FLT3 fms related receptor tyrosine kinase 3
ENSG00000122025 · protein coding · Open Targets profile
Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine FLT3LG and regulates differentiation, proliferation and survival of hematopoietic progenitor cells and of dendritic cells. Promotes phosphorylation of SHC1 and AKT1, and activation of the downstream effector MTOR. Promotes activation of RAS signaling and phosphorylation of downstream kinases, including MAPK1/ERK2 and/or MAPK3/ERK1. Promotes phosphorylation of FES, FER, PTPN6/SHP, PTPN11/SHP-2, PLCG1, and STAT5A and/or STAT5B. Activation of wild-type FLT3 causes only marginal activation of STAT5A or STAT5B. Mutations that cause constitutive kinase activity promote cell proliferation and resistance to apoptosis via the activation of multiple signaling pathways. {ECO:0000269|PubMed:10080542, ECO:0000269|PubMed:11090077, ECO:0000269|PubMed:14504097, ECO:0000269|PubMed:16266983, ECO:0000269|PubMed:16627759, ECO:0000269|PubMed:18490735, ECO:0000269|PubMed:20111072, ECO:0000269|PubMed:21067588, ECO:0000269|PubMed:21262971, ECO:0000269|PubMed:21516120, ECO:0000269|PubMed:7507245}.
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 Withdrawn means the drug was pulled from the market or from development.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, Med-Quality Pocket, Druggable Family
- Antibody
- Phase 1 Clinical, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Literature, UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
- Other modalities
- Advanced Clinical
gnomAD loss of function constraint: o/e 0.3634600043296814 (upper bound 0.4647200107574463). 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 FLT3 (quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, AT-9283, famitinib), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- 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+
- Bristol-Myers SquibbBristol-Myers Squibb · 2 of 3 trials Phase 2+Ph3NCT03952039 · An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinibbrief
- Anna HalpernFred Hutch/University of Washington Cancer Consortium · 2 of 2 trials Phase 2+
- Arun RajanNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Bristol Myers SquibbBristol-Myers Squibb · 2 of 2 trials Phase 2+
- Deepak KilariMedical College of Wisconsin · 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+
- Ghassan Abou-AlfaMemorial Sloan Kettering Cancer Center · 2 of 2 trials Phase 2+
- Juan BerguaHospital San Pedro Alcántara · 2 of 2 trials Phase 2+
- Maria ReigBCLC group. Liver Unit. Hospital Clinic. Ciberehd · 2 of 2 trials Phase 2+
- Najla El JurdiNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Nicholas ShortMD Anderson Center · 2 of 2 trials Phase 2+
- Pau MontesinosHospital Universitario La Fe · 2 of 2 trials Phase 2+
Registry trials of quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, AT-9283, 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; 275 of 380 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. 20 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
311 trials of drugs against FLT3 were stopped early; 25 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, AT-9283, 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 311.
- terminated · 2026-08-25 · Association of Urologic Oncology (AUO) · SunitinibNo reason given
- withdrawn · 2026-08-17 · Thomas Jefferson University · Pacritinib“FDA requested significant changes to the study protocol”
- terminated · 2026-08-11 · Bristol-Myers Squibb · matched by the registry's drug synonymsNo reason given
- terminated · 2026-07-16 · Bristol-Myers Squibb · matched by the registry's drug synonymsNo reason given
- terminated · 2026-05-26 · The University of Texas Health Science Center at San Antonio · pacritinib“Investigational drug limitations”
- terminated · 2026-03-18 · Nerviano Medical Sciences · matched by the registry's drug synonyms“Study stopped due to strategic reasons. The decision is not based on specific safety findings as the safety observed in the phase II part of the study is in line with what was reported in the phase I part.”
- terminated · 2026-01-05 · Astellas Pharma Global Development, Inc. · gilteritinib“The study was terminated due to enrollment challenges.”
- terminated · 2025-12-04 · H. Lee Moffitt Cancer Center and Research Institute · Fedratinib Pill“Did not advance to Phase 2”
- terminated · 2025-11-17 · Medical College of Wisconsin · Pacritinib“futility and drug supplier in favor of new metastatic protocol.”
- suspended · 2025-10-23 · Mirror Biologics, Inc. · Sorafenib“Sponsor unable to fund”
- terminated · 2025-09-23 · Jiangsu HengRui Medicine Co., Ltd. · Camrelizumab;Pemetrexed and Carboplatin; Famitinib;“Terminated by Sponsor”
- terminated · 2025-08-22 · Jiangsu HengRui Medicine Co., Ltd. · SHR-1802＋camrelizumab + famitinib“Adjustment of R\&D strategy”
- withdrawn · 2025-08-06 · M.D. Anderson Cancer Center · Sorafenib“0 participants”
- withdrawn · 2025-04-27 · Medical University of South Carolina · matched by the registry's drug synonyms“study is being rewritten for different disease population”
- terminated · 2025-03-21 · Gulam Manji · matched by the registry's drug synonyms“Poor enrollment”
- terminated · 2025-03-07 · Aptose Biosciences Inc. · matched by the registry's drug synonyms“Change in corporate strategy”
- terminated · 2025-02-26 · Cancer Institute and Hospital, Chinese Academy of Medical Sciences · Sorafenib“IDMC recommended to terminate the study since Interim analysis showed that the experimental group had significant superiority over the control group in TTP and OS (p<0.001), meeting the predefined interim analysis endpoints in the study protocol.”
- terminated · 2025-02-25 · Niguarda Hospital · Midostaurin“didn't reach the primary endpoint”
- terminated · 2025-02-14 · AIO-Studien-gGmbH · matched by the registry's drug synonyms“insufficient accrual rate”
- terminated · 2025-02-12 · Georgetown University · matched by the registry's drug synonyms“Lack of efficacy”
- terminated · 2025-02-12 · Georgetown University · sunitinib and capecitabine“Unanticipated side effects and futility”
- terminated · 2024-12-17 · Henan Cancer Hospital · Famitinib“Investigators' decision”
- terminated · 2024-12-12 · Celgene · FEDRATINIB“Slow accrual.”
- terminated · 2024-11-27 · Astellas Pharma Global Development, Inc. · Gilteritinib“Study terminated due to adverse events related to the combination therapy”
- terminated · 2024-11-20 · Astellas Pharma Inc · matched by the registry's drug synonyms“Company decided not to pursue the development of this drug in this patient population at this time”
Paediatric incentives and obligations for FLT3's drugs
For quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, AT-9283, 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 · FLT3 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 · EMEA-002064-PIP01-16-M06 (Gilteritinib (as fumarate), Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 16 Aug 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-001939-PIP03-16 (pexidartinib, Treatment of benign soft tissue neoplasms): W: decision granting a waiver in all age groups for all conditions or indications, 29 Jan 2019; +3 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/3/13/1200 expired 13 Nov 2013: Treatment of papillary thyroid cancer; EU/3/13/1199 expired 13 Nov 2013: Treatment of follicular thyroid cancer; EU/3/15/1457 withdrawn 19 Mar 2015: Treatment of tenosynovial giant cell tumour, localised and diffuse type; EU/3/10/811 positive 26 Nov 2010: Treatment of post-polycythaemia vera myelofibrosis; +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 · Sunitinib Accord: authorised since 11 Feb 2021; Vanflyta: authorised since 6 Nov 2023; Inrebic: authorised since 8 Feb 2021, orphan medicine; Sorafenib Accord: authorised since 9 Nov 2022; +6 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 quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, at-9283, 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 · 104 interventional studies of quizartinib, pacritinib, pexidartinib, fedratinib, midostaurin, sorafenib, lestaurtinib, gilteritinib, sunitinib, ruserontinib, at-9283, famitinib include patients under 18; 33 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.