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.
Ropeginterferon alfa-2b for injection is under development for the treatment of polycythaemia vera (PV), a rare blood disease in which the body makes too many red blood cells. The extra red blood cells make the blood thicker than normal and as a result, blood clots can form more easily. These clots may block blood flow through arteries and veins, which can cause a heart attack or stroke. Thicker blood also does not flow as quickly and may prevent organs from getting enough oxygen. A mutation, or change, in a gene called JAK2 is the major cause of PV. This gene makes a protein that helps the body produce blood cells. PV develops slowly and may not cause symptoms for years. PV has no cure, but treatments can help control the disease and its complications.