Novartis has announced positive final results from the APPLAUSE-IgAN Phase 3 trial, revealing that its oral complement inhibitor Fabhalta (iptacopan) significantly slowed kidney function decline in adults with IgA nephropathy (IgAN), a progressive autoimmune kidney disease. Over two years, Fabhalta demonstrated a statistically significant and clinically meaningful improvement in estimated glomerular filtration rate (eGFR) slope versus placebo—a key marker for kidney function and disease progression.
IgAN affects roughly 25 people per million worldwide annually, leading to glomerular inflammation, proteinuria, and, in up to half of patients with persistent proteinuria, progressive renal failure over 10 to 20 years. Current supportive care offers no targeted intervention to halt disease progression, making innovative therapies urgently needed.
Fabhalta is the first and only approved complement pathway inhibitor in adults with IgAN, having received accelerated approval in the United States in 2024 based on proteinuria reduction. The recent data now support a traditional FDA submission in 2026. In the pivotal APPLAUSE-IgAN study, Fabhalta was well tolerated with a favorable safety profile, aligning with prior clinical findings in rare kidney diseases and supporting its use in a broad nephrology portfolio.
Novartis is positioning Fabhalta as a targeted therapy offering long-term kidney protection and improved prospects for patients, while advancing additional IgAN candidates. Full study data will be presented at upcoming scientific meetings, and regulatory filings are planned for next year.
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