of myeloma cells harboring FGFR3 translocation in culture and in cell line xenograft models (PMID: 27550940). 27550940
FGFR3 rearrange cholangiocarcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial, Balversa (erdafitinib) treatment resulted in a confirmed objective response rate (ORR) of 40.9% (9/22, 1 complete, 8 partial responses), a median progression-free survival of 5.6 months, and median overall survival of 25.8 months in patients with cholangiocarcinoma harboring an FGFR rearrangement or FGFR short variant, with an ORR of 57.1% (8/14) with FGFR rearrangement and 12.5% (1/8) with FGFR short variant (PMID: 39138436; NCT02699606). 39138436
FGFR3 rearrange myeloid neoplasm sensitive PRN1371 Preclinical - Cell culture Actionable In a preclinical study, PRN1371 inhibited proliferation of myeloma cells harboring FGFR3 rearrangements in culture (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 1249). detail...
FGFR3 rearrange FGFR3 K650E multiple myeloma predicted - sensitive Futibatinib Preclinical - Cell culture Actionable In a preclinical study, Lytgobi (futibatinib) treatment led to inhibition of cell proliferation in a multiple myeloma cell line harboring an FGFR3 rearrangement and FGFR3 K650E in culture (PMID: 32973082). 32973082
FGFR3 rearrange FGFR3 Y373C multiple myeloma sensitive Futibatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Lytgobi (futibatinib) treatment led to inhibition of cell proliferation in a multiple myeloma cell line harboring an FGFR3 rearrangement and FGFR3 Y373C in culture, and led to tumor regression in a cell line xenograft model (PMID: 32973082). 32973082
FGFR3 rearrange NRAS act mut multiple myeloma no benefit Futibatinib Preclinical - Cell culture Actionable In a preclinical study, a multiple myeloma cell line harboring an FGFR3 rearrangement and an NRAS activating mutation was not sensitive to treatment with Lytgobi (futibatinib) in culture (PMID: 32973082). 32973082
FGFR3 rearrange FGFR3 F384L multiple myeloma no benefit Futibatinib Preclinical - Cell culture Actionable In a preclinical study, a multiple myeloma cell line harboring an FGFR3 rearrangement and FGFR3 F384L was not sensitive to treatment with Lytgobi (futibatinib) in culture (PMID: 32973082). 32973082
FGFR3 S249C urinary system cancer sensitive Fexagratinib Preclinical - Cell line xenograft Actionable In a preclinical study, AZD4547 decreased Myc expression and inhibited growth of a urinary tract cancer cell line harboring FGFR3 S249C in culture and in xenograft models (PMID: 27401245). 27401245
FGFR3 S249C renal pelvis transitional cell carcinoma predicted - sensitive Fexagratinib Case Reports/Case Series Actionable In a Phase II (MATCH) trial, AZD4547 treatment resulted in an overall response rate of 10.5% (2/19) in patients with advanced solid tumors harboring FGFR2 or 3 activating single nucleotide variants and a 6-month progression-free survival rate of 6%, including stable disease for greater than 6 months in a patient with transitional cell carcinoma of the renal pelvis harboring FGFR3 S249C (PMID: 32463741; NCT02465060). 32463741
FGFR3 S249C urinary system cancer sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) decreased Myc expression, induced cell cycle arrest, and inhibited growth of a urinary tract cancer cell line harboring FGFR3 S249C in culture (PMID: 27401245). 27401245
FGFR3 S249C Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) decreased viability of a cell line expressing FGFR3 S249C in culture (PMID: 32370101). 32370101
FGFR3 S249C Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) inhibited growth of transformed cells expressing FGFR3 S249C in culture (PMID: 23786770). 23786770
FGFR3 S249C Advanced Solid Tumor resistant Dasatinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 S249C was resistant to Sprycel (dasatinib) treatment in culture (PMID: 32370101). 32370101
FGFR3 S249C bladder urothelial carcinoma sensitive Dovitinib Case Reports/Case Series Actionable In a Phase II trial, Dovitinib (TKI258) treatment resulted in complete response in a patient with BCG-unresponsive, non-muscle-invasive, urothelial carcinoma of the bladder harboring FGFR3 S249C (PMID: 27932416). 27932416
FGFR3 S249C urinary bladder cancer sensitive Dovitinib Preclinical Actionable In a preclinical study, Dovitinib (TKI258) inhibited cell proliferation in bladder cancer cells harboring FGFR3 S249C mutation in culture (PMID: 22238366). 22238366
FGFR3 S249C Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 S249C were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 S249C Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited growth of transformed cells expressing FGFR3 S249C in culture (PMID: 23786770). 23786770
FGFR3 S249C urinary bladder cancer resistant Nintedanib Preclinical Actionable In a preclinical study, bladder cancer cells harboring FGFR3 S249C were resistant to Ofev (Nintedanib) induced inhibition of cell proliferation in culture (PMID: 22238366). 22238366
FGFR3 S249C renal pelvis transitional cell carcinoma predicted - sensitive Pazopanib Clinical Study Actionable In a case study, a patient with urothelial carcinoma of the renal pelvis harboring FGFR3 S249C demonstrated a partial response lasting 9 months following treatment with Votrient (pazopanib) (PMID: 27271022). 27271022
FGFR3 S249C urinary bladder cancer sensitive Ponatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of bladder cancer cells harboring FGFR3 S249C in culture and in cell line xenograft models (PMID: 22238366). 22238366
FGFR3 S249C Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of transformed cells expressing FGFR3 S249C in culture (PMID: 23786770). 23786770
FGFR3 S249C renal pelvis transitional cell carcinoma no benefit RO4987655 Preclinical - Cell culture Actionable In a preclinical study, renal pelvis transitional cell carcinoma cells harboring FGFR3 S249C were not sensitive to RO4987655 in culture (PMID: 26438159). 26438159
FGFR3 S249C renal pelvis transitional cell carcinoma no benefit Selumetinib Preclinical - Cell culture Actionable In a preclinical study, renal pelvis transitional cell carcinoma cells harboring FGFR3 S249C were not sensitive to Selumetinib (AZD-6244) in culture (PMID: 26438159). 26438159
FGFR3 S249C urinary bladder cancer no benefit Brivanib Preclinical Actionable In a preclinical study, Brivanib (BMS-540215) did not inhibit growth of bladder cancer cells harboring FGFR3 S249C in culture (PMID: 22238366). 22238366
FGFR3 S249C urinary bladder cancer sensitive Cediranib Preclinical Actionable In a preclinical study, Cediranib (AZD-2171) inhibited growth of bladder cancer cells harboring FGFR3 S249C mutation in culture (PMID: 22238366). 22238366
FGFR3 S249C urinary bladder cancer sensitive Zoligratinib Preclinical - Cell line xenograft Actionable In a preclinical study, Debio 1347 inhibited proliferation of bladder cancer cells harboring FGFR3 S249C in culture and inhibited tumor growth in FGFR3 S249C-positive bladder cancer cell line xenograft models (PMID: 25169980). 25169980
FGFR3 S249C ovarian cancer predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (RAGNAR), treatment with Balversa (erdafitinib) resulted in a partial response with a duration of response of 5.55 months in a patient with ovarian cancer harboring FGFR3 S249C (PMID: 37541273; NCT04083976). 37541273
FGFR3 S249C transitional cell carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 S249C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 S249C transitional cell carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 S249C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 S249C lung squamous cell carcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (RAGNAR), treatment with Balversa (erdafitinib) resulted in a partial response with a duration of response of 9.03 months in a patient with squamous non-small cell lung cancer harboring FGFR3 S249C (PMID: 37541273; NCT04083976). 37541273
FGFR3 S249C bladder urothelial carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced bladder urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 S249C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 S249C bladder urothelial carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 S249C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 S249C malignant ovarian Brenner tumor predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (MATCH), Balversa (erdafitinib) treatment resulted in an objective response rate of 16% (4/25), median progression-free survival of 3.6 months, and median overall survival of 11.0 months in patients with advanced solid tumors harboring FGFR1-3 mutations or fusions, including a partial response in a patient with malignant Brenner tumor of the ovary harboring FGFR3 S249C (PMID: 38603650; NCT02465060). 38603650
FGFR3 S249C head and neck squamous cell carcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (RAGNAR), treatment with Balversa (erdafitinib) resulted in a partial response with a duration of response of 2.89 months in a patient with squamous cell head and neck cancer harboring FGFR3 S249C (PMID: 37541273; NCT04083976). 37541273
FGFR3 S249C urinary bladder cancer sensitive Erdafitinib Phase II Actionable In a Phase II trial (THOR-2), Balversa (erdafitinib) improved median recurrence-free survival (not reached vs 11.6 mo, HR=0.28, p=0.0008) in patients with recurrent high risk non-muscle invasive bladder cancer harboring FGFR3 mutations such as S249C (n=31), R248C (n=4), G370C, (n=3) or Y373C (n=10) or FGFR2-BICC1 (n=1), FGFR3-BAIAP2L1 (n=1), or FGFR3-TACC3 (n=5) compared to intravesical chemotherapy (PMID: 37871701; NCT04172675). 37871701
FGFR3 S249C transitional cell carcinoma predicted - sensitive Futibatinib Case Reports/Case Series Actionable In a Phase I trial, Lytgobi (futibatinib) treatment led to an overall objective response rate of 15.8% (3/19, 3 partial responses) and a disease control rate of 47.4% (9/19), with stable disease in 6, in urothelial cancer patients harboring an FGFR3 mutation or FGFR1 mutation, including partial responses in 2 patients with urothelial cancer harboring FGFR3 S249C with a progression-free survival of 2.7 and 4.7 mo, and a duration of response of 1.4 and 3.4 mo, respectively (PMID: 34551969; NCT02052778). 34551969
FGFR3 S249C renal pelvis transitional cell carcinoma no benefit RO5126766 Preclinical - Cell culture Actionable In a preclinical study, renal pelvis transitional cell carcinoma cells harboring FGFR3 S249C were not sensitive to RO5126766 in culture (PMID: 26438159). 26438159
FGFR3 S249C bladder carcinoma sensitive Vofatamab Preclinical - Cell line xenograft Actionable In a preclinical study, Vofatamab (B-701) decreased dimer formation and constitutive activation of FGFR3 S249C, and inhibited growth of bladder cancer cell lines harboring FGFR3 S249C in culture and in xenograft models (PMID: 19381019). 19381019
FGFR3 S249C Advanced Solid Tumor sensitive Vofatamab Preclinical - Cell culture Actionable In a preclinical study, Vofatamab (B-701) inhibited proliferation of transformed cells expressing FGFR3 S249C in culture (PMID: 19381019). 19381019
FGFR3 S249C urinary bladder cancer resistant PD173074 Preclinical - Cell culture Actionable In a preclinical study, bladder cancer cells harboring FGFR3 S249C were resistant to PD173074 in culture (PMID: 23558953). 23558953
FGFR3 S249C urinary bladder cancer sensitive ASP5878 Preclinical - Cell line xenograft Actionable In a preclinical study, ASP5878 treatment inhibited proliferation of a bladder cancer cell line harboring FGFR3 S249C in culture, and resulted in tumor regression in xenograft models (Mol Cancer Ther December 2015 14; A170). detail...
FGFR3 S249C transitional cell carcinoma predicted - sensitive Pemigatinib Case Reports/Case Series Actionable In a clinical case study, Pemazyre (pemigatinib) treatment resulted in a partial response with a progression-free survival of 8.3 months in a patient with urothelial cancer harboring FGFR3 S249C (PMID: 37377403). 37377403
FGFR3 S249C transitional cell carcinoma predicted - sensitive Pemigatinib Case Reports/Case Series Actionable In a Phase I trial (FIGHT-101), Pemazyre (pemigatinib) treatment led to a partial response in a urothelial cancer patient harboring FGFR3 S249C (PMID: 35176457; NCT02393248). 35176457
FGFR3 S249C transitional cell carcinoma predicted - sensitive Pemigatinib Phase II Actionable In a Phase II trial (FIGHT-201), Pemazyre (pemigatinib) treatment resulted in an objective response rate (ORR) of 17.9% with continuous dosing and 24.2% with intermittent dosing in patients with urothelial carcinoma harboring FGFR3 mutations, with an ORR in patients harboring FGFR3 S249C of 23.9% with continuous dosing (n=46) and 24.6% with intermittent dosing (n=61) (PMID: 37956738; NCT02872714). 37956738
FGFR3 S249C bladder urothelial carcinoma predicted - sensitive Pembrolizumab + Pemigatinib Case Reports/Case Series Actionable In a Phase I trial (FIGHT-101), treatment with the combination of Pemazyre (pemigatinib) and Keytruda (pembrolizumab) demonstrated safety in patients with advanced solid tumors and resulted in an objective response rate of 26.9% (7/26, all partial responses), including a partial response with a duration of response of 50.3 months in a patient with urothelial bladder cancer harboring FGFR3 S249C (PMID: 38986210; NCT02393248). 38986210
FGFR3 S249C Advanced Solid Tumor predicted - resistant E7090 Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 S249C were resistant to treatment with E7090 in culture (PMID: 34272467). 34272467
FGFR3 S249C urinary bladder cancer predicted - sensitive Cisplatin + Ipatasertib Preclinical - Cell culture Actionable In a preclinical study, the addition of Ipatasertib (GDC0068) treatment resulted in enhanced sensitivity of bladder cancer cells expressing FGFR3 S249C to treatment with Platinol (cisplatin) in culture, demonstrating a greater reduction in cell proliferation and increased apoptotic activity when compared to Platinol (cisplatin) alone (PMID: 31316618). 31316618
FGFR3 S249C urinary bladder cancer predicted - sensitive Cisplatin + LY294002 Preclinical - Cell culture Actionable In a preclinical study, the addition of LY294002 treatment resulted in enhanced sensitivity of bladder cancer cells expressing FGFR3 S249C to treatment with Platinol (cisplatin) in culture, demonstrating a greater reduction in cell proliferation and increased apoptotic activity when compared to Platinol (cisplatin) alone (PMID: 31316618). 31316618
FGFR3 S249C bladder carcinoma sensitive CPL304110 Preclinical - Cell culture Actionable In a preclinical study, CPL304110 treatment inhibited proliferation of a bladder carcinoma cell line harboring FGFR3 S249C in culture (PMID: 33199155). 33199155
FGFR3 S249C urinary bladder cancer sensitive CPL304110 Preclinical - Cell line xenograft Actionable In a preclinical study, CPL304110 inhibited Erk phosphorylation in a bladder cancer cell line harboring FGFR3 S249C in culture and inhibited tumor growth in a cell line xenograft model (PMID: 38282676). 38282676
FGFR3 S249C transitional cell carcinoma sensitive Erdafitinib + Gefitinib Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Balversa (erdafitinib) and Iressa (gefitinib) inhibited cell growth of patient-derived urothelial cancer cells harboring FGFR3 S249C and PIK3CA E545A in culture, and led to synergistic inhibition of tumor growth in a patient-derived xenograft (PDX) model (PMID: 37377403). 37377403
FGFR3 S249C urinary bladder cancer sensitive 3D185 Preclinical - Cell culture Actionable In a preclinical study, 3D185 inhibited downstream signaling and proliferation in a bladder cancer cell line harboring FGFR3 S249C in culture (PMID: 31438996). 31438996
FGFR3 S249C urinary bladder cancer sensitive Erdafitinib + Quisinostat Preclinical - Cell culture Actionable In a preclinical study, the combination of Balversa (erdafitinib) and Quisinostat (JNJ-26481585) synergistically inhibited viability in a bladder cancer cell line harboring FGFR3 S249C in culture (PMID: 37479885). 37479885
FGFR3 S249C urinary bladder cancer predicted - sensitive TYRA-300 Preclinical - Cell line xenograft Actionable In a preclinical study, TYRA-300 induced tumor regression a cell line xenograft model of bladder cancer harboring FGFR3 S249C (Annals of Oncology 33 (2022): S751). detail...
FGFR3 S249C adult spinal cord glioblastoma multiforme predicted - sensitive Anlotinib + Irinotecan Case Reports/Case Series Actionable In a clinical case study, Anlotinib (AL-3818) and Camptosar (irinotecan) combination treatment resulted in symptom improvement and a partial response maintained for at least 10 months in a patient with IDH wild-type primary spinal cord glioblastoma harboring FGFR3 S249C (PMID: 35847381). 35847381
FGFR3 S249C Advanced Solid Tumor sensitive Dasatinib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Sprycel (dasatinib) treatment inhibited viability of a cell line expressing FGFR3 S249C in culture (PMID: 32370101). 32370101
FGFR3 S249C lung squamous cell carcinoma sensitive R3-altibody Preclinical - Pdx Actionable In a preclinical study, R3-altibody inhibited tumor growth in a patient-derived xenograft (PDX) model of lung squamous cell carcinoma harboring FGFR3 S249C (PMID: 39082679). 39082679
FGFR3 S249C bladder urothelial carcinoma sensitive R3-altibody Preclinical - Cell line xenograft Actionable In a preclinical study, R3-altibody inhibited Fgfr3 dimerization and proliferation in a urothelial carcinoma cell line harboring FGFR3 S249C in culture and induced tumor regression in a cell line xenograft model (PMID: 39082679). 39082679
FGFR3 S249C Advanced Solid Tumor sensitive R3-altibody Preclinical - Cell culture Actionable In a preclinical study, R3-altibody inhibited Fgfr3 dimerization and downstream signaling and decreased proliferation in a cell line expressing FGFR3 S249C in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 over exp transitional cell carcinoma sensitive PD173074 Preclinical Actionable In a preclinical study, PD173074 inhibited growth of urothelial carcinoma (UC) cells expressing high levels of FGFR3 S249C, but had reduced efficacy against UC cells with low levels of FGFR3 S249C expression (PMID: 22869148). 22869148
FGFR3 S249C PIK3CA E545K bladder urothelial carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, PIK3CA E545K was identified in the post-progression biopsy of a patient with bladder urothelial cancer harboring FGFR3 S249C who progressed on treatment with Balversa (erdafitinib) (PMID: 37377403). 37377403
FGFR3 S249C PIK3CA E545K bladder urothelial carcinoma predicted - resistant Futibatinib Case Reports/Case Series Actionable In a clinical case study, a patient with bladder urothelial cancer harboring FGFR3 S249C and an acquired PIK3CA E545K experienced primary resistance to treatment with Lytgobi (futibatinib) (PMID: 37377403). 37377403
FGFR3 S249C PIK3CA E545K transitional cell carcinoma sensitive Erdafitinib + Pictilisib Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Balversa (erdafitinib) and Pictilisib (GDC-0941) resulted in synergistic inhibition of tumor growth in a patient-derived xenograft (PDX) model of urothelial cancer harboring FGFR3 S249C and PIK3CA E545K (PMID: 37377403). 37377403
FGFR3 S249C PIK3CA E545A transitional cell carcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a clinical case study, Balversa (erdafitinib) treatment resulted in a partial response with a progression-free survival of 5.8 months in a patient with upper tract urothelial carcinoma harboring FGFR3 S249C and PIK3CA E545A (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 N540K intrahepatic cholangiocarcinoma predicted - sensitive Futibatinib Preclinical - Cell culture Actionable In a preclinical study, Lytgobi (futibatinib) inhibited viability of a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 N540K in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 N540K urinary bladder cancer resistant Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 N540K was resistant to Pemazyre (pemigatinib) in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 N540K urinary bladder cancer sensitive KIN-3248 Preclinical - Cell culture Actionable In a preclinical study, KIN-3248 inhibited viability of a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 N540K in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 N540K PIK3CA E545K transitional cell carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 N540K was identified in the post-progression biopsy of a patient with upper tract urothelial carcinoma harboring FGFR3 S249C and PIK3CA E545K who previously responded to Balversa (erdafitinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 V553L transitional cell carcinoma not predictive Pemigatinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 V553L was identified in the post-progression biopsy of a patient with upper tract urothelial carcinoma harboring FGFR3 S249C who previously responded to Pemazyre (pemigatinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 V555L Advanced Solid Tumor resistant Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 S249C and V555L was resistant to AZD4547 in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 V555L transitional cell carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 V555L was identified in the post-progression circulating tumor DNA of a patient with urothelial cancer harboring FGFR3 S249C who previously responded to Balversa (erdafitinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 V555L Advanced Solid Tumor resistant Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 S249C and V555L was resistant to Balversa (erdafitinib) in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 V555L transitional cell carcinoma predicted - resistant Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-201), FGFR3 V555L was identified in post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 S249C who previously achieved stable disease on Pemazyre (pemigatinib) treatment (PMID: 37956738; NCT02872714). 37956738
FGFR3 S249C FGFR3 V555L Advanced Solid Tumor sensitive R3-altibody Preclinical - Cell culture Actionable In a preclinical study, R3-altibody inhibited Fgfr3 downstream signaling and growth in a cell line expressing FGFR3 S249C and V555L in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 V553M bladder urothelial carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 V553M was identified in the post-progression circulating tumor DNA of a patient with bladder urothelial cancer harboring FGFR3 S249C who previously responded to Balversa (erdafitinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C TSC1 S561fs bladder urothelial carcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a clinical case study, Balversa (erdafitinib) treatment resulted in a partial response with a progression-free survival of 19.6 months in a patient with upper tract urothelial carcinoma harboring FGFR3 S249C and TSC1 S561fs (PMID: 37377403). 37377403
FGFR3 S249C TSC1 Q830* bladder urothelial carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, TSC1 Q830* was identified in the post-progression tissue biopsy and circulating tumor DNA (ctDNA) together with TSC1 Q830* in the post-progression ctDNA of a patient with bladder urothelial cancer harboring FGFR3 S249C who previously responded to Balversa (erdafitinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 amp PTEN C136fs transitional cell carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, PTEN C136fs and FGFR3 amplification (6 copies) were identified in the tissue biopsy of a patient with bladder urothelial cancer harboring FGFR3 S249C who progressed on Balversa (erdafitinib) after 1.4 months of treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 E587Q FGFR3 amp PIK3CA E726K TSC1 S561fs bladder urothelial carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 E587Q was identified in the post-progression circulating tumor DNA and FGFR3 amplification (>10 copies) and PIK3CA E726K were identified in the post-progression tissue biopsy of a patient with bladder urothelial cancer harboring FGFR3 S249C and TSC1 S561fs who previously responded to Balversa (erdafitinib) treatment (PMID: 37377403). 37377403
FGFR3 S249C FGFR3 V553M FGFR3 V555L transitional cell carcinoma predicted - resistant Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-201), FGFR3 V553M and V555L were identified in post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 S249C who previously achieved a partial response on Pemazyre (pemigatinib) treatment (PMID: 37956738; NCT02872714). 37956738
FGFR3 S249C FGFR3 M528I FGFR3 V553M transitional cell carcinoma predicted - resistant Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-201), FGFR3 V553M and M528I were identified in post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 S249C who previously achieved a partial response on Pemazyre (pemigatinib) treatment (PMID: 37956738; NCT02872714). 37956738
FGFR3 S249C FGFR3 N540S transitional cell carcinoma predicted - resistant Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-201), FGFR3 N540S was identified in post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 S249C who previously achieved stable disease on Pemazyre (pemigatinib) treatment (PMID: 37956738; NCT02872714). 37956738
FGFR2 R255W FGFR3 S249C FGFR3 V553M FGFR3 K650M transitional cell carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 V553M, FGFR3 K650M, and FGFR2 R255W, along with AKT1 E17K, was identified on post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 S249C who previously responded to Balversa (erdafitinib) (PMID: 37682528). 37682528
FGFR3 S249C FGFR3 V555M Advanced Solid Tumor resistant Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 S249C and V555M was resistant to AZD4547 in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 V555M urinary bladder cancer resistant Infigratinib Preclinical - Cell culture Actionable In a preclinical study, a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 V555M was resistant to Truseltiq (infigratinib) in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 V555M urinary bladder cancer resistant Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 V555M was resistant to Balversa (erdafitinib) in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 V555M Advanced Solid Tumor resistant Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 S249C and V555M was resistant to Balversa (erdafitinib) in culture (PMID: 39082679). 39082679
FGFR3 S249C FGFR3 V555M urinary bladder cancer resistant Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 V555M was resistant to Pemazyre (pemigatinib) in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 V555M urinary bladder cancer sensitive KIN-3248 Preclinical - Cell culture Actionable In a preclinical study, KIN-3248 inhibited viability of a bladder cancer cell line harboring FGFR3 S249C and expressing FGFR3 V555M in culture (PMID: 38437671). 38437671
FGFR3 S249C FGFR3 V555M Advanced Solid Tumor sensitive R3-altibody Preclinical - Cell culture Actionable In a preclinical study, R3-altibody inhibited Fgfr3 downstream signaling and growth in a cell line expressing FGFR3 S249C and V555M in culture (PMID: 39082679). 39082679
FGFR3 Y375C urinary bladder cancer sensitive Dovitinib Preclinical Actionable In a preclinical study, Dovitinib (TKI258) inhibited cell proliferation in bladder cancer cells harboring FGFR3 Y375C in culture (PMID: 22238366). 22238366
FGFR3 Y375C urinary bladder cancer resistant Nintedanib Preclinical Actionable In a preclinical study, bladder cancer cells harboring FGFR3 Y375C were resistant to growth inhibition by Ofev (Nintedanib) in culture (PMID: 22238366). 22238366
FGFR3 Y375C urinary bladder cancer sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited Fgfr phosphorylation and cell proliferation in bladder cancer cells harboring FGFR3 Y375C in culture (PMID: 22238366). 22238366
FGFR3 Y375C urinary bladder cancer no benefit Brivanib Preclinical Actionable In a preclinical study, Brivanib (BMS-540215) did not inhibit growth of bladder cancer cells harboring FGFR3 Y375C in culture (PMID: 22238366). 22238366
FGFR3 Y375C urinary bladder cancer resistant Cediranib Preclinical Actionable In a preclinical study, bladder cancer cells harboring an FGFR3 Y375C mutation were resistant to growth inhibition by Cediranib (AZD-2171) in culture (PMID: 22238366). 22238366
FGFR3 Y375C Advanced Solid Tumor sensitive Vofatamab Preclinical - Cell culture Actionable In a preclinical study, Vofatamab (B-701) inhibited ligand-dependent proliferation of cells expressing FGFR3 Y375C in culture (PMID: 19381019). 19381019
FGFR3 R248C Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) inhibited growth of transformed cells expressing FGFR3 R248C in culture (PMID: 23786770). 23786770
FGFR3 R248C Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 R248C were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 R248C Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited growth of transformed cells expressing FGFR3 R248C in culture (PMID: 23786770). 23786770
FGFR3 R248C Advanced Solid Tumor sensitive Pazopanib Preclinical - Cell culture Actionable In a preclinical study, Votrient (pazopanib) inhibited growth of transformed cells expressing FGFR3 R248C in culture (PMID: 23786770). 23786770
FGFR3 R248C breast cancer predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (RAGNAR), treatment with Balversa (erdafitinib) resulted in a partial response with a duration of response of 12.75 months in a patient with breast cancer harboring FGFR3 R248C (PMID: 37541273; NCT04083976). 37541273
FGFR3 R248C transitional cell carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 R248C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 R248C transitional cell carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 R248C is included in the companion diagnostic (PMID: 31340094; NCT02365597). 31340094 detail... detail...
FGFR3 R248C transitional cell carcinoma sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase III trial (THOR), Balversa (erdafitinib) treatment led to improved median overall survival (25.4 mo vs. 12.4 mo), median progression-free survival (8.4 mo vs. 2.9 mo), objective response rate (57.1% vs. 15.4%), and disease control rate (92.9% vs. 76.9%) compared to chemotherapy in Japanese patients with metastatic urothelial carcinoma with alterations in FGFR2 or FGFR3 (n=14), including FGFR3 Y373C (n=3), S249C (n=4), G370C (n=2), R248C (n=2), and FGFR3-TACC3 (n=3) (PMID: 39017806; NCT03390504). 39017806
FGFR3 R248C bladder urothelial carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced bladder urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 R248C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 R248C bladder urothelial carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 R248C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 R248C urinary bladder cancer predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (THOR-2), Balversa (erdafitinib) improved median recurrence-free survival (not reached vs 11.6 mo, HR=0.28, p=0.0008) in patients with recurrent high risk non-muscle invasive bladder cancer harboring FGFR3 mutations such as S249C (n=31), R248C (n=4), G370C, (n=3) or Y373C (n=10) or FGFR2-BICC1 (n=1), FGFR3-BAIAP2L1 (n=1), or FGFR3-TACC3 (n=5) compared to intravesical chemotherapy (PMID: 37871701; NCT04172675). 37871701
FGFR3 R248C Advanced Solid Tumor sensitive Vofatamab Preclinical - Cell culture Actionable In a preclinical study, Vofatamab (B-701) inhibited ligand-independent proliferation induced by FGFR3 R248C in cultured cells (PMID: 19381019). 19381019
FGFR3 R248C transitional cell carcinoma sensitive Dasatinib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Sprycel (dasatinib) to treatment with Truseltiq (infigratinib) enhanced inhibition of colony formation and synergistically decreased viability in a urothelial cancer cell line harboring FGFR3 R248C in culture (PMID: 32370101). 32370101
FGFR3 R248C transitional cell carcinoma sensitive Dasatinib + PD173074 Preclinical - Cell culture Actionable In a preclinical study, the addition of Sprycel (dasatinib) to treatment with PD173074 enhanced inhibition of colony formation of a urothelial cancer cell line harboring FGFR3 R248C in culture (PMID: 32370101). 32370101
FGFR3 R248C PIK3CA A1066V urinary bladder cancer sensitive Nintedanib + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with Ofev (nintedanib) and Pictilisib (GDC-0941) inhibited cell proliferation in a bladder cancer cell line harboring FGFR3 R248C and PIK3CA A1066V in culture (PMID: 36805958). 36805958
FGFR3 K650E Advanced Solid Tumor decreased response Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 K650E demonstrated reduced sensitivity to growth inhibition by AZD4547 in culture (PMID: 26992226). 26992226
FGFR3 K650E high grade glioma predicted - sensitive Infigratinib Case Reports/Case Series Actionable In a Phase II trial, Truseltiq (infigratinib) treatment resulted in limited efficacy with a 6-month progression-free survival (PFS) rate of 16.0%, a median PFS of 1.7 months, an objective response rate of 4.8% (1/21), and median overall survival of 6.7 months in patients with recurrent gliomas harboring alterations in FGFR1 or FGFR3, however, resulted in stable disease with PFS of 12.9 months in a patient harboring FGFR3 K650E (PMID: 35344029; NCT01975701). 35344029
FGFR3 K650E Advanced Solid Tumor decreased response Infigratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 K650E demonstrated reduced sensitivity to Truseltiq (infigratinib) in culture (PMID: 28034880). 28034880
FGFR3 K650E Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650E were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 K650E Advanced Solid Tumor conflicting Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). 28034880
FGFR3 K650E Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). 28034880
FGFR3 K650E myeloid neoplasm sensitive Zoligratinib Preclinical Actionable In a preclinical study, Debio 1347 inhibited proliferation of myeloma cell lines harboring FGFR3 K650E in culture (PMID: 25169980). 25169980
FGFR3 K650E Advanced Solid Tumor sensitive Zoligratinib Preclinical - Cell culture Actionable In a preclinical study, Debio 1347 inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). 28034880
FGFR3 K650E Advanced Solid Tumor sensitive Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, Balversa (erdafitinib) inhibited proliferation of transformed cells expressing FGFR3 K650E in culture (PMID: 26992226). 26992226
FGFR3 K650E Advanced Solid Tumor sensitive LY2874455 Preclinical - Cell culture Actionable In a preclinical study, LY2874455 inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). 28034880
FGFR3 K650E myeloid neoplasm no benefit SSR128129E Preclinical Actionable In a preclinical study, SSR128129E did not inhibit proliferation of myeloma cells expressing FGFR3 K650E in culture (PMID: 23597562). 23597562
FGFR3 K650E Advanced Solid Tumor sensitive PD98059 Preclinical Actionable In a preclinical study, PD98059 inhibited FGFR3 K650E-induced transformation of cells in culture (PMID: 14534538). 14534538
FGFR3 K650E myeloid neoplasm sensitive SU5402 Preclinical - Cell culture Actionable In a preclinical study, SU5402 inhibited proliferation of myeloma cells expressing FGFR3 K650E in culture (PMID: 23597562). 23597562
FGFR3 K650E Advanced Solid Tumor sensitive FIIN-2 Preclinical - Cell culture Actionable In a preclinical study, FIIN-2 inhibited proliferation of transformed cells over expressing FGFR3 K650E in culture (PMID: 28034880). 28034880
FGFR3 K650E FGFR3 over exp HRAS K117E multiple myeloma sensitive PD173074 Preclinical - Cell culture Actionable In a preclinical study, PD173074 inhibited Fgfr3 signaling, induced cell cycle arrest, and decreased proliferation of multiple myeloma cells harboring HRAS K117E and overexpression of FGFR3 K650E in culture (PMID: 22869148). 22869148
FGFR3 K650E FGFR3 over exp HRAS K117E myeloid neoplasm sensitive AZ8010 Preclinical Actionable In a preclinical study, AZ8010 inhibited Fgfr3 signaling and decreased proliferation of myeloma cells with HRAS K117E and overexpression of FGFR3 K650E in culture (PMID: 22869148). 22869148
FGFR3 K650M Advanced Solid Tumor resistant Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650M were resistant to treatment with AZD4547 in culture (PMID: 34272467). 34272467
FGFR3 K650M Advanced Solid Tumor resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650M were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 K650M Advanced Solid Tumor sensitive PRN1371 Preclinical - Cell culture Actionable In a preclinical study, PRN1371 inhibited proliferation of transformed cells over expressing FGFR3 K650M in culture (PMID: 28978721). 28978721
FGFR3 K650M Advanced Solid Tumor predicted - sensitive KIN-3248 Preclinical - Biochemical Actionable In a preclinical study, KIN-3248 inhibited kinase activity of FGFR3 K650M in an in vitro assay (PMID: 38437671). 38437671
FGFR3 K650N Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650N were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 K650N Advanced Solid Tumor sensitive Erdafitinib Preclinical - Cell line xenograft Actionable In a preclinical study, cells expressing FGFR3 K650N were sensitive to treatment with Balversa (erdafitinib) in culture, demonstrating reduced cell viability, and Balversa (erdafitinib) treatment led to inhibition of tumor growth in a cell line xenograft model (PMID: 34272467). 34272467
FGFR3 K650N Advanced Solid Tumor sensitive Futibatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650N were sensitive to treatment with Lytgobi (futibatinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 K650N Advanced Solid Tumor sensitive Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650N were sensitive to treatment with Pemazyre (pemigatinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 K650N Advanced Solid Tumor sensitive E7090 Preclinical - Cell line xenograft Actionable In a preclinical study, cells expressing FGFR3 K650N were sensitive to treatment with E7090 in culture, demonstrating reduced cell viability, and E7090 treatment led to inhibition of tumor growth in a cell line xenograft model (PMID: 34272467). 34272467
FGFR3 K650T Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 K650T were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 G370C Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G370C were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G370C Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G370C were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 G370C transitional cell carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 G370C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 G370C transitional cell carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 G370C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 G370C transitional cell carcinoma sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase III trial (THOR), Balversa (erdafitinib) treatment led to improved median overall survival (25.4 mo vs. 12.4 mo), median progression-free survival (8.4 mo vs. 2.9 mo), objective response rate (57.1% vs. 15.4%), and disease control rate (92.9% vs. 76.9%) compared to chemotherapy in Japanese patients with metastatic urothelial carcinoma with alterations in FGFR2 or FGFR3 (n=14), including FGFR3 Y373C (n=3), S249C (n=4), G370C (n=2), R248C (n=2), and FGFR3-TACC3 (n=3) (PMID: 39017806; NCT03390504). 39017806
FGFR3 G370C bladder urothelial carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced bladder urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 G370C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 G370C bladder urothelial carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 G370C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 G370C urinary bladder cancer predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (THOR-2), Balversa (erdafitinib) improved median recurrence-free survival (not reached vs 11.6 mo, HR=0.28, p=0.0008) in patients with recurrent high risk non-muscle invasive bladder cancer harboring FGFR3 mutations such as S249C (n=31), R248C (n=4), G370C, (n=3) or Y373C (n=10) or FGFR2-BICC1 (n=1), FGFR3-BAIAP2L1 (n=1), or FGFR3-TACC3 (n=5) compared to intravesical chemotherapy (PMID: 37871701; NCT04172675). 37871701
FGFR3 G370C Advanced Solid Tumor predicted - resistant E7090 Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G370C were resistant to treatment with E7090 in culture (PMID: 34272467). 34272467
FGFR3 amp urinary system cancer sensitive Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, AZD4547 decreased Myc expression, induced cell cycle arrest, and inhibited growth of a urinary tract cancer cell line harboring FGFR3 amplification in culture (PMID: 27401245). 27401245
FGFR3 amp urinary system cancer sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) decreased Myc expression, induced cell cycle arrest, and inhibited growth of a urinary tract cancer cell line harboring FGFR3 amplification in culture (PMID: 27401245). 27401245
FGFR3 amp Advanced Solid Tumor predicted - sensitive Ponatinib Preclinical Actionable In a preclinical study, Iclusig (ponatinib) inhibited growth of several tumor cell lines with FGFR alterations in culture (Cancer Res April 15, 2011 71:3560). detail...
FGFR3 amp Advanced Solid Tumor predicted - sensitive Zoligratinib Phase I Actionable In a Phase I trial, Debio 1347 treatment resulted in partial response in 10.5% (6/57) and stable disease in 28.1% (16/57) of patients with advanced solid tumors harboring genomic alterations of FGFR1/2/3, including amplifications, fusions, and mutations (PMID: 30745300; NCT01948297). 30745300
FGFR3 amp Advanced Solid Tumor no benefit Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (MATCH), Balversa (erdafitinib) treatment resulted in an objective response rate of 0% (0/18), median progression-free survival of 1.7 months, and median overall survival of 4.2 months in patients with advanced solid tumors with FGFR1 (n=12), FGFR2 (n=3), FGFR3 (n=2), or FGFR4 (n=1) amplification (PMID: 38603651; NCT02465060). 38603651
FGFR3 amp lung non-small cell carcinoma sensitive PRN1371 Preclinical - Pdx Actionable In a preclinical study, PRN1371 treatment resulted in 28.2% tumor growth inhibition in patient-derived xenograft models of FGFR3-amplified non-small cell lung cancer (PMID: 28978721). 28978721
FGFR3 amp urinary bladder cancer sensitive 3D185 Preclinical - Cell culture Actionable In a preclinical study, 3D185 inhibited proliferation of a bladder cancer cell line harboring FGFR3 amplification in culture (PMID: 31438996). 31438996
FGFR3 fusion FGFR3 amp bladder carcinoma sensitive CPL304110 Preclinical - Cell culture Actionable In a preclinical study, CPL304110 treatment inhibited proliferation of a bladder carcinoma cell line harboring FGFR3 amplification and an FGFR3 fusion in culture (PMID: 33199155). 33199155
FGFR3 Y373C Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 Y373C were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 Y373C Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 Y373C were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 Y373C myeloid neoplasm no benefit RO4987655 Preclinical - Cell culture Actionable In a preclinical study, myeloma cells harboring FGFR3 Y373C were not sensitive to RO4987655 in culture (PMID: 26438159). 26438159
FGFR3 Y373C myeloid neoplasm no benefit Selumetinib Preclinical - Cell culture Actionable In a preclinical study, myeloma cells harboring FGFR3 Y373C were not sensitive to Selumetinib (AZD-6244) in culture (PMID: 26438159). 26438159
FGFR3 Y373C myeloid neoplasm sensitive Zoligratinib Preclinical Actionable In a preclinical study, Debio 1347 inhibited proliferation of myeloma cell lines harboring FGFR3 Y373C in culture (PMID: 25169980). 25169980
FGFR3 Y373C transitional cell carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 Y373C (PMID: 38490358; ESMO.org). detail... 38490358
FGFR3 Y373C transitional cell carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 Y373C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 Y373C transitional cell carcinoma sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase III trial (THOR), Balversa (erdafitinib) treatment led to improved median overall survival (25.4 mo vs. 12.4 mo), median progression-free survival (8.4 mo vs. 2.9 mo), objective response rate (57.1% vs. 15.4%), and disease control rate (92.9% vs. 76.9%) compared to chemotherapy in Japanese patients with metastatic urothelial carcinoma with alterations in FGFR2 or FGFR3 (n=14), including FGFR3 Y373C (n=3), S249C (n=4), G370C (n=2), R248C (n=2), and FGFR3-TACC3 (n=3) (PMID: 39017806; NCT03390504). 39017806
FGFR3 Y373C bladder urothelial carcinoma sensitive Erdafitinib Guideline Actionable Balversa (erdafitinib) is included in guidelines for patients with advanced bladder urothelial carcinoma who have received chemotherapy and an immune checkpoint inhibitor and harboring select FGFR mutations including FGFR3 Y373C (PMID: 38490358; ESMO.org). 38490358 detail...
FGFR3 Y373C bladder urothelial carcinoma sensitive Erdafitinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (BCL2001) that supported FDA approval, Balversa (erdafitinib) treatment resulted in an objective response rate of 40% (40/99, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations and FGFR3 Y373C is included in the companion diagnostic (PMID: 31340094; NCT02365597). detail... 31340094 detail...
FGFR3 Y373C urinary bladder cancer sensitive Erdafitinib Phase II Actionable In a Phase II trial (THOR-2), Balversa (erdafitinib) improved median recurrence-free survival (not reached vs 11.6 mo, HR=0.28, p=0.0008) in patients with recurrent high risk non-muscle invasive bladder cancer harboring FGFR3 mutations such as S249C (n=31), R248C (n=4), G370C, (n=3) or Y373C (n=10) or FGFR2-BICC1 (n=1), FGFR3-BAIAP2L1 (n=1), or FGFR3-TACC3 (n=5) compared to intravesical chemotherapy (PMID: 37871701; NCT04172675). 37871701
FGFR3 Y373C myeloid neoplasm no benefit RO5126766 Preclinical - Cell culture Actionable In a preclinical study, myeloma cells harboring FGFR3 Y373C were not sensitive to RO5126766 in culture (PMID: 26438159). 26438159
FGFR3 Y373C bladder urothelial carcinoma predicted - sensitive Pemigatinib Case Reports/Case Series Actionable In a clinical case study, Pemazyre (pemigatinib) treatment resulted in a partial response with a progression-free survival of 8.4 months in a patient with bladder urothelial cancer harboring FGFR3 Y373C (PMID: 37377403). 37377403
FGFR3 Y373C urinary bladder cancer predicted - sensitive Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-207), Pemazyre (pemigatinib) treatment resulted in a partial response with a 50.8% decrease in target lesion and a progression-free survival of 6.2 months, and stable disease with a 30% decrease in target lesion and progression-free survival of 3.9 months in two patients with bladder cancer harboring FGFR3 Y373C (Cancer Res (2023) 83 (8_Supplement): CT016; NCT03822117). detail...
FGFR3 Y373C transitional cell carcinoma predicted - sensitive Pembrolizumab + Pemigatinib Case Reports/Case Series Actionable In a Phase I trial (FIGHT-101), treatment with the combination of Pemazyre (pemigatinib) and Keytruda (pembrolizumab) demonstrated safety in patients with advanced solid tumors and resulted in an objective response rate of 26.9% (7/26, all partial responses), including a partial response with a duration of response of 4.2 months in a patient with urothelial cancer harboring FGFR3 Y373C (PMID: 38986210; NCT02393248). 38986210
FGFR3 Y373C multiple myeloma sensitive E7090 Preclinical - Cell culture Actionable In a preclinical study, a multiple myeloma cell line harboring FGFR3 Y373C (PMID: 19901323) demonstrated sensitivity to E7090 in culture, resulting in decreased cell viability (PMID: 27535969). 19901323 27535969
FGFR3 Y373C Advanced Solid Tumor predicted - resistant E7090 Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 Y373C were resistant to treatment with E7090 in culture (PMID: 34272467). 34272467
FGFR3 Y373C bladder urothelial carcinoma predicted - sensitive Anlotinib + Sintilimab Case Reports/Case Series Actionable In a clinical case study, a patient with metastatic bladder urothelial cancer harboring FGFR3 Y373C, PIK3CA E542K and TP53 R213* who had previously progressed on combination treatment with Sintilimab (IBI308) and Abraxane (nab-paclitaxel), achieved a partial response after 3 cycles of combination treatment with Sintilimab (IBI308) and Anlotinib (AL-3818), and stable disease has been observed for over 11 months (PMID: 34109111). 34109111
FGFR3 Y373C FGFR3 over exp multiple myeloma sensitive PD173074 Preclinical Actionable In a preclinical study, PD173074 inhibited Fgfr3 signaling and decreased proliferation and survival of multiple myeloma cells over expressing FGFR3 Y373C in culture (PMID: 22869148). 22869148
FGFR3 Y373C FGFR3 over exp multiple myeloma sensitive AZ8010 Preclinical Actionable In a preclinical study, AZ8010 inhibited Fgfr3 signaling, induced cell-cycle arrest, and decreased proliferation of multiple myeloma cells over expressing FGFR3 Y373C in culture (PMID: 22869148). 22869148
FGFR3 Y373C FGFR3 N540K FGFR3 V555L FGFR3 L608V bladder urothelial carcinoma predicted - resistant Futibatinib Case Reports/Case Series Actionable In a clinical case study, FGFR3 N540K, FGFR3 V555L, and FGFR3 L608V were identified in the post-progression circulating tumor DNA of a patient with bladder urothelial carcinoma harboring FGFR3 Y373C who previously responded to Lytgobi (futibatinib) treatment (PMID: 37377403). 37377403
FGFR3 Y373C FGFR3 N540K transitional cell carcinoma predicted - resistant Pemigatinib Case Reports/Case Series Actionable In a Phase II trial (FIGHT-201), FGFR3 N540K was identified in post-progression biopsy in a patient with urothelial carcinoma harboring FGFR3 Y373C who previously achieved stable disease on Pemazyre (pemigatinib) treatment (PMID: 37956738; NCT02872714). 37956738
FGFR3 S371C Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 S371C were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 S371C Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 S371C were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 A391E bladder urothelial carcinoma predicted - sensitive Fexagratinib Case Reports/Case Series Actionable In a Phase II (MATCH) trial, AZD4547 treatment resulted in an overall response rate of 10.5% (2/19) in patients with advanced solid tumors harboring FGFR2 or 3 single nucleotide variants and a 6-month progression-free survival (PFS) rate of 6%, with stable disease in 2 of 7 patients with FGFR3 activating mutations, and a partial response in a patient with urinary bladder transitional cell carcinoma harboring FGFR3 A391E (reported as A393E) (PMID: 32463741; NCT02465060). 32463741
FGFR3 A391E Advanced Solid Tumor predicted - sensitive Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 A391E were sensitive to treatment with AZD4547 in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 A391E Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 A391E were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 A391E Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 A391E were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 A391E Advanced Solid Tumor predicted - sensitive Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 A391E were sensitive to treatment with Balversa (erdafitinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 A391E Advanced Solid Tumor predicted - sensitive Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 A391E were sensitive to treatment with Pemazyre (pemigatinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G380R Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380R were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G380R Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380R were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 G380R Advanced Solid Tumor predicted - sensitive Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380R were sensitive to treatment with Balversa (erdafitinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G380R Advanced Solid Tumor predicted - sensitive Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380R were sensitive to treatment with Pemazyre (pemigatinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 F386L myeloid neoplasm sensitive Zoligratinib Preclinical Actionable In a preclinical study, Debio 1347 inhibited proliferation of myeloma cell lines harboring FGFR3 F386L in culture (PMID: 25169980). 25169980
FGFR3 over exp transitional cell carcinoma sensitive Fexagratinib 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 urinary bladder cancer sensitive 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 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 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 transitional cell carcinoma no benefit 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
FGFR3 over exp lung squamous cell carcinoma no benefit 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 head and neck squamous cell carcinoma sensitive 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 Advanced Solid Tumor predicted - sensitive 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 Advanced Solid Tumor sensitive 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 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 multiple myeloma sensitive 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 transitional cell carcinoma sensitive 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 urinary bladder cancer sensitive 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 Advanced Solid Tumor sensitive 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 transitional cell carcinoma sensitive Atezolizumab + Rogaratinib Phase I Actionable In a Phase Ib trial (FORT-2), Tecentriq (atezolizumab) and Rogaratinib (BAY 1163877) combined therapy demonstrated safety and led to an objective response rate (ORR) of 43% (16/37, 5 complete and 11 partial responses), disease control rate of 65% (24/37), median progression-free survival of 6.1 mo, median overall survival of 12.0 mo, and ORR of 54% (14/26) at the RP2D of 600mg in cisplatin-ineligible patients with metastatic urothelial cancer with FGFR1 or FGFR3 overexpression (PMID: 39298147; NCT03473756). 39298147
FGFR3 G384D FGFR3 over exp multiple myeloma decreased response Fexagratinib Preclinical Actionable In a preclinical study, multiple myeloma cells over expressing FGFR3 G384D had reduced sensitivity to AZD4547 in culture, compared to cells with ligand-independent activation of FGFR3 (PMID: 22869148). 22869148
FGFR3 G384D FGFR3 over exp multiple myeloma decreased response PD173074 Preclinical Actionable In a preclinical study, multiple myeloma cells over expressing FGFR3 G384D had reduced sensitivity to PD173074 in culture, compared to cells with ligand-independent activation of FGFR3 (PMID: 22869148). 22869148
FGFR3 G384D FGFR3 over exp multiple myeloma decreased response AZ8010 Preclinical Actionable In a preclinical study, multiple myeloma cells over expressing FGFR3 G384D had reduced sensitivity to AZ8010 in culture, compared to cells with ligand-independent activation of FGFR3 (PMID: 22869148). 22869148
FGFR3 positive lung squamous cell carcinoma predicted - sensitive Rogaratinib Case Reports/Case Series Actionable In a Phase I trial, sensitivity to treatment with Rogaratinib (BAY 1163877) was demonstrated in patients with a variety of FGFR-expressing solid tumor types, including long lasting stable disease in a patient with FGFR3-positive lung squamous cell carcinoma (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #379P; NCT01976741). detail...
FGFR3 positive lung adenocarcinoma predicted - sensitive Rogaratinib Case Reports/Case Series Actionable In a Phase I trial, sensitivity to treatment with Rogaratinib (BAY 1163877) was demonstrated in patients with a variety of FGFR-expressing solid tumor types, including long lasting stable disease in a patient with FGFR3-positive lung adenocarcinoma (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #379P; NCT01976741). detail...
FGFR3 positive head and neck squamous cell carcinoma predicted - sensitive Rogaratinib Case Reports/Case Series Actionable In a Phase I trial, sensitivity to treatment with Rogaratinib (BAY 1163877) was demonstrated in patients with a variety of FGFR-expressing solid tumor types, including a partial response in a patient with FGFR3-positive head and neck squamous cell carcinoma (PMID: 31405822; NCT01976741). 31405822
FGFR3 positive stomach cancer predicted - sensitive Rogaratinib Case Reports/Case Series Actionable In a Phase I trial, sensitivity to treatment with Rogaratinib (BAY 1163877) was demonstrated in patients with a variety of FGFR-expressing solid tumor types, including long lasting stable disease in a patient with FGFR3-positive gastric cancer (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #379P; NCT01976741). detail...
FGFR2 pos FGFR3 pos breast carcinoma sensitive E7090 Preclinical Actionable In a preclinical study, a mouse breast carcinoma cell line xenograft model demonstrated inhibition of tumor growth when treated with E7090, a result of decreased FGFR2 and FGFR3 activity (PMID: 27535969). 27535969
FGFR3 fusion urinary bladder cancer sensitive Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, AZD4547 inhibited survival of bladder cancer cells harboring FGFR3 fusion in culture (PMID: 27550940). 27550940
FGFR3 fusion transitional cell carcinoma sensitive Infigratinib Phase I Actionable In a Phase I trial, Truseltiq (infigratinib) treatment resulted in complete response in 4% (1/25) and partial response in 32% (8/25) of urothelial carcinoma patients harboring FGFR3 mutations or fusions (J Clin Oncol 34, 2016 (suppl; abstr 4517)). detail...
FGFR3 fusion urinary bladder cancer sensitive Regorafenib Preclinical - Cell culture Actionable In a preclinical study, bladder cancer cell lines harboring an FGFR3 fusion were sensitive to treatment with Stivarga (regorafenib), demonstrating inhibition of cell growth (PMID: 33563752). 33563752
FGFR3 fusion Advanced Solid Tumor no benefit Zoligratinib Phase I Actionable In a Phase I trial, Debio 1347 treatment resulted in partial response in 10.5% (6/57) and stable disease in 28.1% (16/57) of patients with advanced solid tumors harboring genomic alterations of FGFR1/2/3, including amplifications, fusions, and mutations (PMID: 30745300; NCT01948297). 30745300
FGFR3 fusion Advanced Solid Tumor no benefit Zoligratinib Phase II Actionable In a Phase II trial (FUZE), Debio 1347 treatment demonstrated manageable toxicity but limited efficacy in patients with advanced solid tumors harboring a fusion in FGFR1, FGFR2, or FGFR3, resulting in an objective response rate of 5% (3/58, all partial responses), with stable disease in in 45% (26/58) of patients, and a median progression-free survival of 3.55 months at a median follow-up of 3.6 months, and further enrollment to the trial was terminated due to lack of efficacy (PMID: 38771739; NCT03834220). 38771739
FGFR3 fusion transitional cell carcinoma predicted - sensitive Erdafitinib Phase I Actionable In a Phase I trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 46% (12/26) in patients with urothelial carcinoma harboring FGFR genomic alterations, including 17 with FGFR3 mutations, and 11 with FGFR2 and/or FGFR3 fusions (PMID: 31088831; NCT01703481). 31088831
FGFR3 fusion Advanced Solid Tumor predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial (MATCH), Balversa (erdafitinib) treatment resulted in an objective response rate of 16% (4/25), median progression-free survival of 3.6 months, and median overall survival of 11.0 months in patients with advanced solid tumors harboring FGFR1, FGFR2, or FGFR3 mutations or fusions (PMID: 38603650; NCT02465060). 38603650
FGFR3 fusion Advanced Solid Tumor predicted - sensitive Erdafitinib Phase II Actionable In a Phase II trial (RAGNAR), Balversa (erdafitinib) treatment resulted in an objective response rate of 29.5% (64/217, 6 complete and 58 partial responses), a disease control rate of 74%, clinical benefit rate of 46%, a median duration of response of 6.9 months, median progression-free survival of 4.2 months, and median overall survival of 10.7 months in patients with advanced solid tumors harboring FGFR1, FGFR2, or FGFR3 mutations or fusions (PMID: 37541273; NCT04083976). 37541273
FGFR3 fusion Advanced Solid Tumor predicted - sensitive Futibatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Lytgobi (futibatinib) demonstrated growth inhibition and reduced FGFR phosphorylation in human cancer cell lines and xenograft models harboring FGFR mutations (Mol Cancer Ther 2013;12(11 Suppl):A270). detail...
FGFR3 fusion transitional cell carcinoma no benefit Derazantinib Phase Ib/II Actionable In a Phase I/II trial (FIDES-02), Derazantinib (ARQ 087) treatment was well tolerated but demonstrated limited efficacy in patients with urothelial carcinoma harboring FGFR1 (n=4), FGFR2 (n=7), or FGFR3 (n=35) mutations or FGFR3 fusions (n=6), with an objective response rate of 8.2% (4/49, all partial responses) and disease control rate of 30.6% (15/49) by independent central review (PMID: 38627238; NCT04045613). 38627238
FGFR3 fusion transitional cell carcinoma sensitive Pemigatinib Phase II Actionable In a Phase II trial (FIGHT-201), Pemazyre (pemigatinib) treatment resulted in an objective response rate (ORR) of 17.8%, median duration of response (mDOR) of 6.2 mo, median progression-free survival (mPFS) of 4.0 mo, and median overall survival (mOS) of 6.8 mo with continuous dosing, and an ORR of 23.3%, mDOR of 6.2 mo, mPFS of 4.3 mo, and mOS of 8.9 mo with intermittent dosing in patients with urothelial carcinoma harboring FGFR3 mutations or fusions/rearrangements (PMID: 37956738; NCT02872714). 37956738
FGFR3 fusion Advanced Solid Tumor predicted - sensitive Pemigatinib Phase II Actionable In a Phase II trial (FIGHT-207), Pemazyre (pemigatinib) treatment demonstrated safety in previously treated patients with advanced solid tumors harboring a fusion in FGFR1, FGFR2, or FGFR3, and resulted in an objective response rate of 26.5% (13/49, 1 complete and 12 partial responses), with a median duration of response of 7.8 months, a clinical benefit rate of 28.6%, a median progression-free survival of 4.5 months, and a median overall survival of 17.5 months (PMID: 38710951; NCT03822117). 38710951
FGFR3 fusion transitional cell carcinoma predicted - sensitive Docetaxel + Vofatamab Phase Ib/II Actionable In a Phase I/II trial, Vofatamab (B-701) in combination with Taxotere (docetaxel) demonstrated safety and preliminary efficacy, resulted in enhanced activity in patients with locally advanced or metastatic urothelial carcinoma harboring FGFR3 mutations or fusions comparing to wild-type patients (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 4534-4534; NCT02401542). detail...
FGFR3 fusion transitional cell carcinoma no benefit Atezolizumab + Derazantinib Phase Ib/II Actionable In a Phase I/II trial (FIDES-02), combination treatment with Derazantinib (ARQ 087) and Tecentriq (atezolizumab) was well tolerated but demonstrated limited efficacy in patients with urothelial carcinoma harboring FGFR3 mutations (n=8) or FGFR3 fusions (n=2), with an objective response rate (ORR) of 0% (0/10) and disease control rate of 20.0% (2/10) by independent central review (PMID: 38627238; NCT04045613). 38627238
FGFR3 fusion transitional cell carcinoma predicted - sensitive ABSK061 Case Reports/Case Series Actionable In a Phase I trial, ABSK061 treatment resulted in a partial response in a patient with urothelial carcinoma harboring an FGFR3 fusion (ESMO Open 9 (2024): 102274); NCT05244551). detail...
FGFR3 V555M Advanced Solid Tumor resistant Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 V555M were resistant to AZD4547 in culture (PMID: 28034880). 28034880
FGFR3 V555M transitional cell carcinoma resistant Infigratinib Case Reports/Case Series Actionable In a clinical study, a patient with metastatic urothelial carcinoma progressed while being treated with Truseltiq (infigratinib) and subsequent cell-free DNA testing revealed FGFR3 V555M (also corresponds to V443M) (PMID: 29848605). 29848605
FGFR3 V555M Advanced Solid Tumor resistant Infigratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 V555M were resistant to Truseltiq (infigratinib) in culture (PMID: 28034880). 28034880
FGFR3 V555M Advanced Solid Tumor sensitive Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited proliferation of transformed cells over expressing FGFR3 V555M in culture (PMID: 28034880). 28034880
FGFR3 V555M Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 V555M in culture (PMID: 28034880). 28034880
FGFR3 V555M Advanced Solid Tumor decreased response Zoligratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 V555M demonstrated reduced sensitivity to Debio 1347 in culture (PMID: 28034880). 28034880
FGFR3 V555M Advanced Solid Tumor sensitive LY2874455 Preclinical - Cell culture Actionable In a preclinical study, LY2874455 inhibited proliferation of transformed cells over expressing FGFR3 V555M in culture (PMID: 28034880). 28034880
FGFR3 V555M Advanced Solid Tumor resistant Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing FGFR3 V555M was resistant to Pemazyre (pemigatinib) in a kinase assay and in culture (PMID: 36698015). 36698015
FGFR3 V555M Advanced Solid Tumor decreased response FIIN-2 Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 V555M demonstrated reduced sensitivity to FIIN-2 in culture (PMID: 28034880). 28034880
FGFR3 V555M FGFR3 L608V transitional cell carcinoma predicted - resistant Infigratinib Case Reports/Case Series Actionable In a clinical study, a patient with metastatic urothelial carcinoma progressed while being treated with Truseltiq (infigratinib) and subsequent cell-free DNA testing revealed FGFR3 V555M (also corresponds to V443M) and FGFR3 L608V (also corresponds to L496V) (PMID: 29848605). 29848605
FGFR3 N540K Advanced Solid Tumor resistant Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 N540K were reistant to growth inhibition by AZD4547 in culture (PMID: 26992226). 26992226
FGFR3 N540K Advanced Solid Tumor resistant Infigratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 N540K were resistant to Truseltiq (infigratinib) in culture (PMID: 28034880). 28034880
FGFR3 N540K Advanced Solid Tumor sensitive Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited proliferation of transformed cells over expressing FGFR3 N540K in culture (PMID: 28034880). 28034880
FGFR3 N540K Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 N540K in culture (PMID: 28034880). 28034880
FGFR3 N540K Advanced Solid Tumor decreased response Zoligratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 N540K demonstrated reduced sensitivity to Debio 1347 in culture (PMID: 28034880). 28034880
FGFR3 N540K Advanced Solid Tumor sensitive Erdafitinib Preclinical - Cell culture Actionable In a preclinicl study, Balversa (erdafitinib) inhibited proliferation of transformed cells expressing FGFR3 N540K in culture (PMID: 26992226). 26992226
FGFR3 N540K Advanced Solid Tumor decreased response LY2874455 Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 N540K demonstrated reduced sensitivity to LY2874455 in culture (PMID: 28034880). 28034880
FGFR3 N540K Advanced Solid Tumor decreased response FIIN-2 Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 N540K demonstrated reduced sensitivity to FIIN-2 in culture (PMID: 28034880). 28034880
FGFR3 D788N head and neck squamous cell carcinoma no benefit Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) inhibited proliferation of a head and neck squamous cell carcinoma cell line expressing FGFR3 D788N in culture, however, cells expressing FGFR3 D788N did not demonstrate increased sensitivity to Truseltiq (infigratinib) compared to cells expressing wild-type FGFR3 (PMID: 27053219). 27053219
FGFR3 S131L head and neck squamous cell carcinoma decreased response Infigratinib Preclinical - Cell culture Actionable In a preclinical study, a head and neck squamous cell carcinoma cell line expressing FGFR3 S131L demonstrated decreased sensitivity to Truseltiq (infigratinib) compared to cells expressing wild-type FGFR3 in culture (PMID: 27053219). 27053219
FGFR3 L608V Advanced Solid Tumor decreased response Fexagratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 L608V demonstrated reduced sensitivity to AZD4547 in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor decreased response Infigratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 L608V demonstrated reduced sensitivity to Truseltiq (infigratinib) in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor sensitive Dovitinib Preclinical - Cell culture Actionable In a preclinical study, Dovitinib (TKI258) inhibited proliferation of transformed cells over expressing FGFR3 L608V in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor sensitive Ponatinib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) inhibited proliferation of transformed cells over expressing FGFR3 L608V in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor decreased response Zoligratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 L608V demonstrated reduced sensitivity to Debio 1347 in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor sensitive LY2874455 Preclinical - Cell culture Actionable In a preclinical study, LY2874455 inhibited proliferation of transformed cells over expressing FGFR3 L608V in culture (PMID: 28034880). 28034880
FGFR3 L608V Advanced Solid Tumor sensitive FIIN-2 Preclinical - Cell culture Actionable In a preclinical study, FIIN-2 inhibited proliferation of transformed cells over expressing FGFR3 L608V in culture (PMID: 28034880). 28034880
FGFR3 V555L transitional cell carcinoma resistant Infigratinib Case Reports/Case Series Actionable In a clinical study, two patients with metastatic urothelial carcinoma progressed while being treated with Truseltiq (infigratinib) and subsequent cell-free DNA testing revealed FGFR3 V555L (also corresponds to V443L) (PMID: 29848605). 29848605
FGFR3 V555L Advanced Solid Tumor decreased response Infigratinib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing FGFR3 V555L demonstrated reduced sensitivity to Truseltiq (infigratinib) in culture (PMID: 28034880). 28034880
FGFR3 G372C Advanced Solid Tumor sensitive Vofatamab Preclinical - Cell culture Actionable In a preclinical study, Vofatamab (B-701) inhibited ligand-dependent proliferation in cells expressing FGFR3 G372C (PMID: 19381019). 19381019
FGFR3 D764H head and neck squamous cell carcinoma no benefit Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) inhibited proliferation of a head and neck squamous cell carcinoma cell line expressing FGFR3 D764H in culture, however, cells expressing FGFR3 D764H did not demonstrate increased sensitivity to Truseltiq (infigratinib) compared to cells expressing wild-type FGFR3 (PMID: 27053219). 27053219
FGFR3 P573S lung non-small cell carcinoma no benefit Fexagratinib Case Reports/Case Series Actionable In a Phase II trial (NLMT), AZD4547 treatment did not result in a confirmed response or durable clinical benefit in a patient with non-small cell lung cancer harboring FGFR3 P575S (corresponds to P573S in the canonical isoform (PMID: 32669708, NCT02664935). 32669708
FGFR3 S756P lung non-small cell carcinoma no benefit Fexagratinib Case Reports/Case Series Actionable In a Phase II trial (NLMT), AZD4547 treatment did not result in a confirmed response or durable clinical benefit in a patient with non-small cell lung cancer harboring FGFR3 S758P (corresponds to S756P in the canonical isoform (PMID: 32669708, NCT02664935). 32669708
FGFR3 G380E Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380E were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G380E Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380E were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 G380E Advanced Solid Tumor predicted - sensitive Erdafitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380E were sensitive to treatment with Balversa (erdafitinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 G380E Advanced Solid Tumor predicted - sensitive Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 G380E were sensitive to treatment with Pemazyre (pemigatinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 R248_S249insC Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 R248_S249insC were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 R248_S249insC Advanced Solid Tumor predicted - resistant E7090 Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 R248_S249insC were resistant to treatment with E7090 in culture (PMID: 34272467). 34272467
FGFR3 H284fs Advanced Solid Tumor predicted - sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 H284fs (FGFR3 H284fs*10) were sensitive to treatment with Truseltiq (infigratinib) in culture, demonstrating reduced cell viability (PMID: 34272467). 34272467
FGFR3 H284fs Advanced Solid Tumor predicted - resistant Dovitinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 H284fs (FGFR3 H284fs*10) were resistant to treatment with Dovitinib (TKI258) in culture (PMID: 34272467). 34272467
FGFR3 H284fs Advanced Solid Tumor predicted - resistant Pemigatinib Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 H284fs (FGFR3 H284fs*10) were resistant to treatment with Pemazyre (pemigatinib) in culture (PMID: 34272467). 34272467
FGFR3 H284fs Advanced Solid Tumor predicted - resistant E7090 Preclinical - Cell culture Actionable In a preclinical study, cells expressing FGFR3 H284fs (FGFR3 H284fs*10) were resistant to treatment with E7090 in culture (PMID: 34272467). 34272467
FGFR3 N542K Advanced Solid Tumor sensitive Infigratinib Preclinical - Cell culture Actionable In a preclinical study, Truseltiq (infigratinib) decreased viability of a cell line expressing FGFR3 N542K in culture (PMID: 32370101). 32370101
FGFR3 N542K Advanced Solid Tumor sensitive Dasatinib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Sprycel (dasatinib) treatment inhibited viability of a cell line expressing FGFR3 N542K in culture (PMID: 32370101). 32370101