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Trade names | Fabhalta |
Other names | LNP023 |
AHFS/Drugs.com | Fabhalta |
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Routes of administration | By mouth |
Drug class | Complement factor B inhibitor |
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Chemical and physical data | |
Formula | C25H30N2O4 |
Molar mass | 422.525 g·mol−1 |
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Iptacopan, sold under the brand name Fabhalta, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria and proteinuria. [6] It is a complement factor B inhibitor that was developed by Novartis. [6] It is taken by mouth. [6]
Iptacopan was approved for medical use in the United States in December 2023, [6] [8] and in the European Union in May 2024. [7] The US Food and Drug Administration considers it to be a first-in-class medication. [9]
Iptacopan is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria; [6] [10] and to reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression, generally a urine protein-to-creatinine ratio ≥1.5 g/g; [6] for the treatment of adults with complement 3 glomerulopathy, to reduce proteinuria. [6]
Complement 3 glomerulopathy is a rare disease that causes inflammation and damage to the kidney glomeruli, which are responsible for filtering blood and producing urine. [11]
Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, generation of downstream effectors, and the amplification of the terminal pathway. [6]
In paroxysmal nocturnal hemoglobinuria, intravascular hemolysis is mediated by the downstream membrane attack complex, while extravascular hemolysis is facilitated by C3b opsonization. [6] Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated extravascular hemolysis and terminal complement mediated intravascular hemolysis. [6]
The US Food and Drug Administration label for iptacopan contains a boxed warning for the risk of serious and life-threatening infections caused by encapsulated bacteria, including Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B. [6]
Iptacopan was approved by the US Food and Drug Administration (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023. [12]
In March 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Fabhalta, intended for the treatment of paroxysmal nocturnal hemoglobinuria. [7] [13] The applicant for this medicinal product is Novartis Europharm Limited. [7] Iptacopan was authorized for medical use in the European Union in May 2024. [7]
In August 2024, the US FDA granted accelerated approval to iptacopan for reducing proteinuria in adults with primary IgA nephropathy. [14]
In March 2025, the US FDA approved iptacopan for the treatment of adults with complement 3 glomerulopathy to reduce proteinuria. [11]
In a clinical study with twelve participants, iptacopan as a single drug led to the normalization of hemolytic markers in most patients, and no serious adverse events occurred during the 12-week study. [15] [16]
Iptacopan is also investigated as a drug in other complement-mediated diseases, like age-related macular degeneration and some types of glomerulopathies. [17]