FDA decision expands the Fabhalta indication from reducing proteinuria to slowing kidney function decline in adults at risk of disease progression.
BASEL, Switzerland, July 17, 2026 – Novartis has announced that the U.S. Food and Drug Administration has granted traditional approval to Fabhalta® (iptacopan) for slowing kidney function decline in adults with primary immunoglobulin A nephropathy, or IgAN, who are at risk of disease progression.
The decision establishes Fabhalta as the first FDA-approved complement inhibitor shown to significantly slow the loss of kidney function in this patient population. It follows the medicine’s accelerated approval in August 2024, which covered the reduction of proteinuria in adults with primary IgAN at risk of rapid progression.
The FDA reviewed the application under its priority review pathway. Traditional approval confirms the clinical benefit that had not yet been established when the earlier proteinuria-based indication was authorized.
Phase III Study Demonstrates Kidney Function Preservation
The traditional approval is supported by results from APPLAUSE-IgAN, a Phase III, randomized, double-blind and placebo-controlled study evaluating the efficacy and safety of iptacopan in people with primary IgAN.
Over the two-year study period, Fabhalta reduced the rate of estimated glomerular filtration rate, or eGFR, decline by approximately 48% compared with placebo. eGFR is an important measurement used to assess how effectively the kidneys filter waste from the blood.
Patients receiving Fabhalta experienced an annualized mean eGFR change of approximately minus 3.0 mL/min/1.73 m² per year. By comparison, participants receiving placebo experienced an annualized mean decline of approximately minus 5.7 mL/min/1.73 m² per year.
These findings suggest that treatment with Fabhalta may help preserve kidney function and delay disease progression in appropriately selected adults with primary IgAN.
Researchers also observed improvements in proteinuria as early as two weeks after treatment began. The reduction was maintained throughout the treatment period. Proteinuria, or excess protein in the urine, is an important marker of kidney damage and disease progression.
Addressing An Important Treatment Goal
Primary IgAN is a chronic, immune-mediated kidney disease caused by the accumulation of abnormal immunoglobulin A deposits in the kidneys. These deposits may trigger inflammation and gradually damage the glomeruli, the small filtering structures responsible for removing waste and excess fluid from the blood.
The disease can lead to persistent proteinuria, declining kidney function and, in some patients, kidney failure. Published estimates cited by Novartis suggest that up to half of patients with persistent proteinuria may progress to kidney failure within 10 to 20 years of diagnosis.
Until recently, many IgAN treatments focused mainly on controlling blood pressure, reducing proteinuria and managing inflammation. Newer targeted medicines are designed to address biological pathways that contribute directly to kidney injury.
Slowing eGFR decline is considered a particularly important treatment objective because it may help delay the need for dialysis or kidney transplantation.
How Fabhalta Works?
Fabhalta is an oral medicine that selectively inhibits Factor B, a protein involved in the alternative complement pathway.
The complement system forms part of the body’s immune defenses. However, excessive or uncontrolled activation of the alternative complement pathway may contribute to inflammation and kidney damage in people with IgAN.
By inhibiting Factor B, iptacopan is designed to reduce complement-mediated injury while preserving activity in other parts of the immune system. The treatment is taken orally, offering a targeted approach to a pathway associated with IgAN progression.
Results from the APPLAUSE-IgAN program provide evidence that targeting this pathway can produce both an early reduction in proteinuria and a longer-term effect on kidney function decline.
Safety Findings And Infection Risk
The safety profile observed during the two-year APPLAUSE-IgAN study was generally consistent with previously reported Fabhalta findings.
The most commonly reported adverse reactions among patients with IgAN included abdominal pain, dizziness and nausea. However, complement inhibition can reduce the body’s ability to defend itself against certain encapsulated bacteria.
Fabhalta may therefore increase the risk of serious and potentially life-threatening infections, including infections caused by Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type B.
Patients should receive the recommended vaccinations before beginning treatment, when possible. Fabhalta is available in the United States only through a Risk Evaluation and Mitigation Strategy program intended to support appropriate prescribing and infection-risk management.
Patients should not start, stop or change treatment without consulting a qualified healthcare professional. They should also seek immediate medical attention when symptoms of a serious infection develop.
Expanding Targeted Treatment Options For IgAN
The traditional approval marks an important development in the changing IgAN treatment landscape. It gives nephrologists another targeted option for adults whose disease remains at risk of progression despite supportive care.
Because IgAN can involve several disease pathways, treatment decisions may depend on proteinuria levels, kidney function, disease progression, existing medical conditions and individual risk factors.
Fabhalta is part of Novartis’ broader kidney disease portfolio, which also includes Vanrafia® (atrasentan) and the investigational therapy zigakibart. The company is studying treatments that address different mechanisms involved in IgAN and other complement-mediated kidney disorders.
The July 2026 FDA decision converts Fabhalta’s IgAN indication from accelerated approval to traditional approval. It also confirms that the treatment’s benefit extends beyond lowering urinary protein to significantly slowing kidney function decline in adults with primary IgAN at risk of progression.