Rusfertide for treating polycythaemia vera

Rusfertide is in clinical development for treating polycythaemia vera (PV). PV is a rare blood cancer that affects the bone marrow. It causes the human body to make too many red blood cells and numbers of white blood cells and platelets can also increase with this condition. It is one of a group of conditions called myeloproliferative neoplasms (MPNs). MPNs are conditions that cause an increase in the number of blood cells. Over 95% of people with PV have a change in the JAK2 gene. This usually happens during life, but in rare cases it can run in families and increase the risk of MPNs. People with PV can experience various symptoms including headaches, confusion, dizziness, blurred vision, night sweats, itching, extreme tiredness, unusual bleeding, red-looking eyes and skin, a swollen spleen and gout. The main goal of treatment in PV is to lower the risk of blood clots by keeping haematocrit (the percentage of blood volume that is made up of red blood cells) below 45%. However, many existing treatments fail to consistently achieve this target, leaving a significant number of patients at continued risk of serious complications.