Iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria
Sign Up to like & getrecommendations! Published in 2024 at "Expert Opinion on Pharmacotherapy"
DOI: 10.1080/14656566.2024.2404110
Abstract: ABSTRACT Introduction Standard-of-care first-line treatments for paroxysmal nocturnal hemoglobinuria (PNH) include the anti-C5 therapies eculizumab and ravulizumab. However, persistent anemia, likely due to extravascular hemolysis, and reduced quality of life (QoL) due to frequent infusions… read more here.
Keywords: treatment; paroxysmal nocturnal; iptacopan; nocturnal hemoglobinuria ... See more keywords
#456 Efficacy and safety of iptacopan in patients with primary IgA nephropathy: interim analysis results of the Phase 3 APPLAUSE-IgAN study
Sign Up to like & getrecommendations! Published in 2024 at "Nephrology Dialysis Transplantation"
DOI: 10.1093/ndt/gfae069.141
Abstract: Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis. An unmet need for targeted disease-modifying treatments addressing its pathophysiology remains. There is compelling evidence for involvement of the alternative complement pathway (AP) in IgAN.… read more here.
Keywords: treatment; applause igan; iptacopan; placebo ... See more keywords
#3018 Iptacopan's rapid and sustained inhibition of overactive complement alternative pathway in C3G: Exploratory analysis from the Phase 3 APPEAR-C3G study
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DOI: 10.1093/ndt/gfaf116.056
Abstract: Primary C3 glomerulopathy (C3G) is an ultra-rare glomerulonephritis driven by overactivation of the alternative complement pathway (AP). Iptacopan (LNP023) is an oral, proximal complement inhibitor that targets Factor B, thereby selectively inhibiting the AP of… read more here.
Keywords: month; serum; c3g; iptacopan ... See more keywords
#2379 Predicting long-term benefits of improvements in estimated glomerular filtration rate (eGFR) with iptacopan on delaying kidney failure in complement 3 glomerulopathy (C3G)
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DOI: 10.1093/ndt/gfaf116.1296
Abstract: Complement 3 glomerulopathy (C3G) is an ultra-rare glomerulonephritis that leads to progressive loss of renal function. Progression to kidney failure occurs in up to 50% of adults within 10 years of diagnosis. Current treatments do… read more here.
Keywords: kidney failure; egfr slope; c3g; iptacopan ... See more keywords
Early Insights into Real-World Iptacopan Use Among Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States: The Apprise-PNH Data Platform
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DOI: 10.1182/blood-2024-194425
Abstract: Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening hematological disorder affecting approximately 24 people per million in the United States (US). Iptacopan, the first orally administered monotherapy for PNH, received US Food and… read more here.
Keywords: data platform; real world; iptacopan; pnh ... See more keywords
Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program
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DOI: 10.1182/blood-2025-3202
Abstract: Iptacopan, the first-in-class oral complement factor B inhibitor that selectively inhibits the alternative pathway of the complement system, has been approved for the treatment (tx) of patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH). In 2023,… read more here.
Keywords: rr1 rr2; paroxysmal nocturnal; iptacopan; program ... See more keywords
A Practical Method for Synthesizing Iptacopan
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DOI: 10.3390/molecules29102289
Abstract: Iptacopan, the first orally available small-molecule complement factor B inhibitor, was developed by Novartis AG of Switzerland. Iptacopan for the treatment of PNH was just approved by the FDA in December 2023. Other indications for… read more here.
Keywords: method synthesizing; practical method; iptacopan; synthesizing iptacopan ... See more keywords