Novartis Receives FDA Approval for Iptacopan as First Oral Monotherapy for Paroxysmal Nocturnal Hemoglobinuria

Novartis today announced that the FDA has approved Fabhalta (iptacopan) capsules as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, life-threatening blood disorder caused by a genetic mutation that leads to the destruction of red blood cells. In the pivotal APPLY-PNH trial, Fabhalta demonstrated superior efficacy compared to anti-C5 therapy, with 82.3% of patients achieving hemoglobin response. The oral twice-daily dosing of Fabhalta offers patients a more convenient treatment option compared to intravenous therapies. This approval represents a significant advancement for PNH patients who have been dependent on IV infusions.