Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G)

Core Insights - Novartis has received FDA approval for oral Fabhalta® (iptacopan), marking it as the first and only treatment for C3 glomerulopathy (C3G) [2][3][10] - The approval is seen as a historic milestone for the C3G community, providing a therapy that targets the underlying cause of the disease [3][4] - Fabhalta is an oral inhibitor of the alternative complement pathway, which was previously not available for C3G patients who relied on supportive care and immunosuppression [3][10] Company Developments - The pivotal Phase III APPEAR-C3G study demonstrated that Fabhalta significantly reduced proteinuria, with effects observable as early as 14 days and sustained over 12 months [5][6][10] - The safety profile of Fabhalta was favorable, with common adverse reactions including nasopharyngitis and viral infections, and it is available through a Risk Evaluation and Mitigation Strategy (REMS) [7][10] - This approval is the third for Fabhalta, following its previous approvals for primary immunoglobulin A nephropathy (IgAN) and paroxysmal nocturnal hemoglobinuria (PNH) [11][10] Industry Context - C3G is an ultra-rare kidney disease, typically diagnosed in young adults, with a poor prognosis where approximately 50% of patients progress to kidney failure within 10 years [4][10] - Novartis aims to transform care in kidney diseases, focusing on conditions with significant unmet needs and developing treatments that target the underlying causes of these diseases [16][10] - The company is also advancing other therapies for kidney diseases, including atrasentan and zigakibart, which are in late-stage development [12][10]