Protein / target

Fibroblast growth factor receptor 1

FGFR1P11362Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
12
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Fibroblast growth factor receptor activity

Primary system

Nervous system

Strongest disease association

hypogonadotropic hypogonadism 2 with or without anosmia

Genetic evidence · score 0.98

Therapeutic maturity

Clinically validated target

12 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

12 approved · 14 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of embryonic development, cell proliferation, differentiation and migration. Required for normal mesoderm patterning and correct axial organization during embryonic development, normal skeletogenesis and normal development of the gonadotropin-releasing hormone (GnRH) neuronal system. Phosphorylates PLCG1, FRS2, GAB1 and SHB. Ligand binding leads to the activation of several signaling cascades. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate. Phosphorylation of FRS2 triggers recruitment of GRB2, GAB1, PIK3R1 and SOS1, and mediates activation of RAS, MAPK1/ERK2, MAPK3/ERK1 and the MAP kinase signaling pathway, as well as of the AKT1 signaling pathway. Promotes phosphorylation of SHC1, STAT1 and PTPN11/SHP2. In the nucleus, enhances RPS6KA1 and CREB1 activity and contributes to the regulation of transcription. FGFR1 signaling is down-regulated by IL17RD/SEF, and by FGFR1 ubiquitination, internalization and degradation

Subcellular location

Cell membraneNucleusCytoplasm, cytosolCytoplasmic vesicle
Domains and Gene Ontology detail (56)

Domains & features

Ig-like C2-type 1Ig-like C2-type 2Ig-like C2-type 3Protein kinase

Gene Ontology

  • Ccytoplasmic vesicle
  • Ccytosol
  • Cextracellular region
  • Cglutamatergic synapse
  • Cmembrane
  • Cnucleus
  • Cplasma membrane
  • Cpostsynapse
  • Creceptor complex
  • FATP binding
  • Ffibroblast growth factor binding
  • Ffibroblast growth factor receptor activity

822 aa · 92 kDa · 21 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Kinase signallingUniProt · GOTranscriptional regulationUniProt · GOSynaptic signallingGOExcitatory neurotransmissionGOMetabolic enzyme activityGO
View supporting evidence

Kinase signalling

  • ·Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth facto…
  • ·protein tyrosine kinase activity
  • ·peptidyl-tyrosine phosphorylation
  • ·positive regulation of MAP kinase activity

Transcriptional regulation

  • ·Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth facto…
  • ·DNA-templated transcription
  • ·negative regulation of transcription by RNA polymerase II

Synaptic signalling

  • ·glutamatergic synapse
  • ·postsynapse
  • ·regulation of postsynaptic density assembly

Excitatory neurotransmission

  • ·glutamatergic synapse

Metabolic enzyme activity

  • ·diphosphate metabolic process
View underlying pathways (22)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

FGF18FRS2FGF23FGF3FGF6FGF20FGF1KLFGF16FGF10FGFR1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

3

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

regorafenib
ApprovedInhibitor

Fibroblast growth factor receptor 1 inhibitor

Appears in clinical studies involving colorectal cancer, metastatic colorectal cancer, colorectal neoplasm, neoplasm

Direct interaction with this protein · 1 of 18 recorded protein targets — broad pharmacology

erdafitinib
ApprovedInhibitor

Fibroblast growth factor receptor inhibitor

Appears in clinical studies involving cancer, urothelial carcinoma, urinary bladder carcinoma, neoplasm

Acts on a complex — shared with FGFR3, FGFR4, FGFR2 · 1 of 4 recorded protein targets

futibatinib
ApprovedInhibitor

Fibroblast growth factor receptor inhibitor

Appears in clinical studies involving cancer, biliary tract cancer, intrahepatic cholangiocarcinoma, cholangiocarcinoma

Acts on a complex — shared with FGFR3, FGFR4, FGFR2 · 1 of 4 recorded protein targets

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

hypogonadotropic hypogonadism 2 with or without anosmia0.98

Genetic · overall 0.85

Kallmann syndrome0.94

Genetic literature · overall 0.74

Hartsfield-Bixler-Demyer syndrome0.91

Genetic · overall 0.83

hypogonadotropic hypogonadism0.87

Genetic literature · overall 0.77

Pfeiffer syndrome0.87

Genetic · overall 0.79

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

cancer0.93

Clinical · overall 0.75

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

osteoglophonic dysplasia0.77

Genetic

encephalocraniocutaneous lipomatosis0.76

Genetic

Jackson-Weiss syndrome0.72

Genetic

trigonocephaly 10.72

Genetic

Show all associations
hypogonadotropic hypogonadism 2 with or without anosmia0.85
Hartsfield-Bixler-Demyer syndrome0.83
Pfeiffer syndrome0.79
osteoglophonic dysplasia0.77
hypogonadotropic hypogonadism0.77
encephalocraniocutaneous lipomatosis0.76
cancer0.75
Kallmann syndrome0.74
Jackson-Weiss syndrome0.72
trigonocephaly 10.72

Open Targets ranks 4,766 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 26 total

TG100-801Phase 2

macular degeneration · age-related macular degeneration

E-7090Approval

cancer · biliary tract cancer · cholangiocarcinoma

NINTEDANIB ESYLATEApproval

idiopathic pulmonary fibrosis · systemic sclerosis · non-small cell lung carcinoma

PAZOPANIBApproval

renal cell carcinoma · neoplasm · renal cell carcinoma

FGFR INHIBITOR DEBIO 1347Phase 2

breast cancer

INFIGRATINIBApproval

cholangiocarcinoma · neoplasm · cancer

FEXAGRATINIBPhase 2

gastric adenocarcinoma · non-small cell lung carcinoma · breast cancer

SURUFATINIBApproval

neoplasm · neuroendocrine carcinoma · neuroendocrine neoplasm

ENMD-981693Phase 2

colorectal carcinoma · breast carcinoma · exocrine pancreatic carcinoma

LY-2874455Phase 2

gastric cancer · non-small cell lung carcinoma · acute myeloid leukemia

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC high confAB · UniProt loc med confAB · UniProt SigP or TMHMMPR · LiteraturePR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life Data

Safety liabilities

anaemiahypophosphataemiadecreased platelet countweight losskeratitisdecreased food intakeHyperphosphataemiaAST elevationsdry eyereduced sweet tastedermatologic toxicityregulation of catalytic activity

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ACTIVE_NOT_RECRUITING · via regorafenib · NCT05395741

TERMINATED · via regorafenib · NCT02402036

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

hypogonadotropic hypogonadism 2 with or without anosmiaWell supported
0.98
agreement 0.861.00
Genetic88%Animal model12%Literature1%Genetic literaturedup

Open Targets aggregate 0.85 · 3 independent evidence families · 1 not counted as duplicate

encephalocraniocutaneous lipomatosisWell supported
0.93
agreement 0.831.00
Genetic61%Somatic mutation29%Animal model7%Literature2%Genetic literaturedup

Open Targets aggregate 0.76 · 4 independent evidence families · 1 not counted as duplicate

Hartsfield-Bixler-Demyer syndromeWell supported
0.93
agreement 0.811.00
Genetic79%Animal model16%Literature5%Genetic literaturedup

Open Targets aggregate 0.83 · 3 independent evidence families · 1 not counted as duplicate

Pfeiffer syndromeWell supported
0.90
agreement 0.781.00
Genetic79%Animal model19%Literature2%Genetic literaturedup

Open Targets aggregate 0.79 · 3 independent evidence families · 1 not counted as duplicate

osteoglophonic dysplasiaWell supported
0.89
agreement 0.771.00
Genetic78%Animal model21%Literature2%Genetic literaturedup

Open Targets aggregate 0.77 · 3 independent evidence families · 1 not counted as duplicate

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

12

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 3 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. New publication2025-01-06
    Efficacy of pembrolizumab in microsatellite-stable, tumor mutational burden-high metastatic colorectal cancer: genomic signatures and clinical outcomes.

    ESMO open · 2025 · 10 citations · Europe PMC · via regorafenib

  2. Regulatory approval2024-08-22

    Approval: Balversa (EMA)

    ema · regulatory · ema · via erdafitinib

  3. New publication2024-06-04
    Lenvatinib Plus Pembrolizumab Versus Standard of Care for Previously Treated Metastatic Colorectal Cancer: Final Analysis of the Randomized, Open-Label, Phase III LEAP-017 Study.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024 · 69 citations · Europe PMC · via regorafenib

  4. New publication2023-11-01
    Clinical and Genomic Landscape of FGFR3-Altered Urothelial Carcinoma and Treatment Outcomes with Erdafitinib: A Real-World Experience.

    Clinical cancer research : an official journal of the American Association for Cancer Research · 2023 · 49 citations · Europe PMC · via erdafitinib

  5. Regulatory approval2023-07-04

    Approval: Lytgobi (EMA)

    ema · regulatory · ema · via futibatinib

  6. New publication2023-01-01
    Futibatinib for <i>FGFR2</i>-Rearranged Intrahepatic Cholangiocarcinoma.

    The New England journal of medicine · 2023 · 420 citations · Europe PMC · via futibatinib

  7. New publication2020-08-11
    Targeted therapy for hepatocellular carcinoma.

    Signal transduction and targeted therapy · 2020 · 565 citations · Europe PMC · via regorafenib

  8. New publication2019-11-04
    Molecular targeted and immune checkpoint therapy for advanced hepatocellular carcinoma.

    Journal of experimental & clinical cancer research : CR · 2019 · 162 citations · Europe PMC · via regorafenib

  9. New publication2019-07-01
    Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma.

    The New England journal of medicine · 2019 · 1,006 citations · Europe PMC · via erdafitinib

  10. New publication2019-04-23
    Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2019 · 199 citations · Europe PMC · via regorafenib

  11. New publication2016-12-06
    Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial.

    Lancet (London, England) · 2017 · 2,767 citations · Europe PMC · via regorafenib

  12. Regulatory approval2013-08-26

    Approval: Stivarga (EMA)

    ema · regulatory · ema · via regorafenib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.