Novartis(NVS) Prnewswire·2025-03-20 23:37Core Insights - Novartis announced FDA approval for oral Fabhalta® (iptacopan) as the first and only treatment for adults with C3 glomerulopathy (C3G) to reduce proteinuria [1][4][9] Company Developments - Fabhalta is the only oral inhibitor targeting the alternative complement pathway, believed to address the underlying cause of C3G, which previously had no approved treatments [2][8] - The approval is seen as a historic milestone for the C3G community, providing a potential new standard of care [2][3] - Novartis has received multiple approvals for Fabhalta, including for primary immunoglobulin A nephropathy (IgAN) and paroxysmal nocturnal hemoglobinuria (PNH) [9][10] Clinical Study Results - The Phase III APPEAR-C3G study demonstrated that Fabhalta significantly reduced proteinuria, with effects observable as early as 14 days and sustained over 12 months [4][5] - The study included a 6-month randomized, double-blind treatment period followed by a 6-month open-label period where all participants received Fabhalta [4][11] Market Context - C3G is an ultra-rare kidney disease, typically diagnosed in young adults, with a poor prognosis where approximately 50% progress to kidney failure within 10 years [3][12] - The average age of diagnosis is around 23 years, highlighting the urgent need for effective treatments in this demographic [3][12] Safety Profile - Fabhalta exhibited a favorable safety profile with no new safety signals identified; common adverse reactions included nasopharyngitis and viral infections [6][8] - The treatment is available only through a Risk Evaluation and Mitigation Strategy (REMS) due to the risk of serious infections [6][17] Future Directions - Novartis is advancing additional therapies for kidney diseases, including atrasentan and zigakibart, targeting conditions with high unmet needs [10][23] - The company aims to transform care in kidney health by addressing the underlying causes of diseases and improving patient outcomes [23]