VKORC1 vitamin K epoxide reductase complex subunit 1
ENSG00000167397 · protein coding · Open Targets profile
Involved in vitamin K metabolism. Catalytic subunit of the vitamin K epoxide reductase (VKOR) complex which reduces inactive vitamin K 2,3-epoxide to active vitamin K. Vitamin K is required for the gamma-carboxylation of various proteins, including clotting factors, and is required for normal blood coagulation, but also for normal bone development. {ECO:0000269|PubMed:14765194, ECO:0000269|PubMed:14765195, ECO:0000269|PubMed:15879509, ECO:0000269|PubMed:16270630, ECO:0000269|PubMed:20978134, ECO:0000269|PubMed:22923610, ECO:0000269|PubMed:33154105}.
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 Withdrawn means the drug was pulled from the market or from development.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
- UniProt SigP or TMHMM
- PROTAC
- Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.8083400130271912 (upper bound 1.3097000122070312). 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 135 registry trials, updated in the last three years, of the most advanced drugs against VKORC1 (phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarin), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Nir UrielNew York Presbyterian / Columbia University Medical Center · 2 of 2 trials Phase 2+
- Zhejie LiuSecond Affiliated Hospital, Zhejiang University, School of Medicine · 2 of 2 trials Phase 2+
- Agnes Y. Y. LeeDirector of Thrombosis, Division of Hematology, University of British Columbia, Canada · 1 of 1 trials Phase 2+
- Alice P ChenNational Cancer Institute (NCI) · 1 of 1 trials Phase 2+
- Anne-Lise KamperDepartment of Nephrology, Copenhagen University Hospital Rigshospitalet · 1 of 1 trials Phase 2+
- Asishana A OshoMassachusetts General Hospital · 1 of 1 trials Phase 2+
- Bao-Huan ZhouClinical Medical College of Yangzhou University · 1 of 1 trials Phase 2+
- Bum Joon KimAsan Medical Center · 1 of 1 trials Phase 2+
- Carol A LambourneDartmouth-Hitchcock Medical Center · 1 of 1 trials Phase 2+
- Christian D PetersDepartment of Nephrology, Aarhus University Hospital · 1 of 1 trials Phase 2+
- Christian Torp-PedersenDepartment of Cardiology, North Zealand Hospital · 1 of 1 trials Phase 2+
- Ditte HansenDepartment of Nephrology, Copenhagen University Hospital Herlev · 1 of 1 trials Phase 2+
- Dou-Sheng Bai BaiClinical Medical College of Yangzhou University · 1 of 1 trials Phase 2+
- Ellen Linnea F BallegaardDepartment of Nephrology, Copenhagen University Hospital Rigshospitalet · 1 of 1 trials Phase 2+
- Emilie Belley-CoteMcMaster University · 1 of 1 trials Phase 2+
Registry trials of phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarin 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; 102 of 125 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. 10 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
73 trials of drugs against VKORC1 were stopped early; 16 give no reason
Terminated, withdrawn and suspended interventional trials of phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarin, 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 73.
- withdrawn · 2026-04-02 · Teleflex · matched by the registry's drug synonyms“This study was never started and is no longer planned for execution. We are now in the process of aligning to a new study design with the FDA.”
- withdrawn · 2025-11-04 · Brian O'Neill MD · Market approved oral anticoagulation (OAC), such as warfarin (Coumadin), dabigatran (Pradaxa), rivaroxaban (Xarelto), apixiban (Eliquis), edoxaban (Savaysa), or betrixaban (Bevyxxa)“Lack of Funding”
- terminated · 2025-08-21 · Janssen Research & Development, LLC · Warfarin 10 mg“The study was stopped as all post-marketing commitments had been fulfilled or released”
- terminated · 2025-06-22 · Imperial College London · matched by the registry's drug synonyms“The trial failed to meet the milestones in the internal pilot of feasibility. The trial was redesigned to improve its deliverability and reduce burden to sites prior to being relaunched. Unfortunately, the trial failed to sufficiently recruit.”
- terminated · 2024-12-27 · National Hospital Organization Osaka National Hospital · matched by the registry's drug synonyms“The trial was terminated at the planned interim analysis for futility on July 18, 2023.”
- terminated · 2024-04-25 · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) · matched by the registry's drug synonymsNo reason given
- terminated · 2024-03-20 · Artivion Inc. · Warfarin“Safety”
- withdrawn · 2023-03-13 · Laval University · Warfarin“Submission process abandoned. No patient enrolled.”
- withdrawn · 2022-03-04 · University of Louisville · matched by the registry's drug synonyms“The study did not receive anticipated funding and therefore never opened to enrollment. The study was never submitted to the IRB for approval since the funding failed.”
- withdrawn · 2021-08-25 · First Affiliated Hospital Xi'an Jiaotong University · Warfarin Sodium“No participants enrolled”
- terminated · 2021-08-19 · Rajaie Cardiovascular Medical and Research Center · Warfarin“COVID-19 has impacted our study recruitment”
- withdrawn · 2021-03-25 · University of Texas Southwestern Medical Center · Warfarin“no accrual for over 12 months”
- terminated · 2021-03-16 · Mayo Clinic · Warfarin“Study terminated due to slower than anticipated enrollment.”
- terminated · 2021-01-07 · Woman's Health University Hospital, Egypt · warfarinNo reason given
- terminated · 2020-10-19 · Ohio State University · matched by the registry's drug synonyms“Low recruitment”
- terminated · 2020-08-12 · Mayo Clinic · matched by the registry's drug synonyms“Due to lack of recruitment of eligible participants”
- terminated · 2020-06-30 · Electrophysiology Research Foundation · Warfarin“Did not meet enrolment target for phase 1”
- terminated · 2020-05-15 · Hospital Geral Roberto Santos · Warfarin“Coronavirus Pandemic”
- terminated · 2019-12-13 · Duke University · Warfarin“Lack of enrollment”
- terminated · 2019-11-08 · University of Louisville · warfarin“study terminated due to low accrual”
- terminated · 2019-08-21 · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) · matched by the registry's drug synonyms“Slow accrual.”
- terminated · 2019-07-26 · Johannes Gutenberg University Mainz · Phenprocoumon“Recruiting problems”
- terminated · 2018-10-24 · PrECOG, LLC. · matched by the registry's drug synonyms“Slow enrollment”
- terminated · 2018-09-27 · Susan Smyth · warfarinNo reason given
- withdrawn · 2018-09-17 · PharmEvo Pvt Ltd · Warfarin“No patients Enrollment”
Paediatric incentives and obligations for VKORC1's drugs
For phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarin: 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: no · VKORC1 is not on the listRule: 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: no · No EMA paediatric investigation plan decision names phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarinRule: 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: no · No EU orphan designation names phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarinRule: 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: no · EMA lists no centrally authorised medicine of phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarinRule: 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 phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarinRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 38 interventional studies of phenprocoumon, phenindione, dicumarol, acenocoumarol, warfarin, tecarfarin include patients under 18; 1 is 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, 2026-11-12
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 7 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.