Clinical Trials

Novartis announces phase 3 results for Fabhalta for IgAN treatment

Novartis has announced new results from a pre-specified interim analysis of its phase 3 APPLAUSE-IgAN trial of Fabhalta (iptacopan),aninvestigationalFactorBinhibitorofthealternative 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.
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.”
David Soergel MD, global head, cardiovascular, renal and metabolism development unit at Novartis, added: “IgAN progresses over many years, and patients’ needs may evolve such that different therapies may be best used at different times. Our renal pipeline includes medicines with a variety of mechanisms, which may allow themtobetargetedtopatientsbasedontheirclinicalcharacteristics.”