Myelofibrosis is a disease characterized by the build-up of scar tissue in the bone marrow, which leads to genetic mutation in a person’s body. The myelofibrosis therapeutics pipeline is anticipated to increase with the growing need to develop a cure for the problem, and is noticed in approximately one in 500,000 people, in the U.S., usually in the age group of 50 to 80 years, as per the as data provided by U.S. national Library of Medicine. It is a rare form of disease and also happens to patients with thrombocythemia and polycythemia. Moreover, the future developments in therapies are expected to shoot-up.
According to National Center for Advancing Translational Sciences, myelofibrosis is disorder of bone marrow, in which bone marrow is replaced by fibrous tissues. In myelofibrosis, bone marrow cannot produce sufficient blood cells and platelets, which leads to weakness, anemia, fatigue and sometimes swelling of spleen and liver. Reduction in number of platelets can cause bleeding and bruising. This condition is also called extramedullary haematopoiesis. The genes of the stem cells such as, JAK2, MPL, CALR, and TET2 genes undergo mutation in somatic stem cells but, the condition is uninherited. Treatments for the problem include chemotherapy, radiation therapy, blood transfusion and surgery.
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In November 2013, Baxter International Inc. entered into a licensing agreement with Cell Therapeutics, Inc., to develop and commercialize – pacritinib. Pacritinib is a novel investigational JAK2/FLT3 inhibitor with activity against genetic mutations linked to myelofibrosis, leukemia and certain solid tumors. Pacritinib is currently in Phase II clinical development in patients having chronic malignant bone marrow disorder, myelofibrosis.
Some of the companies having a pipeline of myelofibrosis therapeutics include Incyte Corporation, Novartis AG, Pfizer, Inc., Constellation Pharmaceuticals, Inc., GlaxoSmithKline Plc, Geron Corporation, Baxter International Inc.