Ravulizumab for paroxysmal nocturnal haemoglobinuria

Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by the deficiency of glycosylphosphatidylinositol (GPI) anchored proteins, such as CD55 and CD59, on hematopoietic cells resulting in uncontrolled comple ment activation and haemolysis. Ravulizumab is a new monoclonal antibody with high affinity for complement factor C5 and hereby inhibits complement activation. Due to its significant longer half-life time compared to eculizumab, another C5 targeting complement inhibitor, ravulizumab, is administered intravenously once every eight weeks. Since November 2023, ravulizumab is reimbursed in Belgium for patients with haemolysis and showing clinical symptoms of high disease burden, as well as for clinically stable patients who are treated with eculizumab for at least six months.