Novartis has announced that the US Food and Drug Administration (FDA) has granted traditional approval to Fabhalta (iptacopan) for adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The approval allows the medicine to be used to slow the decline of kidney function in patients living with the rare autoimmune kidney disease.
Fabhalta previously received accelerated approval from the FDA in August 2024 to reduce protein levels in the urine, a key sign of kidney damage in people with IgA nephropathy. The latest decision converts that accelerated approval into full approval after additional clinical data confirmed the medicine’s long-term benefits.
IgA nephropathy, also known as Berger’s disease, is one of the most common autoimmune kidney diseases worldwide. It occurs when an antibody called immunoglobulin A (IgA) builds up in the kidneys, causing inflammation and gradually damaging the organs. Over time, the disease can reduce the kidneys’ ability to filter waste from the blood and may eventually lead to kidney failure.
Each year, around 25 people per million worldwide are newly diagnosed with IgA nephropathy. Patients with persistent protein in their urine face a high risk of disease progression, with up to half developing kidney failure within 10 to 20 years of diagnosis. Many of these patients eventually require dialysis or a kidney transplant, placing a significant physical, emotional and financial burden on individuals and their families.
Fabhalta is the first medicine in its class to target the complement system, part of the body’s immune response that plays an important role in the development of IgA nephropathy. By blocking this pathway, the treatment aims to slow ongoing kidney damage rather than only managing symptoms.
The FDA approval is based on results from the Phase III APPLAUSE-IgAN clinical trial. The study showed that patients treated with Fabhalta experienced a significantly slower decline in kidney function over two years compared with those receiving a placebo. Researchers measured kidney function using the estimated glomerular filtration rate (eGFR), a standard test used to assess how well the kidneys are working.
Patients taking Fabhalta recorded an average annual decline in eGFR of 3.0 mL/min/1.73 m², compared with 5.7 mL/min/1.73 m² in the placebo group. The medicine also performed better than placebo across several important kidney health measures, demonstrating both statistical and clinical benefits.
Dr. Dana Rizk, Professor of Medicine in the Division of Nephrology at the University of Alabama at Birmingham and a member of the APPLAUSE-IgAN Steering Committee, said slowing the loss of kidney function is one of the most important goals in treating IgA nephropathy. She added that the approval highlights the importance of targeting the underlying disease process, including complement activation, to help preserve kidney health.
Bonnie Schneider, Director and Co-Founder of the IgA Nephropathy Foundation, described the approval as an encouraging milestone for patients and families affected by the disease. She said the availability of a treatment that can help protect kidney function offers renewed hope for the IgAN community.
The study also found that Fabhalta had a safety profile consistent with previous clinical studies. The most commonly reported side effects included abdominal pain, dizziness and nausea. Because the medicine may increase the risk of serious infections caused by certain bacteria, patients must receive recommended vaccinations before starting treatment. Fabhalta is available in the United States through a Risk Evaluation and Mitigation Strategy (REMS) program designed to ensure safe use.
Victor Bultó, President of Novartis US, said the approval reinforces the company’s commitment to developing innovative treatments that address the root causes of kidney disease. He noted that preserving kidney function is an outcome that is especially important for patients at risk of long-term kidney damage.
Novartis said it is also supporting patients through access programs, with nearly all eligible US patients expected to pay $10 or less per month for Fabhalta. The company continues to expand its kidney disease portfolio, which also includes Vanrafia (atrasentan) and the investigational therapy zigakibart, as it works to improve treatment options for people living with IgA nephropathy.