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Protein / target

Platelet-derived growth factor receptor beta

Encoded byPDGFRBP09619Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
19
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Platelet-derived growth factor beta-receptor

Strongest disease association

Infantile myofibromatosis

Via encoding gene PDGFRB · Genetic literature evidence · score 0.87

Therapeutic position

Established drug target

Small molecules and antibodies

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 overview

Tyrosine-protein kinase that acts as a cell-surface receptor for homodimeric PDGFB and PDGFD and for heterodimers formed by PDGFA and PDGFB, and plays an essential role in the regulation of embryonic development, cell proliferation, survival, differentiation, chemotaxis and migration.

View complete UniProt function annotation

Tyrosine-protein kinase that acts as a cell-surface receptor for homodimeric PDGFB and PDGFD and for heterodimers formed by PDGFA and PDGFB, and plays an essential role in the regulation of embryonic development, cell proliferation, survival, differentiation, chemotaxis and migration. Plays an essential role in blood vessel development by promoting proliferation, migration and recruitment of pericytes and smooth muscle cells to endothelial cells. Plays a role in the migration of vascular smooth muscle cells and the formation of neointima at vascular injury sites. Required for normal development of the cardiovascular system. Required for normal recruitment of pericytes (mesangial cells) in the kidney glomerulus, and for normal formation of a branched network of capillaries in kidney glomeruli. Promotes rearrangement of the actin cytoskeleton and the formation of membrane ruffles. Binding of its cognate ligands - homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFD -leads to the activation of several signaling cascades; the response depends on the nature of the bound ligand and is modulated by the formation of heterodimers between PDGFRA and PDGFRB. Phosphorylates PLCG1, PIK3R1, PTPN11, RASA1/GAP, CBL, SHC1 and NCK1. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate, mobilization of cytosolic Ca(2+) and the activation of protein kinase C. Phosphorylation of PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, leads to the activation of the AKT1 signaling pathway. Phosphorylation of SHC1, or of the C-terminus of PTPN11, creates a binding site for GRB2, resulting in the activation of HRAS, RAF1 and down-stream MAP kinases, including MAPK1/ERK2 and/or MAPK3/ERK1. Promotes phosphorylation and activation of SRC family kinases. Promotes phosphorylation of PDCD6IP/ALIX and STAM. Receptor signaling is down-regulated by protein phosphatases that dephosphorylate the receptor and its down-stream effectors, and by rapid internalization of the activated receptor

Subcellular location

Cell membraneCytoplasmic vesicleLysosome lumen
Domains and Gene Ontology detail (62)

Domains & features

Ig-like C2-type 1Ig-like C2-type 2Ig-like C2-type 3Ig-like C2-type 4Ig-like C2-type 5Protein kinase

Gene Ontology

  • Capical plasma membrane
  • Ccytoplasm
  • Ccytoplasmic vesicle
  • Cfocal adhesion
  • Clysosomal lumen
  • Cmembrane
  • Cnucleus
  • Cplasma membrane
  • Creceptor complex
  • FATP binding
  • Fenzyme binding
  • FGTPase activator activity

1106 aa · 124 kDa · 2 isoforms

Biological roles

Reactome v97

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

Cell proliferation & survivalGO · ReactomeCell migrationUniProt · GOGrowth-factor signallingGOReceptor tyrosine kinase signallingGOOncogenic signallingReactome
View supporting evidence

Cell proliferation & survival

  • ·positive regulation of cell population proliferation
  • ·positive regulation of cell proliferation by VEGF-activated platelet derived growth fact…
  • ·PIP3 activates AKT signaling
  • ·PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling

Cell migration

  • ·Tyrosine-protein kinase that acts as a cell-surface receptor for homodimeric PDGFB and P…
  • ·cell chemotaxis
  • ·cell migration involved in coronary angiogenesis
  • ·cell migration involved in vasculogenesis

Growth-factor signalling

  • ·platelet-derived growth factor binding
  • ·vascular endothelial growth factor binding
  • ·platelet-derived growth factor receptor signaling pathway
  • ·positive regulation of cell proliferation by VEGF-activated platelet derived growth fact…

Receptor tyrosine kinase signalling

  • ·cell surface receptor protein tyrosine kinase signaling pathway

Oncogenic signalling

  • ·Constitutive Signaling by Aberrant PI3K in Cancer
View underlying pathways (6)

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.

PDGFAPTPN11PDGFBPIK3R1PDGFDPLCG1GRB2SRCPDGFCEGFRPDGFRB

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.

Approved medicines with mapped indications

4 medicines · 11 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Gastrointestinal Stromal Tumors2 medicines
Carcinoma, Renal Cell1 medicine
Colorectal Neoplasms1 medicine
Leukemia, Myelogenous, Chronic, BCR-ABL Positive1 medicine
Leukemia, Myeloid, Acute1 medicine
Mastocytosis1 medicine
Mastocytosis, Systemic1 medicine
Myelodysplastic-Myeloproliferative Diseases1 medicine
Neuroendocrine Tumors1 medicine
Precursor Cell Lymphoblastic Leukemia-Lymphoma1 medicine
Broader indication categories (1)

Broad umbrella indications (e.g. “Neoplasms”). Shown here because every medicine also appears under a more specific disease above — kept for completeness, de-emphasised for clarity.

19 medicines meet Open Targets' target-level approved-medicine definition; the 4 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

5

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

Imatinib Mesylate
ApprovedInhibitor

Platelet-derived growth factor receptor beta inhibitor

Indicated for Gastrointestinal Stromal Tumors, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic-Myeloproliferative Diseases, Precursor Cell Lymphoblastic Leukemia-Lymphoma

Direct interaction with this protein · 1 of 4 recorded protein targets

sunitinib
ApprovedInhibitor

Platelet-derived growth factor receptor beta inhibitor

Indicated for Carcinoma, Renal Cell, Gastrointestinal Stromal Tumors, Neuroendocrine Tumors, Neoplasms

Direct interaction with this protein · 1 of 9 recorded protein targets — broad pharmacology

catequentinib
Phase 3Inhibitor

Platelet-derived growth factor receptor beta inhibitor

Direct interaction with this protein · 1 of 4 recorded protein targets

regorafenib
ApprovedInhibitor

Platelet-derived growth factor receptor inhibitor

Indicated for Colorectal Neoplasms, Neoplasms

Acts on a complex — shared with PDGFRA · 1 of 18 recorded protein targets — broad pharmacology

midostaurin
ApprovedInhibitor

Platelet-derived growth factor receptor inhibitor

Indicated for Leukemia, Myeloid, Acute, Mastocytosis, Mastocytosis, Systemic, Neoplasms

Acts on a complex — shared with PDGFRA · 1 of 16 recorded protein targets — broad pharmacology

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

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene PDGFRB

Gene-level evidence surfaced through the gene PDGFRBthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Infantile myofibromatosis
0.93Well supported

Genetic evidence dominant · Open Targets 0.75

Myofibromatosis, infantile, 1
0.92Well supported

Genetic evidence dominant · Open Targets 0.76

Bilateral striopallidodentate calcinosis
0.91Well supported

Genetic evidence dominant · Open Targets 0.77

Leukemia, Myeloid, Acute
0.84Well supported

Clinical evidence dominant · Open Targets 0.65

Carcinoma, Hepatocellular
0.83Well supported

Clinical evidence dominant · Open Targets 0.67

View evidence synthesis (5)
Infantile myofibromatosisWell supported
0.93
agreement 0.831.00
Genetic54%Somatic mutation37%Animal model7%Literature3%Genetic literaturedup

Open Targets aggregate 0.75 · 4 independent evidence families · 1 not counted as duplicate

Myofibromatosis, infantile, 1Well supported
0.92
agreement 0.811.00
Genetic62%Somatic mutation38%Genetic literaturedup

Open Targets aggregate 0.76 · 2 independent evidence families · 1 not counted as duplicate

Bilateral striopallidodentate calcinosisWell supported
0.91
agreement 0.801.00
Genetic67%Animal model16%Somatic mutation11%Literature6%Genetic literaturedup

Open Targets aggregate 0.77 · 4 independent evidence families · 1 not counted as duplicate

Leukemia, Myeloid, AcuteWell supported
0.84
agreement 0.750.94
Clinical53%Somatic mutation23%Genetic literature19%Literature5%

Open Targets aggregate 0.65 · 4 independent evidence families

Carcinoma, HepatocellularWell supported
0.83
agreement 0.710.95
Clinical63%Somatic mutation26%Literature11%

Open Targets aggregate 0.67 · 3 independent evidence families

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. 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 per-type scores above show the calculation.

Show all associations
Bilateral striopallidodentate calcinosis0.77
Myofibromatosis, infantile, 10.76
Infantile myofibromatosis0.75
Skeletal overgrowth-craniofacial dysmorphism-hyperelastic skin-white matter lesions syndrome0.74
Acroosteolysis-keloid-like lesions-premature aging syndrome0.74
Carcinoma, Hepatocellular0.67
Gastrointestinal Stromal Tumors0.67
chronic myelogenous leukemia, BCR-ABL1 positive0.65
Leukemia, Myeloid, Acute0.65

Drug development

45 compounds recorded · 19 approved · 26 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 5 drugs that target this protein in Forefront's canonical graph (4 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (10)
NINTEDANIB ESYLATEApproval
TIVOZANIBApproval
TANDUTINIBPhase 2
CEDIRANIBApproval
BECAPLERMINApproval
RINUCUMABPhase 2
FORETINIBPhase 2
SUNITINIBApproval
RG-1530Phase 1
MIDOSTAURINApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and High-Quality Ligand support this modality.

AntibodiesStrong

Advanced Clinical and GO CC high conf support this modality.

Protein degradersEmerging

Feasibility evidence (uniprot ubiquitination and database ubiquitination) — no clinical-stage drug of this modality recorded.

Other modalitiesStrong

Approved Drug support this modality.

View underlying tractability evidence (12)
SM · Approved DrugSM · High-Quality LigandSM · Druggable FamilyAB · Advanced ClinicalAB · GO CC high confAB · UniProt loc med confAB · UniProt SigP or TMHMMPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule BinderOC · Approved Drug

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

heart diseaseForce et al. (2011)

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

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. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (26)

RECRUITING · via catequentinib · NCT06970145

ACTIVE_NOT_RECRUITING · via regorafenib · NCT05395741

ACTIVE_NOT_RECRUITING · via sunitinib · NCT02465060

ClinicalTrials.gov via the drug-target graph.

What's happening now

12

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for 3 drugs that target this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Label change2026-07-13

    Label change: IMATINIB MESYLATE (NDA021588)

    fda · regulatory · fda · via Imatinib Mesylate

  2. Label change2026-01-14

    Label change: IMATINIB MESYLATE (NDA021588)

    fda · regulatory · fda · via Imatinib Mesylate

  3. Label change2024-12-09

    Label change: IMATINIB (ANDA204644)

    fda · regulatory · fda · via Imatinib Mesylate

  4. 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

  5. New publication2024-05-03
    Machine-learning and mechanistic modeling of metastatic breast cancer after neoadjuvant treatment.

    PLoS computational biology · 2024 · 10 citations · Europe PMC · via sunitinib

  6. Supplemental approval2024-03-01

    Supplemental approval: IMATINIB MESYLATE (NDA021588)

    fda · regulatory · fda · via Imatinib Mesylate

  7. New publication2024-02-27
    KIT mutations and expression: current knowledge and new insights for overcoming IM resistance in GIST.

    Cell communication and signaling : CCS · 2024 · 28 citations · Europe PMC · via Imatinib Mesylate

  8. New publication2024-01-05
    Genomic profiling in GIST: Implications in clinical outcome and future challenges.

    Neoplasia (New York, N.Y.) · 2024 · 20 citations · Europe PMC · via sunitinib

  9. Label change2024-01-05

    Label change: IMATINIB (ANDA204644)

    fda · regulatory · fda · via Imatinib Mesylate

  10. Label change2024-01-05

    Label change: IMATINIB (ANDA204644)

    fda · regulatory · fda · via Imatinib Mesylate

  11. Label change2024-01-05

    Label change: IMATINIB (ANDA204644)

    fda · regulatory · fda · via Imatinib Mesylate

  12. Label change2024-01-05

    Label change: IMATINIB (ANDA204644)

    fda · regulatory · fda · via Imatinib Mesylate

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.