VWF von Willebrand factor
ENSG00000110799 · protein coding · Open Targets profile
Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sites of vascular injury by forming a molecular bridge between sub-endothelial collagen matrix and platelet- surface receptor complex GPIb-IX-V. Also acts as a chaperone for coagulation factor VIII, delivering it to the site of injury, stabilizing its heterodimeric structure and protecting it from premature clearance from plasma.
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 Withdrawn means the drug was pulled from the market or from development.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
- Med-Quality Pocket
- Antibody
- Approved Drug, UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Database Ubiquitination
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.5702300071716309 (upper bound 0.6461700201034546). 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 35 registry trials, updated in the last three years, of the most advanced drugs against VWF (caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegol), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Ginger NicolWashington University School of Medicine · 1 of 1 trials Phase 2+
- Marco ValgimigliCardiocentro Ticino · 1 of 1 trials Phase 2+
- Margaret V RagniUniversity of Pittsburgh · 1 of 1 trials Phase 2+
- Martin SiegemundUniversity Hospital, Basel, Switzerland · 1 of 1 trials Phase 2+
- Nicoletta MachinUniversity of Pittsburgh · 1 of 1 trials Phase 2+
- Sandra CamprubiInstituto Grifols, S.A. · 1 of 1 trials Phase 2+
- Elena SantagostinoSobi AB · 0 of 1 trials Phase 2+
- Eric VanbelleUniversity Hospital, Lille · 0 of 1 trials Phase 2+
- Flora PeyvandiA.Bonomi Hemophilia and Thrombosis Center, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico · 0 of 1 trials Phase 2+
- Guillaume LEBRETONAssistance Publique - Hôpitaux de Paris · 0 of 1 trials Phase 2+
- Jean-Luc DiehlAP-HP, Hôpital Européen Georges Pompidou, Paris · 0 of 1 trials Phase 2+
- Michael MazzeffiUVA · 0 of 1 trials Phase 2+
Registry trials of caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegol 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; 19 of 34 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. 1 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
16 trials of drugs against VWF were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegol, 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 16 most recent of 16.
- terminated · 2026-09-30 · Grifols Biologicals, LLC · matched by the registry's drug synonyms“Due to recruitment challenges during the trial, this study was terminated early by the Sponsor.”
- withdrawn · 2026-05-22 · Grifols Therapeutics LLC · matched by the registry's drug synonyms“Due to recruitment difficulties for pediatric patients. The reason for study cancellation is not related to safety concerns or any adverse events.”
- withdrawn · 2026-05-07 · Ezeldeen Abuelkasem · matched by the registry's drug synonyms“No supply of the test substance was provided by the sponsor.”
- terminated · 2026-02-20 · University Hospital, Rouen · Caplacizumab“Recommendations of the independent oversight committee.”
- terminated · 2024-07-01 · Margaret Ragni · matched by the registry's drug synonyms“The study stopped early due to slow recruitment.”
- terminated · 2021-11-23 · Grifols Therapeutics LLC · matched by the registry's drug synonyms“Terminated due to limited enrollment (non-safety related decision)”
- withdrawn · 2020-12-17 · City of Hope Medical Center · matched by the registry's drug synonyms“complete per PI”
- terminated · 2020-05-29 · University of California, Davis · matched by the registry's drug synonyms“Termination of funding from sponsor.”
- terminated · 2019-06-11 · Margaret Ragni · matched by the registry's drug synonyms“Recruitment slow as single center conducting in rare disease”
- terminated · 2017-10-03 · CSL Behring · matched by the registry's drug synonymsNo reason given
- terminated · 2010-03-03 · Bio Products Laboratory · matched by the registry's drug synonyms“Due to slow recruitment and a delay in reaching the recruitment target.”
- terminated · 2010-03-03 · Bio Products Laboratory · matched by the registry's drug synonyms“Due to slow recruitment and a significant delay in reaching the recruitment target.”
- terminated · 2010-02-10 · Archemix Corp. · matched by the registry's drug synonyms“Enrollment slower than expected”
- terminated · 2009-11-26 · Archemix Corp. · matched by the registry's drug synonyms“Enrollment into study was slower than expected.”
- withdrawn · 2009-08-21 · Archemix Corp. · matched by the registry's drug synonyms“Sponsor decided not to go forward with the study.”
- terminated · 2009-01-09 · Archemix Corp. · matched by the registry's drug synonymsNo reason given
Paediatric incentives and obligations for VWF's drugs
For caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegol: 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 · VWF 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: yes · EMEA-001164-PIP01-11-M08 (vonicog alfa, Treatment of von Willebrand disease): PM: decision on the application for modification of an agreed PIP, 18 Jul 2024; EMEA-001157-PIP01-11-M02 (Caplacizumab, Treatment of thrombotic thrombocytopenic purpura (TTP)): PM: decision on the application for modification of an agreed PIP, 12 Sept 2018; compliance positive 23 Aug 2019Rule: 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/3/09/629 positive 30 Apr 2009: Treatment of thrombotic thrombocytopenic purpuraRule: 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 EU/3/09/629, 2026-11-11
- Authorised in the EU: yes · Veyvondi: authorised since 31 Aug 2018; Cablivi: authorised since 30 Aug 2018, orphan medicineRule: 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 caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegolRule: 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 · 32 interventional studies of caplacizumab, vonicog alfa, von willebrand factor human, egaptivon pegol include patients under 18; 5 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, 2026-11-12
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 4 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.