Already armed with an approval in paroxysmal nocturnal hemoglobinuria, Novartis' Factor B inhibitor, Fabhalta, has nabbed a priority review tag in IgA nephropathy (IgAN).
According to Novartis, the APPLAUSE-IgAN trial of Fabhalta is the first and only phase 3 study to demonstrate significant proteinuria (protein in urine) reduction by targeting the complement system in patients with IgAN, a rare kidney disease that can progress to kidney failure within 10 years.
Results from a pre-specified interim analysis demonstrated that patients treated with Fabhalta achieved a 38.3% proteinuria reduction at 9 months when compared to placebo on top of supportive care.
If approved in IgAN, Fabhalta would be the first treatment that specifically targets the alternative complement pathway.
Fabhalta was first approved by the FDA back in December, becoming the first oral monotherapy for the treatment of adults with a rare, chronic blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH).
The standard of care for PNH, C5 inhibitor treatments administered as infusions, may leave symptoms, including hemolysis, bone marrow failure and thrombosis, uncontrolled. While over one-third of patients on anti-C5 treatments require blood transfusions at least once per year, in Novartis' phase 3 APPLY-PNH trial, nearly all patients treated with Fabhalta did not receive blood transfusions.
Beyond PNH and IgAN, Fabhalta is currently in development for a range of rare diseases including C3G, atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN).
