On March 20, 2025, the U.S. Food and Drug Administration (FDA) approved Fabhalta (iptacopan), an oral therapy developed by Novartis Pharmaceuticals, for the treatment of adults with complement 3 glomerulopathy (C3G), a rare and progressive kidney disease. This marks the third FDA-approved indication for Fabhalta, following its earlier approvals for paroxysmal nocturnal hemoglobinuria (PNH) in December 2023 and primary immunoglobulin A nephropathy (IgAN) in August 2024.
Fabhalta works by inhibiting factor B, a key protein in the alternative complement pathway, which plays a crucial role in the excessive activation of the immune system seen in C3G. This immune dysregulation leads to kidney inflammation and proteinuria (protein leakage in the urine), contributing to progressive kidney damage. By targeting the root cause, Fabhalta helps reduce proteinuria and improve renal function.
The FDA’s latest approval is based on clinical trial results showing that Fabhalta significantly lowers protein levels in the urine, offering a new disease-modifying treatment option for patients with C3G. Its oral capsule formulation provides a more convenient alternative to traditional intravenous therapies, potentially improving patient compliance and quality of life. With limited treatment options for rare kidney diseases like C3G, Fabhalta’s approval represents a significant advancement in nephrology, giving new hope to patients at risk of kidney failure.
Disclaimer: Patients should consult their healthcare provider to understand the benefits, risks, and appropriateness of Fabhalta for their specific medical condition.
