Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare intravascular hematologic disorder characterized by uncontrolled terminal complement activation, thrombosis and organ damage. The terminal complement C5 inhibitor, ravulizumab, was approved by the FDA in 2018 for the intravenous treatment of PNH. A subcutaneous (SC) ravulizumab formulation (ravulizumab SC; 480 mg) is currently in clinical development which aims to provide a once weekly, self-administered treatment dose, compared with recently approved pegcetacoplan (1,080 mg); a proximal complement C3 inhibitor, self-administered SC twice weekly–every 3 days.
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