Novartis announced that Fabhalta (iptacopan), a first-in-class complement inhibitor, has received accelerated approval from the FDA for the treatment of proteinuria (too much protein in the urine) in adults with primary IgA nephropathy (IgAN) who are at risk of rapid disease progression. 

The Phase III APPLAUSE-IgAN study involved 518 adult patients with primary IgAN. These participants were randomized to receive either Fabhalta or a placebo, with the primary focus on evaluating the drug’s efficacy in reducing proteinuria and its safety profile over the study period.

Fabhalta demonstrated a:

• 44% reduction in proteinuria at nine months compared to baseline, versus the 
• 9% reduction seen in the placebo group. 

The FDA approval is based on this interim data (38% reduction v. placebo).

The reduction in proteinuria, a key marker of kidney damage, was consistent across all subgroups, including varying:

• Ages,  
• Sexes,  
• Races,  
• Baseline disease characteristics, and 
• Use of SGLT2 inhibitors (drugs that lower blood glucose levels in people with type 2 diabetes)

Full approval contingent on further study results that are expected in 2025.

Fabhalta’s Mechanism and Ongoing Studies

Fabhalta targets the alternative complement pathway (AP), a part of the immune system that plays a crucial role in the body’s defense against pathogens. In the context of IgAN, the AP becomes overactivated in the kidneys, leading to inflammation and subsequent damage to the glomeruli, the filtering units of the kidneys. Inflammation and proteinuria are both hallmark features of IgAN.

Despite current standard treatments, many IgAN patients still progress to kidney failure within 10 to 20 years. The ongoing Phase III study will continue to evaluate Fabhalta’s impact on slowing kidney function decline, with final data expected to support a traditional FDA approval. 

The drug is part of Novartis’s broader efforts to address rare kidney diseases, with additional investigational treatments in development.

*Novartis. (2024, August 8). Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN) [Press release]. https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-fabhalta-iptacopan-first-and-only-complement-inhibitor-reduction-proteinuria-primary-iga-nephropathy-igan 

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