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.
Ropeginterferon alfa-2b belongs to the group ‘interferons’. Interferons are natural substances produced by the body to help it fight against attacks. The exact way alpha interferons work is not fully understood, but are thought to modify how the immune system works by targeting blood cells produced by the JAK2 gene. Ropeginterferon alfa-2b is expected to work in PV by blocking the production of blood cells in the bone marrow. If licensed, ropeginterferon alfa-2b may offer an additional treatment option for patients with PV who currently have few effective therapies available.
Valoctocogene roxaparvovec is a gene therapy; it can modify the genes (functional units of heredity) of individuals with haemophilia A so that they can produce the clotting protein needed to allow the blood to clot. If licensed, valoctocogene roxaparvovec would be the first gene therapy for severe haemophilia A. Valoctocogene roxaparvovec administered as a single treatment would be sufficient to maintain normal levels of factor VIII in adult males with severe haemophilia A, and might reduce the need for regular factor VIII prophylaxis (preventative treatment).