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.
Givosiran is made of a short, synthetic strand of genetic material called ‘small interfering RNA’ (siRNA) that has been designed to interfere with the production of an enzyme involved in an early step in making haem. By blocking this early step of haem production in patients with AHP, givosiran is expected to prevent the next steps which produce substances that accumulate in the body and cause the symptoms of the disease.