PDGFRA platelet derived growth factor receptor alpha
ENSG00000134853 · protein coding · Open Targets profile
Tyrosine-protein kinase that acts as a cell-surface receptor for PDGFA, PDGFB and PDGFC and plays an essential role in the regulation of embryonic development, cell proliferation, survival and chemotaxis. Depending on the context, promotes or inhibits cell proliferation and cell migration. Plays an important role in the differentiation of bone marrow-derived mesenchymal stem cells. Required for normal skeleton development and cephalic closure during embryonic development. Required for normal development of the mucosa lining the gastrointestinal tract, and for recruitment of mesenchymal cells and normal development of intestinal villi. Plays a role in cell migration and chemotaxis in wound healing. Plays a role in platelet activation, secretion of agonists from platelet granules, and in thrombin-induced platelet aggregation. Binding of its cognate ligands - homodimeric PDGFA, homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFC -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 PIK3R1, PLCG1, and PTPN11. 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. Phosphorylates PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, and thereby mediates activation of the AKT1 signaling pathway. Mediates activation of HRAS and of the MAP kinases MAPK1/ERK2 and/or MAPK3/ERK1. Promotes activation of STAT family members STAT1, STAT3 and STAT5A and/or STAT5B. 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:10734113, ECO:0000269|PubMed:10947961, ECO:0000269|PubMed:11297552, ECO:0000269|PubMed:12522257, ECO:0000269|PubMed:1646396, ECO:0000269|PubMed:17087943, ECO:0000269|PubMed:1709159, ECO:0000269|PubMed:17141222, ECO:0000269|PubMed:20972453, ECO:0000269|PubMed:21224473, ECO:0000269|PubMed:21596750, ECO:0000269|PubMed:2554309, ECO:0000269|PubMed:8188664, ECO:0000269|PubMed:8760137, ECO:0000269|PubMed:8943348}.
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, Structure with Ligand, High-Quality Ligand, Druggable Family
- Antibody
- Approved Drug, UniProt loc high conf, GO CC high 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 0.20578999817371368 (upper bound 0.28742000460624695). 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 PDGFRA (cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinib), 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
- Zhimin ShaoFudan University · 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
- Alice P ChenNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Amado ZuritaM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Eric JonaschM.D. Anderson Cancer Center · 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+
- Lin ShenPeking University Cancer Hospital & Institute · 2 of 2 trials Phase 2+
- Nicolas PENELCentre Oscar Lambret · 2 of 2 trials Phase 2+
- Pau MontesinosHospital Universitario La Fe · 2 of 2 trials Phase 2+
Registry trials of cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinib 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; 290 of 376 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. 24 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
282 trials of drugs against PDGFRA were stopped early; 24 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinib, 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 282.
- 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”
- 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”
- 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”
- 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 · 2026-01-20 · 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”
- terminated · 2025-09-23 · Jiangsu HengRui Medicine Co., Ltd. · Camrelizumab;Pemetrexed and Carboplatin; Famitinib;“Terminated by Sponsor”
- 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 · Jiangsu HengRui Medicine Co., Ltd. · SHR-1802＋camrelizumab + famitinib“Adjustment of R\&D strategy”
- terminated · 2025-04-30 · University of Washington · Pazopanib hydrochloride“Study was terminated at completion of the Phase 1 dose escalation due to loss of financial support.”
- terminated · 2025-03-30 · UNICANCER · Regorafenib then Trifluridine/Tipiracil, Trifluridine/Tipiracil then Regorafenib“Following the presentation of the SUNLIGHT study results at ASCO GI and the rapid change in patient management in the majority of centers, the IDMC experts recommended halting inclusion in the SOREGATT study with immediate effect.”
- terminated · 2025-03-11 · Great Novel Therapeutics Biotech & Medicals Corporation · Regorafenib“The overall profile does not support development for Hepatocellular Carcinoma”
- terminated · 2025-03-07 · Aptose Biosciences Inc. · matched by the registry's drug synonyms“Change in corporate strategy”
- terminated · 2025-03-06 · Boehringer Ingelheim · matched by the registry's drug synonymsNo reason given
- terminated · 2025-02-25 · Niguarda Hospital · Midostaurin“didn't reach the primary endpoint”
Paediatric incentives and obligations for PDGFRA's drugs
For cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinib: 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 · PDGFRA 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-002358-PIP02-18-M04 (avapritinib, Treatment of all conditions included in the category of malignant neoplasms (except haematopoietic and lymphoid tissue neoplasms)): PM: decision on the application for modification of an agreed PIP, 25 Oct 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; +10 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/3/19/2177 withdrawn 28 Jun 2019: Treatment of glioma; EU/3/18/2074 positive 26 Oct 2018: Treatment of mastocytosis; EU/3/17/1936 positive 8 Nov 2017: Treatment of gastrointestinal stromal tumours; +9 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 · Ayvakyt: authorised since 24 Sept 2020, orphan medicine, conditional; Ofev: authorised since 14 Jan 2015; Sunitinib Accord: authorised since 11 Feb 2021; Qinlock: authorised since 18 Nov 2021, orphan medicine; +15 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: no · FDA's table lists no pediatric approval of cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinibRule: 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 · 86 interventional studies of cediranib, ripretinib, pazopanib, nintedanib esylate, quizartinib, masitinib, midostaurin, sunitinib, nintedanib, regorafenib, avapritinib, famitinib include patients under 18; 26 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 33 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.