Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, potentially life-threatening hematologic disease characterized by various degrees of hemolysis, bone marrow failure, and thrombophilia. Pegcetacoplan (PEG), a C3 complement-inhibitor approved by the FDA for treatment of adults with PNH, has demonstrated improved hemoglobin (Hb) levels for PNH patients with screening Hb levels <10.5 g/dL and prior suboptimal response to eculizumab (ECU; C5-inhibitor) (Hillmen P, et al., N Engl J Med, 2021 384 (11):1028-1037) or complement-inhibitor naïve PNH patients (Wong RS, et al., Blood, 2020 136 [Supplement 1]). While these studies have demonstrated positive results for patients with lower baseline Hb levels, the efficacy and safety of PEG in PNH patients with baseline Hb ≥10.0 g/dL has not been evaluated.