Understanding Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disease that is associated with abnormally low (below normal) hemoglobin levels. PNH can appear at any age and in any race or gender, and is most often diagnosed in people in their early 30s.1,2
Low hemoglobin levels indicate ongoing hemolysis and can result in frequent transfusions and debilitating symptoms like severe fatigue and chest pain for individuals with PNH.
In PNH, blood cells lack complement regulatory proteins, so the body recognizes these healthy red blood cells as damaged.1 This leads to uncontrolled activation of the complement cascade, initiated at C3, and results in the destruction of oxygen-carrying red blood cells through a process called hemolysis.3 The complement cascade is a part of the immune system that is responsible for recognizing and eliminating pathogens and damaged cells in the blood.
Published data suggest that even with eculizumab treatment, approximately two-thirds of people with PNH have low hemoglobin levels, and 20% require three or more transfusions a year.4,5,6
Pegcetacoplan (APL-2) in PNH
Pegcetacoplan is a targeted C3-inhibitor designed to regulate uncontrolled or excessive complement activation.
With this approach, we believe that pegcetacoplan may have the potential to provide broad control of the red blood cell destruction (hemolysis) that drives the symptoms of PNH.
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