FDA Approves Novartis' Fabhalta for IgAN Treatment

Novartis has recently gained accelerated approval from the US Food and Drug Administration (FDA) for Fabhalta (iptacopan), a medication aimed at reducing proteinuria in adults suffering from primary IgA nephropathy (IgAN), a rare and progressive kidney disease. Fabhalta works as an alternative complement pathway inhibitor and its approval is grounded on interim results from the Phase III APPLAUSE-IgAN study.

The APPLAUSE-IgAN study is a comprehensive, multicenter, randomized, double-blind, placebo-controlled trial that includes 518 adult patients diagnosed with IgAN. This study primarily evaluates the safety and efficacy of Fabhalta, focusing on reducing proteinuria and monitoring the decline in estimated glomerular filtration rate (eGFR) over a 24-month period.

Key secondary objectives of the trial encompass the reduction in the urine protein-to-creatinine ratio, the occurrence of composite kidney failure events, and changes in patients' levels of fatigue. The interim analysis has shown a substantial 44% reduction in proteinuria at the nine-month mark compared to the placebo, signaling a significant improvement in kidney damage markers. For continuous approval, additional evidence of clinical benefit is necessary, with the final results of the study anticipated in 2025.

The trial's interim findings specifically highlight a significant 38% reduction in proteinuria compared to the placebo group. Participants in the study were on stable doses of renin-angiotensin system inhibitors, either with or without the addition of sodium-glucose cotransporter-2 inhibitors. The safety profile of Fabhalta in the study was in line with previous research findings.

Novartis is actively engaged in developing two additional therapies for IgAN, namely atrasentan and zigakibart, which are currently in late-stage trials. Novartis US President Victor Bultó emphasized the importance of this approval, calling Fabhalta a first-in-class medication for IgA nephropathy. He highlighted the company's dedication to improving care for those with rare renal diseases and expressed optimism about ongoing collaborations within this community as they advance their extensive treatment portfolio.

Atrasentan has already received FDA filing acceptance, signaling progress towards potentially offering another therapeutic option for IgAN patients. Meanwhile, zigakibart is undergoing Phase III development, further underlining Novartis' commitment to addressing the urgent need for new treatments in the realm of rare kidney diseases.