Immunoglobulin A nephropathy (IgAN) is a rare autoimmune kidney disease caused by an abnormal protein damaging the kidney’s filtering unit, resulting in lower kidney function. It’s estimated that 20-40% of people with IgAN will go on to develop kidney failure, and that up to 30% of people with chronic proteinuria (elevated levels of protein in their urine) will develop kidney failure within 10 years.

Fortunately, results of the phase 3 APPLAUSE-IgAN study indicate that the drug iptacopan (brand name: Fabhalta) may reduce proteinuria in patients living with IgAN, which would help improve the management and treatment of this disease.*

What the Researchers Did

For the study, 443 participants were randomized to receive either iptacopan (a drug approved for treating a rare blood disorder) or a placebo. Those who received iptacopan were given a 200 mg dose twice daily. 

The research team compared the effects of iptacopan to the placebo’s effects over the course of nine months, focusing on changes in levels of protein in the urine, both from 24-hour collections and from samples taken during the first morning void.

What They Found

The interim analysis revealed that iptacopan led to a:

• 38.3% reduction in 24-hour urine protein levels, and a
• 35.8% reduction from morning samples, compared to the placebo. 

In addition, twice as many patients on Iptacopan saw their protein levels drop below a key threshold that indicates better kidney health, compared to those on the placebo. 

The benefits of iptacopan were consistent across various patient groups, differing by:

• Sex, 
• Region, and 
• Severity of disease at the start of the study. 

The treatment was found to be generally safe, with most side effects being mild or moderate, and no patients required kidney replacement therapy during the trial period.

What It Means

These findings highlight the potential of iptacopan as a promising new treatment for IgAN, particularly in its ability to slow the progression of kidney disease and possibly prevent kidney failure, avoiding the need for dialysis and/or kidney transplantation. 

The targeted approach of iptacopan may offer a more effective treatment option compared to current broader immunosuppressive therapies. Full study results are expected in 2025. 

*Monaco, K. (2024, May 16). Blood Disorder Drug Cuts Proteinuria in Rare Kidney Disorder. MedPage Today. https://www.medpagetoday.com/meetingcoverage/nkf/110167 

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