FGFR2

Chr 10AD

fibroblast growth factor receptor 2

Also known as: BBDS, BEK, BFR-1, CD332, CEK3, CFD1, ECT1, JWS

This gene encodes a transmembrane tyrosine kinase receptor that binds fibroblast growth factors and activates downstream signaling cascades that regulate cell proliferation and differentiation. Mutations cause multiple autosomal dominant craniosynostosis syndromes including Apert, Crouzon, Pfeiffer, Jackson-Weiss, Beare-Stevenson, and Saethre-Chotzen syndromes, as well as other skeletal dysplasias like Antley-Bixler syndrome and bent bone dysplasia. The pathogenic mutations typically result in gain-of-function effects that lead to premature fusion of cranial sutures and associated craniofacial abnormalities.

OMIMResearchSummary from RefSeq, OMIM, UniProt
GOFmechanismADLOEUF 0.2714 OMIM phenotypes
Clinical SummaryFGFR2
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Gene-Disease Validity (ClinGen)
Apert syndrome · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

6 total gene-disease associations curated

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.27LOEUF
pLI 0.997
Z-score 5.29
OE 0.14 (0.070.27)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
2.40Z-score
OE missense 0.69 (0.630.75)
323 obs / 469.5 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.14 (0.070.27)
00.351.4
Missense OE0.69 (0.630.75)
00.61.4
Synonymous OE1.11
01.21.6
LoF obs/exp: 6 / 43.8Missense obs/exp: 323 / 469.5Syn Z: -1.17
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveFGFR2-related lacrimo-auriculo-dento-digital syndrome (LADD)GOFAD
definitiveFGFR2-related Beare-Stevenson cutis gyrata syndromeGOFAD
definitiveFGFR2-related Jackson-Weiss syndromeGOFAD
strongFGFR2-related Antley-Bixler syndromeOTHERAD
definitiveFGFR2-related Crouzon syndromeGOFAD
definitiveFGFR2-related Apert SyndromeGOFAD
definitiveFGFR2-related Pfeiffer syndromeGOFAD
DN
0.5476th %ile
GOF
0.7029th %ile
LOF
0.59top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function and gain-of-function). The Badonyi & Marsh model scores gain-of-function highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports loss-of-function (haploinsufficiency). Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOF1 literature citation · LOEUF 0.27
GOFprediction above median · 1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

GOFGain-of-function missense mutations in FGF receptor 2 (FGFR2) are responsible for a variety of craniosynostosis syndromes including Apert syndrome (AS), Pfeiffer syndrome (PS) and Crouzon syndrome (CS).PMID:15282208
LOFPfeiffer syndrome caused by haploinsufficient mutation of FGFR2.PMID:10731087

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

FGFR2 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Rare Malignant Neoplasm

Marker Assisted Selective ThErapy in Rare Cancers: Knowledge Database Establishing registrY Asia

RECRUITING
NCT05217407National Cancer Center, JapanStarted 2021-11-30
Genomic sequence
Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR)

RECRUITING
NCT03297606Phase PHASE2Canadian Cancer Trials GroupStarted 2018-03-23
OlaparibDasatinibNivolumab plus Ipilimumab
Biliary Tract Cancer

A Post-marketing Observational Study of Tasfygo in Participants With Unresectable Biliary Tract Cancer With Fibroblast Growth Factor Receptor 2 (FGFR2) Fusion Gene Positivity Who Progressed After Chemotherapy

RECRUITING
NCT06793709Eisai Co., Ltd.Started 2025-07-10
No Intervention
Intrahepatic Cholangiocarcinoma (Icc)CholangiocarcinomaOther Solid Tumors, Adult

A Study of an FGFR2/3 Inhibitor (CGT4859) in Patients With Cholangiocarcinoma and Other Advanced Solid Tumors

ACTIVE NOT RECRUITING
NCT06777316Phase PHASE1, PHASE2Cogent Biosciences, Inc.Started 2025-01-22
CGT4859
Locally Advanced Bladder Urothelial CarcinomaLocally Advanced Renal Pelvis Urothelial CarcinomaLocally Advanced Ureter Urothelial Carcinoma

Testing Combination Erdafitinib and Enfortumab Vedotin in Metastatic Bladder Cancer After Treatment With Chemotherapy and Immunotherapy

ACTIVE NOT RECRUITING
NCT04963153Phase PHASE1National Cancer Institute (NCI)Started 2022-07-07
Biospecimen CollectionBone ScanComputed Tomography
Advanced Malignant Solid NeoplasmAnn Arbor Stage III Non-Hodgkin LymphomaAnn Arbor Stage IV Non-Hodgkin Lymphoma

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

ACTIVE NOT RECRUITING
NCT03155620Phase PHASE2National Cancer Institute (NCI)Started 2017-07-31
Biopsy ProcedureBiospecimen CollectionBone Marrow Aspiration and Biopsy
Intrahepatic CholangiocarcinomaFGFR2 Gene RearrangementFGFR2 Gene Mutation

A Phase II Study of Pemigatinib Plus Durvalumab in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients With FGFR-2 Fusion or Rearrangement

RECRUITING
NCT06728410Phase PHASE2Mehmet AkceStarted 2026-01-27
PemigatinibDurvalumab
Gastric and Cardia Adenocarcinomas

Biomoleculars Markers of Sensitivity to Pre- and Post-operative Chemotherapy of Gastric and Cardia Adenocarcinomas: a Pilot Study

RECRUITING
NCT02491840Phase NAUniversity Hospital, Strasbourg, FranceStarted 2016-05
Biopsy
Intrahepatic Cholangiocarcinoma (Icc)Advanced Cholangiocarcinoma

Efficacy and Safety of TT-00420 (Tinengotinib) Tablets Versus Chemotherapy in Patients With Advanced Intrahepatic Cholangiocarcinoma Harboring FGFR2 Fusions/Rearrangements or Mutations

NOT YET RECRUITING
NCT07328919Phase PHASE3TransThera Sciences (Nanjing), Inc.Started 2026-01
TT-00420 (tinengotinib)Oxaliplatin, fluorouracil, calcium folinate, irinotecan, capecitabine
Locally Advanced CholangiocarcinomaIntrahepatic CholangiocarcinomaSolid Tumor

Safety and Anti-Tumor Activity of TYRA-200 in Advanced Cholangiocarcinoma With Activating FGFR2 Gene Alterations

RECRUITING
NCT06160752Phase PHASE1Tyra Biosciences, IncStarted 2023-11-22
Phase 1 Part A - dose escalation TYRA-200 taken once daily by mouth in 28-day cyclesPhase 1 Part B - dose expansion TYRA-200 taken once daily by mouth in 28-day cycles
HER2-Gastric/Gastroesophageal Junction CancerUrothelial CarcinomaNon-Small Cell Lung Cancer

A Phase 2 Clinical Study of ABSK061 and ABSK043

RECRUITING
NCT06632262Phase PHASE2Abbisko Therapeutics Co, LtdStarted 2024-11-13
ABSK061 + ABSK043ABSK061+ABSK043 in combination with CAPOX
Advanced CholangiocarcinomaFGFR2 FusionsGene Rearrangement

Study of Futibatinib in Patients With Advanced Cholangiocarcinoma With FGFR2 Fusion or Rearrangement

RECRUITING
NCT05727176Phase PHASE2Taiho Oncology, Inc.Started 2023-07-05
TAS-120
Clinical Literature
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Key Publications
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