Disease · MONDO_0100244

paroxysmal nocturnal hemoglobinuria

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. paroxysmal nocturnal hemoglobinuria. Diseases, record MONDO_0100244. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0100244

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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: paroxysmal nocturnal hemoglobinuria; Open Targets
Open Targets ID: MONDO_0100244; Open Targets
Other names: Marchiafava-Micheli disease, PNH, acquired paroxysmal nocturnal hemoglobinuria, hereditary paroxysmal nocturnal hemoglobinuria, inherited paroxysmal nocturnal hemoglobinuria, paroxysmal hemoglobinuria, paroxysmal nocturnal haemoglobinuria; Open Targets
Disease Ontology: DOID:0060284; Disease Ontology
NCI Thesaurus: C61233; NCI Thesaurus
Orphanet: ORPHA:447; Orphanet
Highest stage of its drugs: Approval, for 7 of its 33 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs33

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
ECULIZUMAB	CHEMBL1201828	Antibody	Approval	ClinicalTrials.gov, DailyMed, EMA Human Drugs and PMDA	34	27	27	Drugs and targets
RAVULIZUMAB	CHEMBL3989986	Antibody	Approval	DailyMed, EMA Human Drugs, FDA, PMDA and TTD	18	12	12	Drugs and targets
IPTACOPAN	CHEMBL4594448	Small molecule	Approval	EMA Human Drugs and TTD	14	11	11	Drugs and targets
DANICOPAN	CHEMBL4250860	Small molecule	Approval	EMA Human Drugs and PMDA	14	10	10	Drugs and targets
PEGCETACOPLAN	CHEMBL4298211	Protein	Approval	EMA Human Drugs, FDA, PMDA and TTD	13	8	8	Drugs and targets
CROVALIMAB	CHEMBL4594316	Antibody	Approval	EMA Human Drugs and PMDA	7	4	4	Drugs and targets
IPTACOPAN HYDROCHLORIDE	CHEMBL5095401	Small molecule	Approval	FDA and PMDA	3	0	0	Drugs and targets
POZELIMAB	CHEMBL4298086	Antibody	Phase 3	ClinicalTrials.gov	11	9	9	Drugs and targets
CEMDISIRAN	CHEMBL4297722	Oligonucleotide	Phase 3	ClinicalTrials.gov	10	8	8	Drugs and targets
NOMACOPAN	CHEMBL4297928		Phase 3	ClinicalTrials.gov and TTD	5	4	3	Drugs and targets

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Targets15

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
C5	Complement C5	8	Complement C5 binding agent; Complement C5 inhibitor; Complement C5 mRNA RNAi inhibitor	Approval	https://conductscience.org/library/genes/727	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
CFB	Complement factor B	2	Complement factor B inhibitor	Approval	https://conductscience.org/library/genes/629	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	2	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
C3	Complement C3	1	Complement C3 inhibitor	Approval	https://conductscience.org/library/genes/718	Drugs and targets
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 1/2	https://conductscience.org/library/genes/1043	Drugs and targets
CFD	Complement factor D	1	Complement factor D inhibitor	Approval	https://conductscience.org/library/genes/1675	Drugs and targets
DHFR	Dihydrofolate reductase	1	Dihydrofolate reductase inhibitor	Phase 2	https://conductscience.org/library/genes/1719	Drugs and targets
IMPDH1	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3614	Drugs and targets
IMPDH2	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3615	Drugs and targets

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Trials93

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

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT02946463	ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3	272	2019-02-12	Paroxysmal Nocturnal Hemoglobinuria (PNH)	ECULIZUMAB, RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT04434092	A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors	Phase 3	210	2025-11-10	Paroxysmal Nocturnal Hemoglobinuria	CROVALIMAB, ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00003838	Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow	Phase 2	202	2022-10-07	Myeloproliferative Disorders, Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia	CYCLOSPORINE, METHOTREXATE	Its record names the disease	ClinicalTrials.gov
NCT03056040	ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab	Phase 3	202	2019-03-21	Paroxysmal Nocturnal Hemoglobinuria (PNH)	ECULIZUMAB, RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00122317	Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3	187	2017-06-05	Paroxysmal Hemoglobinuria, Nocturnal	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03748823	Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab	Phase 3	139	2022-10-20	Paroxysmal Nocturnal Hemoglobinuria	RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00587054	Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders	Phase 2	129	2017-03-10	Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH)	FLUDARABINE, THIOTEPA	Its record names the disease	ClinicalTrials.gov
NCT04558918	Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment	Phase 3	97	2023-11-28	Paroxysmal Nocturnal Hemoglobinuria (PNH)	IPTACOPAN	Its record names the disease	ClinicalTrials.gov
NCT04469465	Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)	Phase 3	86	2023-07-24	Paroxysmal Nocturnal Hemoglobinuria	DANICOPAN, ECULIZUMAB, RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03500549	Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3	80	2022-03-25	Paroxysmal Nocturnal Hemoglobinuria	PEGCETACOPLAN	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials38

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
Ravulizumab IV/SC Treatment Group vs Ravulizumab SC/SC Treatment Group: deaths from any cause	2 of 45	2 of 84	129	NCT03748823	Trial comparisons
Ravulizumab IV/SC Treatment Group vs Ravulizumab SC/SC Treatment Group: serious adverse events	16 of 45	30 of 84	129	NCT03748823	Trial comparisons
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants That Remained Disease-free Survival	26 of 43	56 of 57	100	NCT00003838	Trial comparisons
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants Who Experienced Transplant Related Mortality	5 of 43	4 of 57	100	NCT00003838	Trial comparisons
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants Overall Survival	17 of 43	48 of 57	100	NCT00003838	Trial comparisons
SB12 vs Soliris: deaths from any cause	0 of 47	1 of 47	94	NCT04058158	Trial comparisons
SB12 vs Soliris: serious adverse events	3 of 47	2 of 47	94	NCT04058158	Trial comparisons
Overall Pegcetacoplan vs Standard of Care: deaths from any cause	1 of 46	1 of 18	64	NCT04085601	Trial comparisons
Overall Pegcetacoplan vs Standard of Care: serious adverse events	6 of 46	3 of 18	64	NCT04085601	Trial comparisons
Pegcetacoplan vs Standard of Care: Number of Subjects With an Hb Response in the Absence of Transfusions	25 of 35	1 of 18	53	NCT04085601	Trial comparisons

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Papers from its trials63

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
38030318	RESULT	1	PubMed
37534517	RESULT	1	PubMed
35561315	RESULT	1	PubMed
33765419	RESULT	1	PubMed
33121236	RESULT	1	PubMed
31840801	RESULT	1	PubMed
23934275	RESULT	1	PubMed
21222185	RESULT	1	PubMed
16990386	RESULT	1	PubMed
42287326	DERIVED	1	PubMed

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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_0100244

GET https://conductscience.org/api/diseases/v1/MONDO_0100244/drugs?page=2

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