Disease overview

Alterations

155

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

156

Natural agents

Natural products

Merged ranked

181

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, GreenMedInfo, Examine.com · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

No spontaneous remission; lifelong clotting factor deficiency; joint damage progressive with each bleed without treatment; with prophylaxis, normal lifespan achievable

Best-intervention remission

Emicizumab prophylaxis (HAVEN studies): 87% reduction in bleeds vs on-demand FVIII; fitusiran (antithrombin RNAi): monthly subcutaneous injection, 89% reduction in bleeds; gene therapy (valoctocogene roxaparvovec): 94% reduction in bleeds at 5 years (GENEr8-1)

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Large — gene therapy offers functional cure but access is essentially the entire gap

Primary barrier

access

Barrier detail: Gene therapy for haemophilia A (valoctocogene roxaparvovec/Roctavian, FDA approved 2023) and B (fidanacogene elaparvovec/Beqvez) represent functional cures in eligible patients but cost $2.9-3.5M; IgG immune responses limit retreatment; durability questions persist at 5 years; most patients in LMICs remain on factor concentrates

Last SoC change: 2023 — fidanacogene elaparvovec (Beqvez, gene therapy for HemB) FDA approved; 2023 valoctocogene roxaparvovec (Roctavian) FDA approved for HemA; fitusiran (antithrombin siRNA) 2023; emicizumab (Hemlibra) 2017; factor concentrates standard since 1960s

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0018660 · ORPHA: ORPHA:169808

Alterations

Showing 80 of 155 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of the elbow	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of the intrinsic pathway	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arthralgia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arthropathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bleeding with minor or no trauma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bruising susceptibility	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cephalohematoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Delayed onset bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Epidural hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Epistaxis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastrointestinal hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gingival bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hematemesis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hematuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intracranial hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intramuscular hematoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intraventricular hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Joint hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Joint swelling	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Limb joint contracture	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Melena	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Menometrorrhagia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Menorrhagia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Muscle hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Oral cavity bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Osteoarthritis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Persistent bleeding after trauma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Petechiae	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Poor wound healing	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Post-partum hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged bleeding after dental extraction	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged bleeding after surgery	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged bleeding following procedure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged bleeding time	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged partial thromboplastin time	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged prothrombin time	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged whole-blood clotting time	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Purpura	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reduced factor IX activity	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Reduced factor VIII activity	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Reduced factor XI activity	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Reduced von Willebrand factor activity	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Splenic rupture	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Spontaneous hematomas	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Spontaneous, recurrent epistaxis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Synovitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thromboembolism	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
ACVRL1 activin A receptor like type 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COL4A1 collagen type IV alpha 1 chain	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CRP C-reactive protein	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Change From Baseline in Body Temperature	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
F10 coagulation factor X	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
F11 coagulation factor XI	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
F7 coagulation factor VII	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
F8 coagulation factor VIII	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
F9 coagulation factor IX	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FAU FAU ubiquitin like and ribosomal protein S30 fusion	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FGA fibrinogen alpha chain	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FUNDC2 FUN14 domain containing 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
H2AB1 H2A.B variant histone 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IFNAR1 interferon alpha and beta receptor subunit 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IFNAR2 interferon alpha and beta receptor subunit 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL11RA interleukin 11 receptor subunit alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL6 interleukin 6	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Incidence of adverse events	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Incremental Recovery	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
LMAN1 lectin, mannose binding 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MS4A1 membrane spanning 4-domains A1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Number of treated bleeds	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
PLG plasminogen	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PROS1 protein S	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Percentages of Success as Defined as the Combination of "Good" and "Excellent" Responses by the Investigator or Designee	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Proportion of successfully treated bleeding episodes	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
RPL10 ribosomal protein L10	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RPL10A ribosomal protein L10a	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RPL11 ribosomal protein L11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RPL12 ribosomal protein L12	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RPL13 ribosomal protein L13	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
RPL13A ribosomal protein L13a	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABCIXIMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALBUTREPENONACOG ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALTEPLASE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANDEXANET ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ANIFROLUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ANISTREPLASE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ANTIHEMOPHILIC FACTOR (RECOMBINANT) PEGYLATED-EXEI Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ANTITHROMBIN ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ANTITHROMBIN III Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APIXABAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZATHIOPRINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BETRIXABAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
COAGULATION FACTOR IX (RECOMBINANT), GLYCOPEGYLATED Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
COAGULATION FACTOR IX HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR IX RECOMBINANT HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR VII HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
COAGULATION FACTOR X HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR X, HUMAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
COLLAGENASE CLOSTRIDIUM HISTOLYTICUM Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
COUMARIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DAMOCTOCOG ALFA PEGOL Repurposing	Small molecule	Approved	Strong	95	8 trials 2 Trial pubs 1 RCT View evidence Registry trials NCT07088458 — An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A (NOT_YET_RECRUITING) NCT04461639 — Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (ACTIVE_NOT_RECRUITING) NCT04085458 — Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) (COMPLETED) PHASE4 NCT04015492 — Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII) (COMPLETED) PHASE1 NCT05643560 — An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A. (ACTIVE_NOT_RECRUITING) NCT03932201 — Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A (ACTIVE_NOT_RECRUITING) NCT05395858 — A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers (TERMINATED) NCT06222697 — A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A (RECRUITING) Published literature Clinical trial publication A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study (Advances in therapy, 2026) Clinical trial publication Direct comparison of two extended half-life PEGylated recombinant FVIII products: a randomized, crossover pharmacokinetic study in patients with severe hemophilia A (Annals of hematology, 2020) CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DANAPAROID Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DESMOPRESSIN Repurposing	Small molecule	Approved	Strong	95	8 trials 2 Cochrane 1 Meta-analysis 11 Trial pubs 5 RCT View evidence Registry trials NCT03054662 — Non Substitutive Strategies to Improve Haemophilia Care in Developing Countries. Experience in Ivory Coast. (COMPLETED) NA NCT01403740 — Turkish Acquired Haemophilia Registry (UNKNOWN) NCT02506023 — Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers (COMPLETED) PHASE1 NCT03136003 — DDAVP vs. Exercise in Patients With Mild Hemophilia A (UNKNOWN) PHASE4 NCT05628558 — Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A (UNKNOWN) NCT00994929 — Efficacy and Safety of IL-11 in DDAVP Unresponsive (COMPLETED) PHASE2 NCT03379974 — Exercise Versus DDAVP in Patients With Mild Hemophilia A (COMPLETED) PHASE4 NCT06020456 — Genetic Factors of the Desmopressin Response in Carriers of Hemophilia A (COMPLETED) Published literature Cochrane review Desmopressin as a Treatment in Patients With Von Willebrand Disease: A Systematic Review (Cureus, 2023) Cochrane review Efficacy of parenteral formulations of desmopressin in the treatment of bleeding disorders: A systematic review (Thrombosis research, 2022) Meta-analysis DDAVP response and its determinants in bleeding disorders: a systematic review and meta-analysis (Blood, 2025) Systematic review Maternal and neonatal bleeding complications in relation to peripartum management in hemophilia carriers: A systematic review (Blood reviews, 2021) Systematic review Inherited Bleeding Disorders in the Obstetric Patient (Transfusion medicine reviews, 2018) Clinical trial publication Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trial (Blood, 2022) Clinical trial publication Molecular and phenotypic determinants of the response to desmopressin in adult patients with mild hemophilia A (Journal of thrombosis and haemostasis : JTH, 2009) Clinical trial publication Circumcision in hemophilia: a cost-effective method using a novel device (Journal of pediatric surgery, 2004) Clinical trial publication Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand disease (The Journal of pediatrics, 2002) Clinical trial publication Adverse events during use of intranasal desmopressin acetate for haemophilia A and von Willebrand disease: a case report and review of 40 patients (Haemophilia : the official journal of the World Federation of Hemophilia, 2000) Randomized controlled trial [Clinical assessment of desmopressin to reduce blood loss in patients after cardiopulmonary bypass] (Ma zui xue za zhi = Anaesthesiologica Sinica, 1993) Randomized controlled trial Adventures in hemostasis. Desmopressin in cardiac surgery (Archives of surgery (Chicago, Ill. : 1960), 1993) CT.gov search · Cochrane · PubMed	DGIdb	—
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DROTRECOGIN ALFA Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EDOXABAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EDOXABAN TOSYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EFANESOCTOCOG ALFA Repurposing	Small molecule	Approved	Strong	95	8 trials 1 Meta-analysis 2 RCT View evidence Registry trials NCT06684314 — A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan (RECRUITING) NCT06716814 — Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa (RECRUITING) PHASE3 NCT04161495 — A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A (COMPLETED) PHASE3 NCT06941870 — Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy (RECRUITING) PHASE4 NCT06940830 — Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa (RECRUITING) PHASE4 NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A (COMPLETED) PHASE1 NCT04759131 — Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (COMPLETED) PHASE3 NCT04644575 — Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (ACTIVE_NOT_RECRUITING) PHASE3 CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
EFMOROCTOCOG ALFA Repurposing	Small molecule	Approved	Strong	95	8 trials 2 Trial pubs View evidence Registry trials NCT04293523 — A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health (ACTIVE_NOT_RECRUITING) NCT02196207 — Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform (WITHDRAWN) PHASE3 NCT02586012 — Weight-based Dosing in Hemophilia A (TERMINATED) PHASE2 NCT03103542 — Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies (COMPLETED) PHASE4 NCT02976753 — Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products (COMPLETED) NCT02546622 — ATHN 2: Factor Switching Study (COMPLETED) NCT02392156 — Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes (TERMINATED) NCT05856266 — An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa (TERMINATED) PHASE4 Published literature Clinical trial publication A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A (Hematology (Amsterdam, Netherlands), 2025) Clinical trial publication A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial (Blood advances, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
EFTRENONACOG ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EMICIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPTACOG ALFA (ACTIVATED) Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EPTACOG BETA (ACTIVATED) Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EPTIFIBATIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETRANACOGENE DEZAPARVOVEC Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Sources	Links
COAGULATION FACTOR IX RECOMBINANT HUMAN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
EFTRENONACOG ALFA	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
FITUSIRAN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
EPTACOG ALFA (ACTIVATED)	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ARC-19499	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
BAY-1093884	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
MARZEPTACOG ALFA (ACTIVATED)	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
APIXABAN	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
COAGULATION FACTOR IX HUMAN	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
CONCIZUMAB	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
DAMOCTOCOG ALFA PEGOL	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
EFANESOCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
EFMOROCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
LONOCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
MARSTACIMAB	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
MOROCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
OCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
RIVAROXABAN	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
SIMOCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
SUSOCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
TUROCTOCOG ALFA	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
VALOCTOCOGENE ROXAPARVOVEC	Gene therapy	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
VITAMIN K	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
VON WILLEBRAND FACTOR HUMAN	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABCIXIMAB Repurposing via FGA	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing via CRP	Small molecule	Approved	Strong	95	—	DGIdb	—
ALBUTREPENONACOG ALFA Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALTEPLASE Repurposing via FGA	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANDEXANET ALFA Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
ANIFROLUMAB Repurposing via IFNAR2	Small molecule	Approved	Strong	95	—	DGIdb	—
ANISTREPLASE Repurposing via FGA	Small molecule	Approved	Strong	95	—	DGIdb	—
ANTIHEMOPHILIC FACTOR (RECOMBINANT) PEGYLATED-EXEI Repurposing via F8	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT Repurposing via F8	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ANTITHROMBIN ALFA Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ANTITHROMBIN III Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
APIXABAN Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing via FAU	Small molecule	Approved	Strong	95	—	DGIdb	—
AZATHIOPRINE Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	—
BETRIXABAN Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOMIPRAMINE Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
COAGULATION FACTOR IX (RECOMBINANT), GLYCOPEGYLATED Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	—
COAGULATION FACTOR IX HUMAN Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR IX RECOMBINANT HUMAN Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR VII HUMAN Repurposing via F7	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW Repurposing via F7	Small molecule	Approved	Strong	95	—	DGIdb	—
COAGULATION FACTOR X HUMAN Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
COAGULATION FACTOR X, HUMAN Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
COLLAGENASE CLOSTRIDIUM HISTOLYTICUM Repurposing via COL4A1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
COUMARIN Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	—
DAMOCTOCOG ALFA PEGOL Repurposing via F8	Small molecule	Approved	Strong	95	8 trials 2 Trial pubs 1 RCT View evidence Registry trials NCT07088458 — An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A (NOT_YET_RECRUITING) NCT04461639 — Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (ACTIVE_NOT_RECRUITING) NCT04085458 — Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) (COMPLETED) PHASE4 NCT04015492 — Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII) (COMPLETED) PHASE1 NCT05643560 — An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A. (ACTIVE_NOT_RECRUITING) NCT03932201 — Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A (ACTIVE_NOT_RECRUITING) NCT05395858 — A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers (TERMINATED) NCT06222697 — A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A (RECRUITING) Published literature Clinical trial publication A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study (Advances in therapy, 2026) Clinical trial publication Direct comparison of two extended half-life PEGylated recombinant FVIII products: a randomized, crossover pharmacokinetic study in patients with severe hemophilia A (Annals of hematology, 2020) CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DANAPAROID Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
DESMOPRESSIN Repurposing via F8	Small molecule	Approved	Strong	95	8 trials 2 Cochrane 1 Meta-analysis 11 Trial pubs 5 RCT View evidence Registry trials NCT03054662 — Non Substitutive Strategies to Improve Haemophilia Care in Developing Countries. Experience in Ivory Coast. (COMPLETED) NA NCT01403740 — Turkish Acquired Haemophilia Registry (UNKNOWN) NCT02506023 — Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers (COMPLETED) PHASE1 NCT03136003 — DDAVP vs. Exercise in Patients With Mild Hemophilia A (UNKNOWN) PHASE4 NCT05628558 — Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A (UNKNOWN) NCT00994929 — Efficacy and Safety of IL-11 in DDAVP Unresponsive (COMPLETED) PHASE2 NCT03379974 — Exercise Versus DDAVP in Patients With Mild Hemophilia A (COMPLETED) PHASE4 NCT06020456 — Genetic Factors of the Desmopressin Response in Carriers of Hemophilia A (COMPLETED) Published literature Cochrane review Desmopressin as a Treatment in Patients With Von Willebrand Disease: A Systematic Review (Cureus, 2023) Cochrane review Efficacy of parenteral formulations of desmopressin in the treatment of bleeding disorders: A systematic review (Thrombosis research, 2022) Meta-analysis DDAVP response and its determinants in bleeding disorders: a systematic review and meta-analysis (Blood, 2025) Systematic review Maternal and neonatal bleeding complications in relation to peripartum management in hemophilia carriers: A systematic review (Blood reviews, 2021) Systematic review Inherited Bleeding Disorders in the Obstetric Patient (Transfusion medicine reviews, 2018) Clinical trial publication Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trial (Blood, 2022) Clinical trial publication Molecular and phenotypic determinants of the response to desmopressin in adult patients with mild hemophilia A (Journal of thrombosis and haemostasis : JTH, 2009) Clinical trial publication Circumcision in hemophilia: a cost-effective method using a novel device (Journal of pediatric surgery, 2004) Clinical trial publication Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand disease (The Journal of pediatrics, 2002) Clinical trial publication Adverse events during use of intranasal desmopressin acetate for haemophilia A and von Willebrand disease: a case report and review of 40 patients (Haemophilia : the official journal of the World Federation of Hemophilia, 2000) Randomized controlled trial [Clinical assessment of desmopressin to reduce blood loss in patients after cardiopulmonary bypass] (Ma zui xue za zhi = Anaesthesiologica Sinica, 1993) Randomized controlled trial Adventures in hemostasis. Desmopressin in cardiac surgery (Archives of surgery (Chicago, Ill. : 1960), 1993) CT.gov search · Cochrane · PubMed	DGIdb	—
DEXAMETHASONE Repurposing via F9	Small molecule	Approved	Strong	95	—	DGIdb	—
DIAZEPAM Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ACVRL1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DROTRECOGIN ALFA Repurposing via F8	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EDOXABAN Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	—
EDOXABAN TOSYLATE Repurposing via F10	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets

Natural Products

1 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked)

Reference lookups: GreenMedInfo · Examine.com

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Kefir Natural product Animal Study Therapeutic effects of kefir peptides on hemophilia-induced osteoporosi…	Nutraceutical	Preclinical	Emerging	0	1 pubs View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

Haemophilia A / B

Disease overview

Remission & chronicity

Alterations

Therapeutics

Natural Products

Part of the Open Source Medicine Foundation Network