Ravulizumab for paroxysmal nocturnal haemoglobinuria in paediatrics

Ravulizumab is in clinical development for the treatment of paediatric patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare blood condition in which red blood cells are attacked by the body’s immune system. PNH is a chronic condition that is associated with complications that can be severely debilitating and life-threatening including abdominal pain, kidney problems, fatigue, shortness of breath, bleeding and blood clots, dysphagia, organ damage and premature mortality.
Ravulizumab, administered intravenously, works by attaching to the complement component 5 (C5) protein, which is part of the complement system (a component of the immune system). By attaching to the C5 protein, ravulizumab blocks its effect and thereby reduces the destruction of red blood cells. If licensed, ravulizumab will provide a treatment option for paediatric patients with PNH.