Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t 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, a protein in red blood cells that carries oxygen throughout the body . Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
Luspatercept, administered via subcutaneous injection, 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-thalassaemia 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.
Deferiprone is in clinical development for patients with sickle-cell disorder (SCD) and other anaemias that are suffering from iron overload due to frequent transfusions to increase their red blood cell count. SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle red blood cells die early, and patients often require blood transfusions. Iron overload is an effect of frequent transfusions in SCD. Excess iron in the body can be toxic to major organs like the heart and liver.