Disease · MONDO_0018660

hemophilia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hemophilia. Diseases, record MONDO_0018660. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018660

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: hemophilia; Open Targets
Open Targets ID: MONDO_0018660; Open Targets
Disease Ontology: DOID:0061030; Disease Ontology
NCI Thesaurus: C3093; NCI Thesaurus
Orphanet: ORPHA:448; Orphanet
Highest stage of its drugs: Approval, for 8 of its 18 drugs, from TTD; Open Targets

Drugs18

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
OCTOCOG ALFA	CHEMBL4297873	Protein	Approval	TTD	3	1	0	Drugs and targets
MOROCTOCOG ALFA	CHEMBL2109137	Protein	Approval	TTD	3	0	0	Drugs and targets
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	CHEMBL4594584		Approval	TTD	2	0	0	Drugs and targets
LONOCTOCOG ALFA	CHEMBL4594603	Protein	Approval	TTD	2	0	0	Drugs and targets
COAGULATION FACTOR IX HUMAN	CHEMBL4297986		Approval	TTD	1	0	0	Drugs and targets
EFMOROCTOCOG ALFA	CHEMBL4297945	Protein	Approval	TTD	1	0	0	Drugs and targets
TUROCTOCOG ALFA	CHEMBL2108286	Protein	Approval	TTD	1	0	0	Drugs and targets
VON WILLEBRAND FACTOR HUMAN	CHEMBL4298126		Approval	TTD	1	0	0	Drugs and targets
MARSTACIMAB	CHEMBL3990039	Antibody	Phase 3	TTD	5	4	4	Drugs and targets
COAGULATION FACTOR IX RECOMBINANT HUMAN	CHEMBL2109205	Protein	Phase 3	ClinicalTrials.gov	2	2	0	Drugs and targets

1 to 10 of 18

See all 18 drugs

Targets6

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
F8	Coagulation factor VIII	7	Coagulation factor VIII exogenous protein	Approval	https://conductscience.org/library/genes/2157	Drugs and targets
F9	Coagulation factor IX	3	Coagulation factor IX exogenous protein	Approval	https://conductscience.org/library/genes/2158	Drugs and targets
TFPI	Tissue factor pathway inhibitor	2	Tissue factor pathway inhibitor inhibitor	Phase 3	https://conductscience.org/library/genes/7035	Drugs and targets
F7	Coagulation factor VII	1	Coagulation factor VII exogenous protein	Phase 2	https://conductscience.org/library/genes/2155	Drugs and targets
SERPINC1	Antithrombin mRNA	1	Antithrombin mRNA RNAi inhibitor	Phase 3	https://conductscience.org/library/genes/462	Drugs and targets
VWF	von Willebrand factor	1	von Willebrand factor exogenous protein	Approval	https://conductscience.org/library/genes/7450	Drugs and targets

Trials9

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 3 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00486278	Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds	Phase 2	51	2014-12-25	Congenital Bleeding Disorder, Haemophilia A, Haemophilia B	EPTACOG ALFA (ACTIVATED)	Its record names the disease	ClinicalTrials.gov
NCT03363321	PF-06741086 Long-term Treatment in Severe Hemophilia	Phase 2	20	2021-07-27	Hemophilia A or B	MARSTACIMAB	Its record names the disease	ClinicalTrials.gov
NCT04878731	Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia	Phase 1	6	2023-07-07	Severe Hemophilia	MARSTACIMAB	Its record names the disease	ClinicalTrials.gov
NCT06992076	Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry		100		Hemophilia	MARSTACIMAB	Its record names the disease	ClinicalTrials.gov
NCT07161687	A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.		50		Hemophilia A or B	MARSTACIMAB	Its record names the disease	ClinicalTrials.gov
NCT01191372	First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients	Phase 1	17		Hemophilia	ARC-19499	Its record names the disease	ClinicalTrials.gov
NCT01830712	A Long-term Assessment of Physical Activity, Range of Motion, and Functional Status Following Elective Orthopedic Surgery in Hemophilia Patients With Inhibitors		15		Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors	EPTACOG ALFA (ACTIVATED)	Its record names the disease	ClinicalTrials.gov
NCT03481946	A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia	Phase 1	6		Hemophilia A; Hemophilia B	BAY-1093884	Its record names the disease	ClinicalTrials.gov
NCT03996486	Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia	Phase 1	0		Hemophilia	BAY-1093884	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

None in this release.

Papers from its trials3

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
22470921	RESULT	1	PubMed
42698050	DERIVED	2	PubMed
36220152	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0018660

GET https://conductscience.org/api/diseases/v1/MONDO_0018660/drugs

API reference For AI assistants