Novartis announced positive topline results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta®? (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb 10g/dL following treatment with eculizumab or ravulizumab). Data will be presented at an upcoming medical meeting in 2025.

Fabhalta was recently granted accelerated approval by the US Food and Drug Administration (FDA) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases. Fabhalta, discovered at Novartis, received approval by the FDA in December 2023 for the treatment of adults with adults with PNH and by the European Medicines Agency (EMA) in May 2024 for the treatment of adults with hemolytic anemia. In August 2024, Fabhalta was granted accelerated approval by the U.S Food and Drug Administration (F FDA) for the reduction of proteinur in adults with primary immunoglOBulin A nephropathy ("IgAN") at risk of rapid disease progression and development is ongoing in multiple complementary-mediated diseases.