Novartis reports significant proteinuria reduction by Fabhalta in Phase 3 IgAN trial

Novartis has shared encouraging results from the Phase III APPLAUSE-IgAN study, revealing that Fabhalta (iptacopan) led to a significant 38.3% reduction in proteinuria, as measured by the 24-hour urine protein to creatinine ratio (UPCR), at 9 months compared to placebo. These findings, which signal potential in slowing kidney disease progression in IgA nephropathy (IgAN) patients, were presented at the late-breaking clinical trials session of the World Congress of Nephrology in Buenos Aires, Argentina.

The APPLAUSE-IgAN study is a multicenter, randomized, double-blind, placebo-controlled trial designed to assess the efficacy and safety of Fabhalta, an oral Factor B inhibitor targeting the alternative complement pathway, critical in the inflammatory process affecting kidney function in IgAN. The study involved 250 patients in the efficacy analysis and 443 in the safety analysis, with the drug demonstrating a favorable safety profile.

Professor Dana Rizk, a member of the APPLAUSE-IgAN Steering Committee, highlighted the drug’s innovative approach, “Fabhalta is the first potential treatment for IgAN that specifically targets the alternative complement pathway, aiming to mitigate kidney damage and improve patient outcomes significantly.”

Looking forward, the study is set to continue in a double-blind manner, with further results anticipated by 2025, including primary endpoints like the annualized total estimated glomerular filtration rate (eGFR) slope over 24 months. Novartis has already received priority review from the FDA for possible accelerated approval based on these interim results.

David Soergel, M.D., Global Head of Cardiovascular, Renal, and Metabolism Development Unit at Novartis, discussed the broader implications for their renal pipeline, stating, “Our renal pipeline includes medicines with a variety of mechanisms which may allow them to be targeted to patients based on their clinical characteristics.”

The results of the APPLAUSE-IgAN study could mark a significant advancement in the treatment landscape of IgAN, providing a targeted therapy option where limited treatments were available. The continued investigation into Fabhalta and its application in related renal conditions underscores Novartis’s commitment to addressing complex kidney diseases.

In conclusion, the ongoing research and data presented by Novartis at the World Congress of Nephrology offer a promising glimpse into the future of IgAN treatment, potentially setting a new standard in the care and management of this challenging renal condition. The nephrology community and patients alike eagerly await the final analysis, hoping for positive outcomes that could transform IgAN management.