FDA Grants Full Approval to Iptacopan for IgA Nephropathy

The FDA has granted full approval to iptacopan (Fabhalta) to slow the decline in kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The approval provides a new treatment option aimed at reducing the risk of worsening kidney disease in this chronic condition.

 Fabhalta, a first-in-class complement inhibitor, received approval under a priority review designation after an initial FDA accelerated approval in August 2024 for the reduction of proteinuria in primary IgAN1.

“IgAN is a chronic, immune-mediated disease leading to kidney failure that can have a severe impact on patients’ lives,” said Dana Rizk, M.D., Professor of Medicine in the Division of Nephrology at the University of Alabama at Birmingham and APPLAUSE-IgAN Steering Committee Member. “The ability to significantly slow kidney function decline is a critical treatment goal. This approval of Fabhalta reinforces the importance of targeting underlying disease mechanisms, including complement activation, in treating IgAN to help preserve kidney health.”

Each year, approximately 25 people per million worldwide are newly diagnosed with IgAN, one of the most common autoimmune kidney diseases. Up to 50 percent of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis and/or kidney transplantation, placing a significant burden on patients.

“This milestone is a moment of great hope for the IgAN community,” said Bonnie Schneider, Director and Co-Founder, IgA Nephropathy Foundation. “For patients and families impacted by this progressive disease, knowing that Fabhalta can help preserve kidney function brings renewed confidence and optimism for the future of the IgAN treatment landscape.”

Data supporting approval

The approval of Fabhalta was based on data from the Phase III APPLAUSE-IgAN study. Results demonstrated statistically significant and clinically meaningful improvement in estimated glomerular filtration rate (eGFR) over two years, with Fabhalta showing an annualized mean change from baseline in eGFR of -3.0 mL/min/1.73 m2/yr compared with -5.7 mL/min/1.73 m2/yr for placebo1. Fabhalta consistently outperformed placebo across key kidney outcomes.

The APPLAUSE-IgAN study showed that Fabhalta has a favorable safety profile, consistent with previously reported data. The most common adverse events with Fabhalta in patients with IgAN were abdominal pain, dizziness and nausea1. Fabhalta may increase the risk of serious infections caused by encapsulated bacteria and is available only through a Risk Evaluation and Mitigation Strategy (REMS) program requiring appropriate vaccinations prior to treatment.

Transforming care in kidney disease

“Today’s approval reinforces Fabhalta’s role in preserving kidney function by significantly slowing disease progression, an outcome that matters deeply to patients at risk of long-term kidney damage,” said Victor Bultó, President, US, Novartis. “This milestone underscores the importance of continued innovation for people living with IgAN and our commitment to addressing the underlying drivers of disease.”

As each person’s IgAN journey is unique, access to effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for their patients. Alongside Fabhalta, Novartis is supporting this community through its growing IgAN portfolio, which includes Vanrafia® (atrasentan) and investigational compound zigakibart. Novartis is committed to helping IgAN patients access Fabhalta through a variety of support programs, with nearly 100 percent of US patients paying $10 or less per month.

About Fabhalta® (iptacopan)

Fabhalta (iptacopan) is an oral Factor B inhibitor designed to selectively target the alternative complement pathway, one of several key drivers of glomerular inflammation and kidney damage in IgAN. By inhibiting Factor B, Fabhalta aims to reduce ongoing complement-mediated injury and slow disease progression. Fabhalta has received regulatory approvals in multiple complement-mediated diseases, including IgAN, and is being evaluated across a range of rare kidney conditions.