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 March 2011, Novartis AG in collaboration with Incyte Corporation, started clinical trials for a drug, Ruxolitinib, which is currently in Phase I clinical development. Ruxolitinib acts as an inhibitor of Janus Kinase type 1 and type 2. It is administered orally. In June 2016 Incyte Corporation started clinical trials for a drug in Phase II clinical study, for assessing safety and efficacy of drug INCB050465, in combination with Ruxolitinib, in patients of myelofibrosis. INCB050465 is administered orally. In February 2013, Celgene Corporation started clinical trials for a drug, Sotatercept, which is currently in Phase II clinical development. Sotatercept binds to the sequesters ligands of the transforming growth factor beta, which leads to relieving of blockage of terminal erythroid differentiation. It is administered intravenously and is under the study to find out, as to whether it can control myeloproliferative neoplasm associated myelofibrosis and anemia. In September 2016, Celgene Corporation started clinical trials for the drug Durvalumab, a monoclonal antibody, which is being administered intravenously in patients with 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.
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