Novartis shares new data about Fabhalta for IgAN treatment

Betsy Goodfellow | April 16, 2024 | News story | Research and Development Fabhalta, IgAN, Nephrology, Novartis, nephropathy 

Novartis has announced new results from a pre-specified interim analysis of its phase 3 APPLAUSE-IgAN trial of Fabhalta (iptacopan), an investigational Factor B inhibitor of the alternative complement pathway, in patients with IgA nephropathy (IgAN).

In this analysis, patients treated with Fabhalta reached a 38.3% proteinuria reduction (measured by a 24-hour urine protein to creatinine ratio [UPCR]) at nine months compared to placebo and supportive care.

This interim analysis included 250 patients for its efficacy analysis and 443 patients for its safety analysis, however the study is ongoing and remains double-blinded.

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The US Food and Drug Administration has accepted its submission for accelerated approval, and the drug has received priority review.

Professor Dana Rizk, investigator and APPLAUSE-IgAN steering committee member and professor in the UAB Division of Nephrology, commented: “In IgAN, part of the immune system called the alternative complement pathway can become overly activated in the kidneys, which causes an inflammatory response, leading to progressive kidney damage and gradual loss of kidney function. The loss of kidney function, together with potential side effects of IgAN treatments available until recently, significantly impact patients’ lives. Fabhalta is the first potential treatment for IgAN that specifically targets the alternative complement pathway.”

Betsy Goodfellow

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