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.
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.