FDA Approves Expanded Use Of Novartis Rare Disease Drug As Only Treatment For Rare Type Of Kidney Disease

On Thursday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s NVS oral Fabhalta (iptacopan) for adults with C3 glomerulopathy (C3G) to reduce proteinuria (protein in the urine), making it the first and only treatment approved for this condition.

C3G is a rare kidney disease characterized by damage to the glomeruli (kidney filters) due to dysregulation of the complement system, specifically the alternative pathway, leading to C3 protein accumulation.

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The pivotal Phase 3 APPEAR-C3G study evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G.

Treatment with Fabhalta resulted in a clinically meaningful reduction in proteinuria, which was seen as early as 14 days and sustained at 12 months.

Similarly, in the open-label period, proteinuria reduction was seen in participants who switched to Fabhalta.

Last month, Fabhalta received a positive CHMP Opinion in C3G by the European Medicines Agency (EMA). Regulatory reviews for this indication are ongoing in China and Japan.

This is the third US approval for Fabhalta and its second within the Novartis kidney disease portfolio since August 2024, when Fabhalta was granted accelerated approval by the FDA for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.

Continued approval for this indication is contingent upon confirmatory evidence.

Fabhalta received its first FDA approval in December 2023 for adults with paroxysmal nocturnal hemoglobinuria (PNH).

In the fourth quarter of 2024, Fabhalta generated sales of $57 million.

In addition to Fabhalta, Novartis is advancing the late-stage development of two additional IgAN therapies with highly differentiated mechanisms of action: atrasentan, an investigational oral endothelin A receptor antagonist that received FDA filing acceptance in Q2 2024 with a decision anticipated in H1 2025, and zigakibart, an investigational subcutaneously administered anti-APRIL monoclonal antibody currently in Phase 3 development.

Price Action: NVS stock is down 0.45% at $112.70 at the last check Friday.

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