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Name
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Iptacopan
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Synonyms
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4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid;
LNP023; Benzoic acid, 4-([(2S,4S)-4-ethoxy-1-(5-methoxy-7-methyl-1H-indol-4-yl)methyl]-2-piperidinyl)-; LNP023(LNP - 023); 4-((2S,4S)-4-Ethoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid, iptacopan |
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CAS
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1644670-37-0
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MF
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C₂₅H₃₀N₂O₄
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MW
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422.52
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EINECS
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202-303-5
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Boiling Point
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599.1±50.0 °C (Predicted)
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Density
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1.25±0.1 g/cm³ (Predicted)
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Storage Conditions
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Store at -20°C
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Solubility
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DMSO: 50.0 (maximum concentration mg/mL); 118.34 (maximum concentration mM)
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pKa (Acidity Coefficient)
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4.07±0.10 (Predicted)
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Form
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Solid
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Color
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Off - white to gray
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Usage
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Iptacopan is an oral complement Factor B inhibitor developed by Novartis. It targets the alternative complement pathway for the treatment of complement-mediated diseases.
Mechanism of Action
Iptacopan specifically binds to Factor B, inhibiting the formation of the C3 convertase precursor (C3bB). This blocks the amplification of the complement cascade, ultimately reducing the production of the membrane attack complex (MAC) and cell lysis.
Clinical Applications
As the world's first oral complement Factor B inhibitor, Iptacopan is currently approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) - a rare chronic complement-mediated hematological disease previously managed solely with intravenous anti-C5 antibodies (e.g., eculizumab).
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