Myelofibrosis is a cancer of the bone marrow in which the marrow is replaced by scar tissue. The early stages of myelofibrosis may be asymptomatic in some people while others may have severe symptoms from the onset. As the bone marrow becomes more scarred, it is less able to produce blood cells leading to enlargement of spleen and liver. Enlargement of spleen may cause abdominal pain, shortness of breath, early satiety and faecal incontinence, along with progressive anaemia. Other symptoms include incurable itch, general malaise, weight loss, night sweats, low grade fever, anaemia, fatigue, and pallor. Many people with myelofibrosis have mutations in a gene known as JAK2 gene.
Fedratinib is in development for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis, including patients who have been previously exposed with ruxolitinib. Fedratinib is an inhibitor of JAK2 with potential to kill cancer cells. Fedratinib is given by mouth as capsule. If licensed fedratinib will offer an additional option for the treatment for patients with myelofibrosis.
Ixazomib citrate is a novel oral medicinal product that is already licensed in the UK for the treatment of MM in patients who have received at least one prior therapy (in combination with lenalidomide and dexamethasone). Ixazomib citrate offers the potential advantage over similar medicines in its class of being more effective in its anticancer activity, less toxic (reduced side effects) and more convenient to administer (through its weekly oral dosing). If approved as maintenance therapy following stem cell transplant in newly diagnosed MM patients, ixazomib citrate has the potential to improve the success rates of treatment by improving progression free survival and overall survival as well as presenting a more convenient way of administration that allows long term administration and improvement of patients’ quality of life.