Fabhalta becomes the first-ever approved treatment for C3 glomerulopathy, a breakthrough in kidney disease care

March 20, 2025 — Basel, Switzerland — Novartis has announced a major milestone with the FDA approval of Fabhalta® (iptacopan) for the treatment of C3 glomerulopathy (C3G) in adults. This marks the first and only FDA-approved therapy for C3G, an ultra-rare kidney condition that predominantly affects young adults and often leads to irreversible kidney failure.

What is C3G and why does this matter?

C3G is a progressive, complement-mediated kidney disease that disrupts the glomeruli—the kidney’s filtration system—due to overactivation of the alternative complement pathway. Affecting approximately 1–2 people per million annually, it is diagnosed primarily in individuals around the age of 23. Prior to this approval, patients had no specific treatment options beyond immunosuppressive therapies and symptom management.

“With this additional approval for Fabhalta, we are one step closer to transforming care for people with rare kidney diseases,” said Victor Bultó, President, US, Novartis. “We’re grateful to the patients and clinical teams whose participation made this approval possible.”

About Fabhalta: A First-in-Class Oral Therapy

Fabhalta is an oral, twice-daily therapy that works by selectively inhibiting the alternative complement pathway—thought to be the root cause of C3G. In the pivotal APPEAR-C3G Phase III trial, Fabhalta demonstrated:

• Significant and sustained reduction in proteinuria, a hallmark of kidney disease

• Early response visible within 14 days, maintained through 12 months

• A favorable safety profile with no new safety signals

• Common side effects including mild viral infections and nasopharyngitis

Notably, Fabhalta may increase the risk of serious infections caused by encapsulated bacteria, so treatment requires compliance with a Risk Evaluation and Mitigation Strategy (REMS) program, including pre-treatment vaccinations.

A New Standard of Care

“This approval is historic for the entire C3G community,” said Dr. Carla Nester, Professor of Pediatrics-Nephrology at the University of Iowa and study co-investigator. “Fabhalta offers a real chance to alter the disease course by targeting its underlying biology.”

Patient advocate Lindsey Fuller, co-leader of C3G Warriors, added: “To finally have a treatment that’s oral and targeted—it’s the hope my family and others have waited for.”

Novartis Expands Its Kidney Disease Leadership

This is Fabhalta’s third U.S. approval, following previous FDA authorizations for paroxysmal nocturnal hemoglobinuria (PNH) and immunoglobulin A nephropathy (IgAN). With several other investigational kidney therapies in development, including atrasentan and zigakibart, Novartis is positioning itself as a leader in tackling unmet needs in nephrology.

In parallel, Fabhalta has received a positive opinion from the European Medicines Agency (EMA) for C3G and is under regulatory review in China and Japan.