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摘要
En 中文
Iptacopan (FABHALTA (R)) is an oral complement Factor B inhibitor developed by Novartis Pharmaceuticals for the treatment of complement-mediated diseases. Acting upstream of complement 5 in the alternative pathway, iptacopan inhibits both terminal complement-mediated intravascular haemolysis and complement 3-mediated extravascular haemolysis. On 5 December 2023, iptacopan received approval in the USA for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). This article summarizes the milestones in the development of iptacopan leading to this first approval for PNH.
Keyword:
PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA
LONG-TERM EFFICACY
FACTOR B INHIBITOR
OPEN-LABEL
ECULIZUMAB
HEMOLYSIS
SAFETY
MONOTHERAPY
PHASE-2
PNH
期刊
IF:
14.4
论文数:
8.4K
被引数:
2.3W
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Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial.png)
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LANCET HAEMATOLOGYIF17.7
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收藏The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient surveyANNALS OF HEMATOLOGY
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ANNALS OF HEMATOLOGYIF2.4
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