December 2020
Deferiprone for transfusional iron overload in sickle cell disease and other anaemias – First Line
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.
November 2020
Imetelstat for Myelodysplastic syndrome
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.
November 2020
Mitapivat for treating pyruvate kinase deficiency
Mitapivat is currently in clinical development for the treatment of adult patients with pyruvate kinase deficiency (PKD) who have regular blood transfusions and those who do not have regular blood transfusions. PKD is a genetic blood disorder caused by low levels of the enzyme pyruvate kinase (PK). Low levels of PK result in a deficiency in energy and causes red blood cells to break down too early. This is known as haemolytic anaemia. Symptoms of PKD vary significantly with some patients requiring no treatment and some patients requiring blood transfusions, surgery to remove the spleen or haematopoietic stem cell transplant which are all associated with risks and complications. There are currently no disease modifying treatments approved for the treatment of PKD.
November 2020
Maribavir for cytomegalovirus infections after transplant
Maribavir, administered orally, is thought to block the action of an enzyme of the virus called UL97 kinase. By blocking the enzyme, the medicine is expected to prevent viruses from reaching maturity, so that no new infectious viruses can be produced. If licensed, maribavir would offer an alternative treatment option for patients with CMV infections that are clinically refractory and/or genetically resistant to GCV, VGCV, CDV or FOS after stem cell or solid organ transplantation.
