Iptacopan hydrochloride monohydrateProduct ingredient for Iptacopan
- Name
- Iptacopan hydrochloride monohydrate
- Drug Entry
- Iptacopan
Iptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor B.1 Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase.2 This is of particular importance to PNH, where one of the disease hallmarks is the mutation of the PIGA gene. Due to this mutation, all progeny erythrocytes will lack the glycosyl phosphatidylinositol–anchored proteins that normally anchor 2 membrane proteins, CD55 and CD59, that protect blood cells against the alternative complement pathway.3 Additionally, iptacopan has the benefit of targeting factor B, which only affect the alternative complement pathway, leaving the classic and lectin pathway untouched for the body to still mount adequate immune responses against pathogens.2
On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. This approval was based on favorable results obtained from the phase III APPL-PNH and APPOINT-PNH studies, where 82.3% and 77.5% of patients experienced a sustained hemoglobin improvement without transfusions respectively.5
- Accession Number
- DBSALT003493
- Structure
- Synonyms
- Iptacopan HCl / Iptacopan hydrochloride
- External IDs
- LNP-023 hydrochloride monohydrate / LNP-023-AAB / LNP023-AAB
- UNII
- XW5CK7C6YH
- CAS Number
- 2447007-60-3
- Weight
- Average: 477.0
Monoisotopic: 476.2077999 - Chemical Formula
- C25H33ClN2O5
- InChI Key
- JUWBBUFSAGEROP-VVJLZRNGSA-N
- InChI
- InChI=1S/C25H30N2O4.ClH.H2O/c1-4-31-19-10-12-27(22(14-19)17-5-7-18(8-6-17)25(28)29)15-21-20-9-11-26-24(20)16(2)13-23(21)30-3;;/h5-9,11,13,19,22,26H,4,10,12,14-15H2,1-3H3,(H,28,29);1H;1H2/t19-,22-;;/m0../s1
- IUPAC Name
- 4-[(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl]benzoic acid hydrate hydrochloride
- SMILES
- O.Cl.CCO[C@H]1CCN(CC2=C(OC)C=C(C)C3=C2C=CN3)[C@@H](C1)C1=CC=C(C=C1)C(O)=O
- External Links
- ChemSpider
- 129235270
- Predicted Properties
Property Value Source logP 1.4 Chemaxon pKa (Strongest Acidic) 3.7 Chemaxon pKa (Strongest Basic) 8.75 Chemaxon Physiological Charge 0 Chemaxon Hydrogen Acceptor Count 5 Chemaxon Hydrogen Donor Count 2 Chemaxon Polar Surface Area 74.79 Å2 Chemaxon Rotatable Bond Count 7 Chemaxon Refractivity 121.95 m3·mol-1 Chemaxon Polarizability 47.46 Å3 Chemaxon Number of Rings 4 Chemaxon Bioavailability 1 Chemaxon Rule of Five Yes Chemaxon Ghose Filter Yes Chemaxon Veber's Rule No Chemaxon MDDR-like Rule Yes Chemaxon