Imetelstat is in clinical development for the treatment of relapsed/refractory low or intermediate-1 risk myelodysplastic syndrome (MDS) in transfusion-dependent patients, following erythropoiesis-stimulating agent (ESA) treatment. MDS are a group of disorders in which red blood cells, white blood cells, and platelets produced by the bone marrow do not grow and mature normally. MDS are long-term debilitating and life-threatening diseases. MDS patients may require repeated blood transfusions and currently have few treatment options.
Imetelstat sodium is an intravenously administered drug that works by blocking the activity of an enzyme called telomerase. Telomerase is involved in regulating cell growth and division. By blocking the activity of telomerase, this medicine is expected to stop the uncontrolled division of abnormal immature blood cells, therefore slowing the progression of MDS. If licensed, imetelstat will provide an additional treatment option for relapsed/refractory low or intermediate-1 risk MDS in transfusion-dependent patients, following ESA Treatment.
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.