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Hereditary or acquired coagulation disorder characterized by a qualitative or quantitative deficiency of the von Willebrand factor. The latter plays an important role in platelet adhesion. Signs and symptoms include bruises, nose bleeding, gum bleeding following a dental procedure, heavy menstrual bleeding, and gastrointestinal bleeding.
Biomarker and diagnostic research for von Willebrand disease (hereditary or acquired) has been reported in the published literature.
5 FDA-approved treatments are available for von Willebrand disease (hereditary or acquired), including Antihemophilic Factor/von Willebrand Factor Complex (Human) (Alphanate, approved 1978), von Willebrand factor (Recombinant) (Vonvendi, approved 2015), and Antihemophilic Factor (Recombinant) (Helixate Fs, Kogenate, Kogenate Fs, approved 2002). An additional 4 compounds hold orphan drug designation.
Brand Name | Generic Name | Mechanism | Approved |
|---|
37 clinical trials registered, 24 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and medical devices. Pipeline includes 2 PHASE4, 6 PHASE3, 3 PHASE2. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT06090201](https://clinicaltrials.gov/study/NCT06090201) |
Data assembled from 4 of 12 sources · Last updated Nov 4, 2026, 11:03 AM UTC
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Market Status
Vonvendi | von Willebrand factor (Recombinant) | — | 2015 | Available |
Nuwiq | Antihemophilic Factor (Recombinant) | — | 2015 | Available |
Novoeight | Antihemophilic Factor (Recombinant) | — | 2013 | Available |
Monoclate, Monoclate-P | Antihemophilic Factor (Human) | — | 2003 | Available |
Helixate Fs, Kogenate, Kogenate Fs | Antihemophilic Factor (Recombinant) | — | 2002 | Available |
Refacto | Antihemophilic Factor (Recombinant) | — | 2001 | Available |
Hemofil M | Antihemophilic Factor (Human) | — | 2001 | Available |
DDAVP | DESMOPRESSIN ACETATE | — | 1995 | Available |
Bioclate (Armour), Recombinate | Antihemophilic Factor (Recombinant) | — | 1992 | Available |
Alphanate | Antihemophilic Factor/von Willebrand Factor Complex (Human) | — | 1978 | Available |
Koate, Koate-Dvi | Antihemophilic Factor (Human) | — | 1974 | Available |
The following drugs have received orphan drug designation from the FDA for von Willebrand disease (hereditary or acquired). Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor | Designated | Exclusivity End | Designation Status |
|---|---|---|---|---|---|
recombinant humanized anti-TFPI monoclonal antibody | recombinant humanized anti-TFPI monoclonal antibody | Suzhou Alphamab Co., Ltd. | 2024 | — | Designated |
IgG4 monoclonal antibody directed against protein S | IgG4 monoclonal antibody directed against protein S | Vega Therapeutics, Inc. | 2023 | — | Designated |
PEGylated aptamer targeting Von Willebrand Factor A1 domain | PEGylated aptamer targeting Von Willebrand Factor A1 domain | Band Therapeutics, LLC | 2021 | — | Designated |
von Willebrand Factor Human Concentrate | von Willebrand Factor Human Concentrate | LFB USA, Inc. | 2014 | — | Withdrawn |
Gene therapy approaches for von Willebrand disease (hereditary or acquired) have been reported in the published literature.
37 trials found
Severe Congenital Hemostatic Defects, Cerebral MIcrobleeds and COGnition |
— |
University Hospital, Lille |
RECRUITING |
[NCT07404644](https://clinicaltrials.gov/study/NCT07404644) | An Observational Study of Vonicog Alfa (rVWF) in Pediatric Participants With Von Willebrand Disease (vWD) | — | Takeda | RECRUITING |
[NCT06754852](https://clinicaltrials.gov/study/NCT06754852) | A Study Assessing HMB-002 in Participants With Von Willebrand Disease | PHASE1 | Hemab ApS | RECRUITING |
[NCT05500807](https://clinicaltrials.gov/study/NCT05500807) | Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A | PHASE1 | Bleeding and Clotting Disorders Institute Peoria, Illinois | RECRUITING |
[NCT06651255](https://clinicaltrials.gov/study/NCT06651255) | Algorithm-based Management to Reduce the Recurrence of GI Bleeding and Severe Epistaxis in Von Willebrand Disease | NA | University Hospital, Lille | RECRUITING |
238 publications have been identified in PubMed for von Willebrand disease (hereditary or acquired). Research spans Review / Meta-Analysis (24%), Case Report / Case Series (22%), and Epidemiology / Natural History (16%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 56 | 24% |
Patient case studies | 53 | 22% |
Disease patterns and progression | 39 | 16% |
Laboratory research | 30 | 13% |
Testing and diagnosis research | 19 | 8% |
New treatment approaches | 19 | 8% |
Clinical study results | 16 | 7% |
Other research | 6 | 3% |
Denis CV (2026). [PMID: 42241704](https://pubmed.ncbi.nlm.nih.gov/42241704/). *Blood Adv*. [Epidemiology / Natural History]
Luke NP (2026). [PMID: 33351443](https://pubmed.ncbi.nlm.nih.gov/33351443/). *Unknown Journal*. [Review / Meta-Analysis]
Salazar E (2026). [PMID: 42140677](https://pubmed.ncbi.nlm.nih.gov/42140677/). *Clin Lab Med*. [Review / Meta-Analysis]
Altug Inan M (2026). [PMID: 41789952](https://pubmed.ncbi.nlm.nih.gov/41789952/). *Haemophilia*. [Gene Therapy / Novel Therapeutics]
Djambas Khayat C (2026). [PMID: 41614378](https://pubmed.ncbi.nlm.nih.gov/41614378/). *Eur J Haematol*. [Clinical Trial Publication]
Cabrero Segurado MA (2026). [PMID: 41606623](https://pubmed.ncbi.nlm.nih.gov/41606623/). *BMC Womens Health*. [Epidemiology / Natural History]
Castaman G (2026). [PMID: 41496701](https://pubmed.ncbi.nlm.nih.gov/41496701/). *Haematologica*. [Epidemiology / Natural History]
Haberichter SL (2026). [PMID: 41496700](https://pubmed.ncbi.nlm.nih.gov/41496700/). *Haematologica*. [Basic Science / Preclinical]
Napoles RA (2026). [PMID: 42104596](https://pubmed.ncbi.nlm.nih.gov/42104596/). *Am J Case Rep*. [Case Report / Case Series]
Seidizadeh O (2026). [PMID: 40820832](https://pubmed.ncbi.nlm.nih.gov/40820832/). *Haematologica*. [Clinical Trial Publication]
AI-curated news mentioning von Willebrand disease (hereditary or acquired)
Updated Oct 14, 2026
Research demonstrates that small interfering RNA targeting protein Z-dependent protease inhibitor enhances hemostasis in mice deficient in von Willebrand factor and Factor X. This study could pave the way for new therapeutic strategies in bleeding disorders.
A case report details comprehensive dental management under general anesthesia for a pediatric patient with type 3 von Willebrand disease. This study highlights the challenges and considerations in treating dental issues in patients with bleeding disorders.
A recent study highlights persistent gingival bleeding after dental scaling as a potential diagnostic indicator for von Willebrand disease. This finding could enhance early detection and management of the condition.
FDA classifies the von Willebrand factor assay into class II, establishing special controls to ensure safety and effectiveness. This classification aims to enhance regulatory oversight for devices related to von Willebrand disease.
If you’re looking for a sign that biotech’s IPO renaissance remains on track then the pair of upsized Nasdaq listings on Friday morning is a good place to start. | "We want to develop those programs as far as we can ourselves,” Seaport CEO Daphne Zohar told Fierce. The IPO funds are expected to help finance a phase 3 study in Glanzmann thrombasthenia, as well as an ongoing phase 2 trial in another bleeding disorder called Factor VII deficiency. Meanwhile, Hemab wants to push its monovalent antibody, HMB-002, through a phase 1/2 study for von Willebrand disease, another genetic disorder that impacts blood clotting. Seaport Therapeutics Hemab Therapeutics IPO biotech IPO Biotech Zohar co-founded Karuna Therapeutics, the neuroscience biotech bought by Bristol Myers Squibb for $14 billion to acquire the schizophrenia drug that would hit the market as Cobenfy. When asked by Fierce why Seaport hadn’t also opted for a Big Pharma acquisition, Zohar suggested that companies try and keep both options open. Both depression-focused Seaport Therapeutics and clotting company Hemab Therapeutics had been priming investors earlier this week to expect IPOs of around the $180 million range. But the companies significantly overshot their targets. Boston-based Seaport sold nearly 14.2 million shares for its offering—flying past the 11.8 million shares that the company had been expecting as recently as Monday. With the shares priced at $18 apiece, it means the biotech will rake in $254.9 million in gross proceeds from the IPO. Seaport’s stock began trading on the Nasdaq this morning under the ticker “SPTX.” It was joined by another biotech entrant in the form of Hemab. Like Seaport, Hemab had been banking on an IPO of around $180 million, but the end result has come out above $300 million, according to an April 30 release.