Luspatercept is a subcutaneous injection medicine under clinical development for the treatment of adult patients with beta-thalassemia who regularly require blood transfusion. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin. Beta-thalassemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin which can make them very anaemic (tired, short of breath and pale). Beta-thalassaemia major is the most severe type of beta-thalassaemia with patients requiring regular blood transfusions for survival. Patients with beta-thalassemia intermedia also need transfusions at different times of their lives. Current treatment options for beta-thalassemia are limited to blood transfusions with its associated risks and complications.
Luspatercept is a recombinant engineered protein designed to attach to certain proteins that slow down the maturation of red blood cells. This leads to the production of healthy red blood cells and increased haemoglobin levels, leading to improved symptoms in patients with beta-thalassemia intermedia and major. Luspatercept is a novel approach for treating anaemia, with potential to improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.
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.