ravulizumab

Ravulizumab, sold under the brand name Ultomiris, is a humanized monoclonal antibody complement inhibitor medication designed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). It is designed to bind to and prevent the activation of complement component 5 (C5).

Paroxysmal nocturnal hemoglobinuria is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells). In paroxysmal nocturnal hemoglobinuria, proteins known as the "complement system", which is part of the immune system, become overactive because of a genetic mutation and start to attack the patients' own red blood cells. Ravulizumab is a monoclonal antibody (a type of protein) designed to attach to the C5 protein, which is part of the complement system. By attaching to the C5 protein, the medicine blocks its effect and thereby reduces the destruction of red blood cells.