Novartis has shared positive results from a late-stage trial of its oral factor B inhibitor Fabhalta (iptacopan) in patients with the rare kidney disease IgA nephropathy (IgAN).
Approximately 25 people per million worldwide are diagnosed each year with IgAN, which occurs when IgA accumulates in the kidneys and damages them. This impairs their filtering function and, as a result, the kidneys start to let substances such as blood and protein leak into the urine.
IgAN is a major cause of chronic kidney disease and kidney failure, with up to 30% of IgAN patients with persistently higher levels of proteinuria (protein in the urine) progressing to kidney failure within ten years.
The ongoing APPLAUSE-IgAN study has been evaluating the efficacy and safety of twice-daily Fabhalta in adult primary IgAN patients.
According to results from a pre-specified interim analysis of the trial presented at the World Congress of Nephrology, patients treated with Fabhalta achieved a 38.3% proteinuria reduction at nine months compared to placebo plus supportive care.
The study also showed that Fabhalta was well tolerated, with a favourable safety profile that was consistent with previously reported data.
Professor Dana Rizk, investigator and APPLAUSE-IgAN steering committee member and professor in the UAB Division of Nephrology, said: “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.”
Beyond IgAN, Fabhalta is currently in development for a range of rare diseases, including immunoglobulin A nephropathy, C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis and atypical haemolytic uraemic syndrome.
The therapy has already been approved by the US Food and Drug Administration and recommended by the European Medicines Agency’s human medicines committee to treat adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
Victor Bultó, president US, Novartis, described the US authorisation as an “extraordinary moment” for the PNH community, while Patrick Horber, president, international, Novartis, said at the time of the European recommendation that the therapy “could provide a much-needed alternative to support many people living with PNH who often have to structure their lives around managing their condition”.