Iptacopan
Iptacopan is a small molecule pharmaceutical. It is currently being investigated in clinical studies. It is known to target complement factor B.
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Therapeutic Areas
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Trade Name
FDA
EMA
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Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
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Labels
FDA
EMA
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Indications
FDA
EMA
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Agency Specific
FDA
EMA
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Patent Expiration
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ATC Codes
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HCPCS
No data
Clinical
Clinical Trials
21 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Paroxysmal hemoglobinuria | D006457 | Orphanet_447 | D59.12 | — | 3 | 4 | — | — | 7 |
| Iga glomerulonephritis | D005922 | EFO_0004194 | — | 1 | 2 | — | — | 3 | |
| Atypical hemolytic uremic syndrome | D065766 | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Glomerulonephritis | D005921 | N05 | — | 2 | — | — | 1 | 3 | |
| Membranous glomerulonephritis | D015433 | EFO_0004254 | N03.2 | — | 1 | — | — | — | 1 |
| Lupus nephritis | D008181 | EFO_0005761 | — | 1 | — | — | — | 1 | |
| Macular degeneration | D008268 | EFO_0001365 | H35.30 | — | 1 | — | — | — | 1 |
| Thrombocytopenic purpura idiopathic | D016553 | EFO_0007160 | D69.3 | — | 1 | — | — | — | 1 |
| Hemolytic anemia autoimmune | D000744 | EFO_1001264 | D59.10 | — | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Hepatic insufficiency | D048550 | 1 | — | — | — | — | 1 |
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | IPTACOPAN |
| INN | iptacopan |
| Description | Iptacopan (LNP023) is a drug developed by Novartis designed to treat paroxysmal nocturnal hemoglobinuria (PNH), a disease in which the innate immune system destroys red blood cells. It is the first drug that selectively inhibits factor B, the active component of the complement's C3 and C5 convertases. In contrast to other PNH treatments like eculizumab, iptacopan is a small molecule.
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| Classification | Small molecule |
| Drug class | complement receptor antagonists/complement inhibitors |
| Image (chem structure or protein) | |
| Structure (InChI/SMILES or Protein Sequence) | CCO[C@H]1CCN(Cc2c(OC)cc(C)c3[nH]ccc23)[C@H](c2ccc(C(=O)O)cc2)C1 |
Identifiers
| PDB | — |
| CAS-ID | 1644670-37-0 |
| RxCUI | — |
| ChEMBL ID | CHEMBL4594448 |
| ChEBI ID | — |
| PubChem CID | 90467622 |
| DrugBank | — |
| UNII ID | 8E05T07Z6W (ChemIDplus, GSRS) |
Target
Agency Approved
No data
Alternate
CFB
CFB
Organism
Homo sapiens
Gene name
CFB
Gene synonyms
BF, BFD
NCBI Gene ID
Protein name
complement factor B
Protein synonyms
B-factor, properdin, C3 proaccelerator, C3 proactivator, C3/C5 convertase, GBG, Glycine-rich beta glycoprotein, glycine-rich beta-glycoprotein, PBF2, Properdin factor B
Uniprot ID
Mouse ortholog
Cfb (14962)
complement factor B (P04186)
Variants
Clinical Variant
No data
Financial
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Trends
PubMed Central
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
0 adverse events reported
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