Novartis Advances in IgA Nephropathy Treatment with Positive Phase 3 Results for Fabhalta

Novartis has reported significant progress in its efforts to treat IgA nephropathy (IgAN), a rare kidney disease, with positive final results from a Phase 3 trial of its drug Fabhalta. The Swiss pharmaceutical giant is now poised to seek full regulatory approval, potentially strengthening its position in an increasingly competitive market for IgAN treatments.

Fabhalta Demonstrates Efficacy in Slowing Kidney Function Decline

The Phase 3 Applause-IgAN trial, which compared twice-daily doses of Fabhalta (iptacopan) with a placebo over two years, has shown that the drug can significantly slow the decline of kidney function in IgAN patients. The study measured kidney function using estimated glomerular filtration rate (eGFR) slopes, a key indicator of disease progression.

Novartis plans to leverage these data for regulatory submissions next year, aiming to secure full, traditional approval from the U.S. Food and Drug Administration (FDA). Fabhalta had previously received accelerated approval for IgAN in April 2024, based on its ability to reduce proteinuria at nine months.

Expanding Indications and Market Potential

Fabhalta, a complement factor B inhibitor, has already gained FDA approval for multiple indications:

• Paroxysmal nocturnal hemoglobinuria (PNH) in 2023
• IgA nephropathy (accelerated approval) in 2024
• C3 glomerulopathy in 2025

The drug's commercial performance has been promising, with sales reaching $120 million in the second quarter of 2025. Novartis has identified Fabhalta as one of its "priority brands" and has ongoing studies to potentially expand its use to at least three more indications in the coming years.

Competitive Landscape in IgAN Treatment

The positive results for Fabhalta come at a time of intense focus on IgAN within the pharmaceutical industry. Several companies are advancing new therapies targeting the disease:

• Travere Therapeutics' Filspari, approved to slow kidney function decline in adults with primary IgAN at risk of progression
• Calliditas Therapeutics' Tarpeyo, cleared for patients at risk of disease progression
• Otsuka Pharmaceutical's sibeprenlimab, which could receive FDA approval within weeks

Novartis has further bolstered its IgAN portfolio through the acquisition of Chinook Therapeutics for up to $3.5 billion in 2023. This deal brought two additional experimental IgAN drugs into Novartis' pipeline, including Vanrafia, which received FDA approval for IgAN in April 2025.

As the understanding of IgAN biology improves and new approaches targeting immune-mediated drivers of the disease emerge, the competition in this space is likely to intensify. With Fabhalta's latest results and its expanding indications, Novartis appears well-positioned to maintain a strong presence in the evolving IgAN treatment landscape.