Deferiprone for transfusional iron overload in sickle cell disease and other anaemias – First Line

Plinabulin in combination with G-CSF is being developed for the prevention of chemotherapy-induced neutropenia (CIN) in patients with solid tumours receiving myelosuppressive chemotherapy with docetaxel, doxorubicin, and cyclophosphamide (TAC). Neutropenia is a condition associated with low levels of neutrophils, a type of white blood cell, which is a common side effect of cancer treatment. Severe neutropenia can lead to life-threatening complications due to resulting infections, and cause reduction or delay of chemotherapy treatment, potentially compromising the benefit to cancer patients.

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.

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.

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.