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Factor VII (FVII) deficiency is a rare hereditary hemorrhagic disease caused by the diminution or absence of this coagulation factor.
Data assembled from 8 of 12 sources · Last updated Sep 19, 2026, 4:30 PM UTC
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Features include always present findings: Reduced factor VII activity and Prolonged bleeding after dental extraction; and very common findings: Intracranial hemorrhage and Gastrointestinal hemorrhage. 16 total HPO annotations.
Organ System | Phenotype Count | Example Features |
|---|---|---|
Blood and immune system | 4 | Abnormal bleeding tendency (abnormal bleeding), Prolonged bleeding after dental extraction, Gingival bleeding |
Bones and joints | 1 | Joint hemorrhage |
Heart and blood vessels | 1 | Intracranial hemorrhage |
Digestive system | 1 | Gastrointestinal hemorrhage |
F7 encodes coagulation factor VII (466 aa). Initiates the extrinsic pathway of blood coagulation. Serine protease that circulates in the blood in a zymogen form. Highest expression in Liver (83.6 TPM) and Testis (3.2 TPM).
Congenital factor VII deficiency is associated with mutations in the F7 gene on chromosome 13.
The F7 protein participates in factor VII propeptide pathway.
F7 is classified as a druggable target (Druggable Genome, Enzyme, and Protease categories) with score 0.6.
Genetic testing for F7 is available. Testing is considered confirmatory for diagnosis.
Biomarker and diagnostic research for congenital factor VII deficiency has been reported in the published literature.
No approved treatments are currently available for congenital factor VII deficiency. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for congenital factor VII deficiency, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for congenital factor VII deficiency. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor | Designated | Exclusivity End | Designation Status |
|---|---|---|---|---|---|
recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) | recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) | CSL Behring | 2013 | — | Designated |
Gene therapy approaches for congenital factor VII deficiency have been reported in the published literature.
4 trials found
Phenotype severity distribution: 2 always present features, 2 very common features, 7 common features.
Estimated prevalence: 1-9 in 1,000,000 (Rare).
4 clinical trials registered, 2 recruiting. Interventions under study include drug therapy and other interventions. Pipeline includes 1 PHASE2, 1 PHASE1, 1 NA. Research is sponsored by a mix of industry and academic institutions.
109 publications have been identified in PubMed for congenital factor VII deficiency. Research spans Case Report / Case Series (47%), Review / Meta-Analysis (19%), and Basic Science / Preclinical (11%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 51 | 47% |
Research summaries | 21 | 19% |
Laboratory research | 12 | 11% |
Disease patterns and progression | 11 | 10% |
Clinical study results | 5 | 5% |
Other research | 3 | 3% |
Testing and diagnosis research | 3 | 3% |
New treatment approaches | 3 | 3% |
El Messari C (2026). [PMID: 42211626](https://pubmed.ncbi.nlm.nih.gov/42211626/). *Cureus*. [Case Report / Case Series]
Araki Y (2026). [PMID: 40930828](https://pubmed.ncbi.nlm.nih.gov/40930828/). *Intern Med*. [Case Report / Case Series]
Pownder SL (2026). [PMID: 40719108](https://pubmed.ncbi.nlm.nih.gov/40719108/). *Vet Comp Orthop Traumatol*. [Case Report / Case Series]
Haisma B (2026). [PMID: 41443372](https://pubmed.ncbi.nlm.nih.gov/41443372/). *J Thromb Haemost*. [Diagnostic / Biomarker]
Löwing Svensson L (2026). [PMID: 41552754](https://pubmed.ncbi.nlm.nih.gov/41552754/). *Res Pract Thromb Haemost*. [Epidemiology / Natural History]
Khan MAM (2026). [PMID: 42011469](https://pubmed.ncbi.nlm.nih.gov/42011469/). *Cureus*. [Case Report / Case Series]
Hernández Zanata JR (2026). [PMID: 41567700](https://pubmed.ncbi.nlm.nih.gov/41567700/). *Case Rep Womens Health*. [Case Report / Case Series]
Langer S (2026). [PMID: 41522530](https://pubmed.ncbi.nlm.nih.gov/41522530/). *Indian J Hematol Blood Transfus*. [Review / Meta-Analysis]
Conca P (2026). [PMID: 41512903](https://pubmed.ncbi.nlm.nih.gov/41512903/). *Semin Thromb Hemost*. [Basic Science / Preclinical]
García-Jaén P (2026). [PMID: 41443233](https://pubmed.ncbi.nlm.nih.gov/41443233/). *Thromb Haemost*. [Case Report / Case Series]
AI-curated news mentioning congenital factor VII deficiency
Updated May 3, 2026
Hemab Therapeutics and Seaport Therapeutics are the newest biotech companies to join the public markets, each with an upsized IPO. According to IPO research firm Renaissance Capital, April was the biggest month for biotech IPO proceeds in more than five years. Here’s a look at the two newest biotech companies that joined the Nasdaq on Friday. ... How to turn analytics into actual policy outcomes. ... Hemab develops drugs for rare bleeding disorders. The company’s lead disease target is Glanzmann thomboasthenia, a genetic disorder that results in improperly functioning platelets. In Phase 1/2 testing, Hemab reported a significant reduction in the annualized treated bleed rate, according to the IPO filing. Sutacimig’s Phase 3 study is expected to begin in the second half of this year. A Phase 2 test of sutacimig is ongoing in a different disorder, Factor VII deficiency. Hemab expects to report clinical data from this study late this year or early next year. Hemab’s next drug candidate, HMB-002, is in development as a prophylactic treatment for von Willebrand disease, a disorder in which patients have low levels of von Willebrand factor (VWF). With that capital and the IPO proceeds, Hemab plans to spend $120 million to $130 million for clinical testing of sutacimig in Glanzmann thrombasthenia and Factor VII deficiency; $60 million to $70 million on clinical testing of HMB-002 for Von Willebrand disease; and $25 million to $35 million for other programs. This past week closed with three biotech companies joining the public markets, a flurry of activity that helped make April the biggest month for biotech IPOs since March 2021, according to Renaissance Capital.
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.
Both Seaport Therapeutics and Hemab Therapeutics are hoping to bring in around $180 million each from their public listings this week, in the latest sign that biotech IPOs are regaining momentum. Both Seaport and Hemab are hoping to bring in around $180 million from their listings, in the latest sign that biotech IPOs are regaining momentum. The biotech has already tested sutacimig in Glanzmann thrombasthenia, a rare genetic bleeding disorder caused by a deficiency of the platelet integrin alpha IIb beta3, which prevents blood from properly clotting. Hemab has previously pointed to phase 2 study as demonstrating that sutacimig achieved clinically meaningful bleeding reduction in these patients. The IPO funds would 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. Should the final share price fall in the middle of this range, the biotech expects the IPO to bring in $183.5 million in net proceeds—rising to $211.4 million if underwriters fully take up their 30-day offer to buy an additional 1.77 million shares at the same price. The biotech employs 58 people and was originally assembled by PureTech Health—where Zohar was previously CEO—and supplied with $100 million when it launched in the spring of 2024. Seaport followed that up with a $225 million series B a few months later. Out of the $325 million raised to date, Seaport entered 2026 with a hefty chunk of funds—$233.7 million, to be precise—still intact. When combining these funds with the IPO proceeds, Seaport said today that it has earmarked $121 million to advance its lead depression candidate SPT-300, also known as GlyphAllo.