FDA Approves Novartis Drug for Rare Kidney Disease Treatment

Core Insights - Novartis (NVS) received FDA approval for Fabhalta (iptacopan) for treating adults with C3 glomerulopathy (C3G), marking it as the first and only approved treatment for this rare kidney disease [1][6] - The approval is supported by data from the phase III APPEAR-C3G study, which demonstrated significant proteinuria reduction in patients [2][3] - Novartis shares have increased by 20.3% over the past year, contrasting with a 2% decline in the large-cap pharmaceutical industry [2][6] Drug Development and Market Performance - Fabhalta is an oral Factor B inhibitor, and prior to this approval, patients relied on supportive care and immunosuppression for C3G [2] - The APPEAR-C3G study showed clinically meaningful proteinuria reduction as early as 14 days and sustained at 12 months [3] - Fabhalta also received approvals for other indications, including paroxysmal nocturnal hemoglobinuria (PNH) and primary IgA nephropathy (IgAN) [4][5] Financial Performance and Future Outlook - Novartis reported fourth-quarter 2024 results that exceeded expectations, with net sales projected to grow in the mid to high single digits for 2025 [9] - The company is focusing on label expansions of existing drugs to counteract generic competition and is pursuing strategic acquisitions to enhance its pipeline [10][11] - An acquisition of Anthos Therapeutics for $925 million upfront, with potential additional payments of up to$2.15 billion, is expected to strengthen Novartis' position in the market [12] Pipeline and Strategic Initiatives - Novartis is advancing late-stage development of additional therapies for IgAN, including atrasentan and zigakibart [8] - The company is evaluating Fabhalta for a range of rare kidney diseases, indicating a broad strategy to address unmet medical needs [7]