Disease · MONDO_0010604

hemophilia B

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hemophilia B. Diseases, record MONDO_0010604. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010604

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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 B; Open Targets
Open Targets ID: MONDO_0010604; Open Targets
Other names: Christmas disease, congenital factor IX deficiency, congenital factor IX disorder, factor IX deficiency, haemophilia b, X-linked recessive, haemophilia type B, hemophilia b, X-linked recessive, hemophilia type B, hereditary Factor IX deficiency, hereditary Factor IX deficiency disease; Open Targets
Disease Ontology: DOID:12259; Disease Ontology
MeSH: D002836; MeSH
NCI Thesaurus: C26721; NCI Thesaurus
Orphanet: ORPHA:98879; Orphanet
Highest stage of its drugs: Approval, for 11 of its 23 drugs, from EMA Human Drugs, FDA and PMDA; Open Targets

Drugs23

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
COAGULATION FACTOR IX RECOMBINANT HUMAN	CHEMBL2109205	Protein	Approval	EMA Human Drugs and PMDA	29	25	15	Drugs and targets
EFTRENONACOG ALFA	CHEMBL4594598	Protein	Approval	EMA Human Drugs	14	13	3	Drugs and targets
EPTACOG ALFA (ACTIVATED)	CHEMBL2108334	Protein	Approval	EMA Human Drugs	11	9	7	Drugs and targets
NONACOG BETA PEGOL	CHEMBL2108348	Protein	Approval	EMA Human Drugs	9	7	7	Drugs and targets
FIDANACOGENE ELAPARVOVEC	CHEMBL3990034	Gene	Approval	EMA Human Drugs, at withdrawal	4	3	3	Drugs and targets
ETRANACOGENE DEZAPARVOVEC	CHEMBL5095451	Gene	Approval	EMA Human Drugs	4	2	2	Drugs and targets
MARSTACIMAB	CHEMBL3990039	Antibody	Approval	EMA Human Drugs and FDA	4	2	2	Drugs and targets
CONCIZUMAB	CHEMBL4297730	Antibody	Approval	EMA Human Drugs	4	1	1	Drugs and targets
ALBUTREPENONACOG ALFA	CHEMBL5315076	Protein	Approval	EMA Human Drugs	3	1	1	Drugs and targets
COAGULATION FACTOR IX HUMAN	CHEMBL4297986		Approval	EMA Human Drugs, at withdrawal	1	0	0	Drugs and targets

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Targets83

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
F9	Coagulation factor IX	7	Coagulation factor IX exogenous gene; Coagulation factor IX exogenous protein	Approval	https://conductscience.org/library/genes/2158	Drugs and targets
F8	Coagulation factor VIII	5	Coagulation factor VIII exogenous protein	Phase 3	https://conductscience.org/library/genes/2157	Drugs and targets
TFPI	Tissue factor pathway inhibitor	3	Tissue factor pathway inhibitor inhibitor	Approval	https://conductscience.org/library/genes/7035	Drugs and targets
F7	Coagulation factor VII	2	Coagulation factor VII exogenous protein	Approval	https://conductscience.org/library/genes/2155	Drugs and targets
FAU	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/2197	Drugs and targets
RPL10	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/6134	Drugs and targets
RPL10A	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/4736	Drugs and targets
RPL11	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/6135	Drugs and targets
RPL12	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/6136	Drugs and targets
RPL13	80S Ribosome	1	80S Ribosome modulator	Phase 2	https://conductscience.org/library/genes/6137	Drugs and targets

1 to 10 of 83

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Trials54

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. 13 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01154231	BeneFIX Drug Use Results Survey [All-Case Surveillance]		314	2017-04-11	Hemophilia B	COAGULATION FACTOR IX RECOMBINANT HUMAN	Its record names the disease	ClinicalTrials.gov
NCT00484185	Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B		183	2013-08-12	Hemophilia B	COAGULATION FACTOR IX RECOMBINANT HUMAN	Its record names the disease	ClinicalTrials.gov
NCT01027364	Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B	Phase 3	123	2014-09-04	Severe Hemophilia B	COAGULATION FACTOR IX RECOMBINANT HUMAN, EFTRENONACOG ALFA	Its record names the disease	ClinicalTrials.gov
NCT03417245	A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors	Phase 3	120	2022-02-04	Hemophilia A, Hemophilia B	FITUSIRAN	Its record names the disease	ClinicalTrials.gov
NCT01174446	Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients	Phase 3	86	2013-11-25	Hemophilia B	COAGULATION FACTOR IX RECOMBINANT HUMAN	Its record names the disease	ClinicalTrials.gov
NCT03549871	A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis	Phase 3	80	2023-02-06	Hemophilia	FITUSIRAN	Its record names the disease	ClinicalTrials.gov
NCT00768287	Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B	Phase 2/3	77	2018-09-10	Hemophilia B	COAGULATION FACTOR IX RECOMBINANT HUMAN	Its record names the disease	ClinicalTrials.gov
NCT01333111	Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients	Phase 3	74	2017-07-28	Congenital Bleeding Disorder, Haemophilia B	NONACOG BETA PEGOL	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT01395810	Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773	Phase 3	71	2018-05-09	Congenital Bleeding Disorder, Haemophilia B	NONACOG BETA PEGOL	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT02336178	Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China	Phase 4	70	2017-05-04	HEMOPHILIA B	COAGULATION FACTOR IX RECOMBINANT HUMAN	Its record names the disease	ClinicalTrials.gov

1 to 10 of 54

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Comparisons in its trials17

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.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Factor On-demand vs Fitusiran 80 mg Prophylaxis: deaths from any cause	0 of 40	0 of 79	119	NCT03417245	Trial comparisons
Factor On-demand vs Fitusiran 80 mg Prophylaxis: serious adverse events	5 of 40	5 of 79	119	NCT03417245	Trial comparisons
Pre-prophylaxis vs Prophylaxis: deaths from any cause	1 of 34	1 of 51	85	NCT02141074	Trial comparisons
Pre-prophylaxis vs Prophylaxis: serious adverse events	9 of 34	19 of 51	85	NCT02141074	Trial comparisons
Pre-prophylaxis vs Prophylaxis: Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)	2 of 20	2 of 51	71	NCT02141074	Trial comparisons
Pre-prophylaxis vs Prophylaxis: Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)	2 of 19	2 of 47	66	NCT02141074	Trial comparisons
Pre-prophylaxis vs Prophylaxis: Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)	2 of 34	0 of 28	62	NCT02141074	Trial comparisons
Bypassing Agents (BPA) On-demand vs Fitusiran 80 mg Prophylaxis: deaths from any cause	0 of 19	0 of 41	60	NCT03417102	Trial comparisons
Bypassing Agents (BPA) On-demand vs Fitusiran 80 mg Prophylaxis: serious adverse events	5 of 19	7 of 41	60	NCT03417102	Trial comparisons
Original Dose Regimen (SAS 1) vs AT-Based Dose Regimen (SAS 2): Number of Participants With Potentially Clinically Significant Abnormality: Physical Examination	17 of 34	2 of 18	52	NCT02554773	Trial comparisons

1 to 10 of 17

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Papers from its trials44

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
32369845	RESULT	1	PubMed
28833808	RESULT	1	PubMed
27352908	RESULT	1	PubMed
27174727	RESULT	1	PubMed
26970716	RESULT	1	PubMed
25641556	RESULT	1	PubMed
24697870	RESULT	1	PubMed
24304002	RESULT	1	PubMed
23834666	RESULT	1	PubMed
15230949	RESULT	1	PubMed

1 to 10 of 44

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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_0010604

GET https://conductscience.org/api/diseases/v1/MONDO_0010604/drugs?page=2

API reference For AI assistants