PDGFRB platelet derived growth factor receptor beta
ENSG00000113721 · protein coding · Open Targets profile
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. {ECO:0000269|PubMed:11297552, ECO:0000269|PubMed:11331881, ECO:0000269|PubMed:1314164, ECO:0000269|PubMed:1396585, ECO:0000269|PubMed:1653029, ECO:0000269|PubMed:1709159, ECO:0000269|PubMed:1846866, ECO:0000269|PubMed:20494825, ECO:0000269|PubMed:20529858, ECO:0000269|PubMed:21098708, ECO:0000269|PubMed:21679854, ECO:0000269|PubMed:21733313, ECO:0000269|PubMed:2554309, ECO:0000269|PubMed:26599395, ECO:0000269|PubMed:2835772, ECO:0000269|PubMed:2850496, ECO:0000269|PubMed:7685273, ECO:0000269|PubMed:7691811, ECO:0000269|PubMed:7692233, ECO:0000269|PubMed:8195171}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, High-Quality Ligand, Druggable Family
- Antibody
- Advanced Clinical, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Half-life Data, Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.3495199978351593 (upper bound 0.45208001136779785). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 400 registry trials, updated in the last three years, of the most advanced drugs against PDGFRB (nintedanib esylate, tivozanib, cediranib, midostaurin, pazopanib, masitinib, sorafenib, quizartinib, sunitinib, regorafenib, imatinib, nintedanib), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Joyce F LiuNRG Oncology · 3 of 3 trials Phase 2+Ph3NCT02446600 · Testing the Use of A Single Drug (Olaparib) or the Combination of Two Drugs (Cediranib and Olaparib) Compared to the Usual Chemotherapy for Women With Platinum Sensitive Ovarian, Fallopian Tube, or Primary Peritoneal Cancerbrief
- Musa YilmazM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+Ph1/Ph2NCT04128748 · Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndromebrief
- Nicolas PenelCentre Oscar Lambret · 3 of 3 trials Phase 2+
- Jean-Yves BLAYCentre Leon Berard · 2 of 3 trials Phase 2+Ph2NCT02029001 · Adapting Treatment to the Tumor Molecular Alterations for Patients With Advanced Solid Tumors: MyOwnSpecificTreatmentbrief
- Amado ZuritaM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Emmanuelle SAMALINCRLC Val d'Aurelle-Paul Lamarque · 2 of 2 trials Phase 2+
- Eric JonaschM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Eric ShermanMemorial Sloan Kettering Cancer Center · 2 of 2 trials Phase 2+
- Geneviève CheneUniversity Hospital, Bordeaux, France · 2 of 2 trials Phase 2+
- Jessica GartrellSt. Jude Children's Research Hospital · 2 of 2 trials Phase 2+
- Joseph W KimYale University Cancer Center LAO · 2 of 2 trials Phase 2+
- Juan BerguaHospital San Pedro Alcántara · 2 of 2 trials Phase 2+
- Jung-min LeeNRG Oncology · 2 of 2 trials Phase 2+
- Kanwal RaghavM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Lillian SiuPrincess Margaret Cancer Centre · 2 of 2 trials Phase 2+
Registry trials of nintedanib esylate, tivozanib, cediranib, midostaurin, pazopanib, masitinib, sorafenib, quizartinib, sunitinib, regorafenib, imatinib, nintedanib updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 279 of 385 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 15 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
537 trials of drugs against PDGFRB were stopped early; 17 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of nintedanib esylate, tivozanib, cediranib, midostaurin, pazopanib, masitinib, sorafenib, quizartinib, sunitinib, regorafenib, imatinib, nintedanib, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 537.
- terminated · 2026-11-03 · Peking University Cancer Hospital & Institute · regorafenib“The study was stopped early due to lack of efficacy.”
- suspended · 2026-10-20 · City of Hope Medical Center · Regorafenib“resources”
- terminated · 2026-10-05 · Children's Oncology Group · Regorafenib“Drug supply issues”
- withdrawn · 2026-10-01 · City of Hope Medical Center · Tivozanib“Study Abandoned withdrawn”
- terminated · 2026-08-26 · Bristol-Myers Squibb · RegorafenibNo reason given
- terminated · 2026-08-25 · Association of Urologic Oncology (AUO) · SunitinibNo reason given
- terminated · 2026-07-06 · Syndax Pharmaceuticals · matched by the registry's drug synonyms“Study was terminated by sponsor after Phase 1b as pre-defined signals of efficacy were not met in the Phase 1b dose expansion cohort.”
- withdrawn · 2026-06-15 · University of Miami · Regorafenib“Investigator decision”
- terminated · 2026-05-29 · Sarit Assouline · Imatinib“Insufficient patient accrual”
- terminated · 2026-05-20 · Massachusetts General Hospital · Tivozanib“Slow accrual”
- suspended · 2026-05-06 · AB Science · Masitinib (4.5)“The study has been suspended but may start again in the future.”
- terminated · 2026-04-14 · Institut Claudius Regaud · Pazopanib“Slow recruitment rate”
- terminated · 2026-03-25 · University of Maryland, Baltimore · Imatinib“The Principal Investigator left the institution. The study was halted prematurely.”
- terminated · 2026-03-13 · Centre Oscar Lambret · Imatinib“Study stopped at the end of the first step”
- withdrawn · 2026-03-11 · M.D. Anderson Cancer Center · Regorafenib“0 participants enrolled”
- terminated · 2026-03-09 · Translational Research in Oncology · Regorafenib in combination with pembrolizumab“The trial ended earlier than planned due to slow enrolment, which has made it unfeasible to achieve the target sample size within a reasonable timeframe. Consequently, a business decision has been made to discontinue funding for the trial.”
- terminated · 2026-03-05 · Wake Forest University Health Sciences · Regorafenib“Slow accruals”
- withdrawn · 2026-02-25 · M.D. Anderson Cancer Center · Regorafenib“0 participants enrolled”
- terminated · 2026-01-29 · Indiana University · Imatinib Mesylate“low accrual”
- terminated · 2026-01-20 · Anwaar Saeed · Regorafenib“Terminated per directive from Bayer due to funding.”
- terminated · 2026-01-13 · Novartis Pharmaceuticals · matched by the registry's drug synonyms“Sponsor Decision”
- suspended · 2025-10-23 · Mirror Biologics, Inc. · Sorafenib“Sponsor unable to fund”
- terminated · 2025-09-19 · European Organisation for Research and Treatment of Cancer - EORTC · Nintedanib“lack of scientific relevance (change in the treatment landscape) and poor recruitment”
- terminated · 2025-09-02 · McGill University Health Centre/Research Institute of the McGill University Health Centre · Nintedanib 150 milligrams [Ofev]“Slow recruitment”
- terminated · 2025-08-22 · OHSU Knight Cancer Institute · imatinib mesylateNo reason given
Paediatric incentives and obligations for PDGFRB's drugs
For nintedanib esylate, tivozanib, cediranib, midostaurin, pazopanib, masitinib, sorafenib, quizartinib, sunitinib, regorafenib, imatinib, nintedanib: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: yes · PDGFRB is on the list. Membership alone neither requires nor waives a pediatric study.Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMA/PE/0000182170 (regorafenib, Treatment of all conditions contained in the category of malignant neoplasms (except haematopoietic and lymphoid tissue)): PM: decision on the application for modification of an agreed PIP, 5 Dec 2024; EMEA-000780-PIP01-09-M08 (Midostaurin, Treatment of acute myeloid leukaemia;Treatment of malignant mastocytosis;Treatment of mast cell leukaemia): PM: decision on the application for modification of an agreed PIP, 16 Aug 2024; EMEA-001821-PIP01-15-M08 (quizartinib, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 14 Jun 2024; EMEA-001006-PIP05-18-M01 (nintedanib, Treatment of fibrosing Interstitial Lung Diseases (ILD)): PM: decision on the application for modification of an agreed PIP, 29 Oct 2021; compliance positive 22 Jul 2022; +12 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/16/1722 positive 29 Aug 2016: Treatment of amyotrophic lateral sclerosis; EU/3/13/1200 expired 13 Nov 2013: Treatment of papillary thyroid cancer; EU/3/13/1199 expired 13 Nov 2013: Treatment of follicular thyroid cancer; EU/3/21/2449 positive 21 Jun 2021: Treatment of pulmonary arterial hypertension; +20 moreRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Glivec: authorised since 7 Nov 2001; Ofev: authorised since 14 Jan 2015; Sunitinib Accord: authorised since 11 Feb 2021; Vanflyta: authorised since 6 Nov 2023; +24 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: yes · Imatinib Mesylate [Gleevec] &, 25 Jan 2013: Pediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy; Imatinib Mesylate [Gleevec] &, 27 Sept 2006: Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phaseRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
- Registered studies open to children: yes · 261 interventional studies of nintedanib esylate, tivozanib, cediranib, midostaurin, pazopanib, masitinib, sorafenib, quizartinib, sunitinib, regorafenib, imatinib, nintedanib include patients under 18; 49 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 45 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.