Accelerated Approval for IgA Nephropathy Treatment

Dana Rizk, MD, Professor of Medicine at the University of Alabama at Birmingham, discusses the U.S. Food and Drug Administration (FDA) accelerated approval of Fabhalta (iptacopan) for the reduction of proteinuria in adults with IgA nephropathy (IgAN).

IgAN is a rare kidney disorder that occurs when immunoglobulin A (IgA), a protein that helps the body fight infections, settles in the kidneys. In the early stages, IgA nephropathy has no symptoms. The first sign of this condition may be blood in the urine. End-stage kidney disease may develop. In most instances, the cause of this condition is unknown; however, certain disorders have been linked with IgA nephropathy, such as cirrhosis of the liver, celiac disease, and HIV infection. Familial IgA nephropathy is linked to genetic material on the long arm of chromosome 6.

Iptacopan is an oral Factor B inhibitor of the alternative complement pathway. It was first approved by the FDA in 2023 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Now, it is the first and only complement inhibitor approved for the reduction of proteinuria in adults with IgAN.
The accelerated approval follows an interim analysis of the phase 3 APPLAUSE-IgAN clinical trial, a multicenter, randomized, double-blind, placebo-controlled, parallel-group study evaluating the safety and efficacy of iptacopan in adults with primary IgAN. The analysis measured reduction in proteinuria at 9 months versus placebo, which was observed as a 44% reduction in proteinuria. This demonstrates a statistically significant reduction versus placebo of 38%.

Chapters:
Primary IgA Nephropathy Overview 00:00
Current Management 2:02
Fabhalta (Iptacopan) Overview 4:10
Accelerated Approval 6:00