Luspatercept is an erythroid (red blood cell) maturation agent administered by subcutaneous injection and is in clinical development for the treatment of adult patients who have serious blood disorders such as anaemia associated with myelodysplastic syndromes (MDS). MDS are a group of disorders in which red blood cells, white blood cells, and platelets produced by the bone marrow (the spongy tissue inside the large bones) do not grow and mature normally. MDS are long-term debilitating and life-threatening diseases. They can lead to severe anaemia, infections or bleeding, and can result in leukaemia (cancer of the white blood cells). MDS patients currently have few approved therapeutic options and may require repeated blood transfusions.
Luspatercept is a recombinant engineered protein designed to enhance the maturation of red blood cells produced in the bone marrow. This leads to the production of healthy red blood cells and increased haemoglobin levels, improving symptoms in patients with anaemia. Luspatercept is a novel approach for treating anaemia associated with MDS, with potential to improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.