Ravulizumab is a medicinal product that is being developed for the treatment of atypical haemolytic uraemic syndrome (aHUS) in adults and children. aHUS is an ultra-rare disease caused by a fault in part of the body’s immune response, called the complement system. This fault causes the complement system to attack the body’s own cells, especially those that line the blood vessels. This leads to clots forming within the small vessels and eventually serious medical problems such as kidney failure which, in most cases, progress to end stage kidney failure. aHUS occurs in childhood more frequently than in adulthood. It often results from a combination of environmental and genetic factors. Faults (mutations) in some genes increase the risk of developing aHUS.
Ravulizumab works by inhibiting a component in the complement system called C5. It is given intravenously and has the potential to increase patient’s quality of life and to decrease treatment burden due to its extended effect that enables every 8-week dosing. If licensed, ravulizumab will offer an additional first-line treatment option for adults and children with aHUS.
Sutimlimab is a first-in-class monoclonal antibody in development for the treatment of cold agglutinin disease, a rare form of autoimmune haemolytic anaemia, caused by cold-reacting autoantibodies. These antibodies bind to red blood cell membranes and destroy them, leading to anaemia. Symptoms include chronic debilitating fatigue, acrocyanosis, shortness of breath and other anaemia-related symptoms, leading to a poor quality of life and increased health resource utilization. In chronic cold agglutinin disease, the patient is more symptomatic during winter months. Therapeutic options may range from using warm clothing and avoiding exposure to cold weather to blood transfusions and chemotherapy.