Myelofibrosis is a rare disorder characterized by the formation of excessive scar tissue in the bone marrow, that impairs its ability to produce red blood cells. However, it is believed by the researchers that myelofibrosis occurs due to the presence of abnormal red blood stem cells in the bone marrow, that produce mature cells which grow quickly and form scar tissue leading to chronic inflammation. JAK2, MPL, CALR and TET2 genes play a key role in the formation of proteins that regulate growth, and division as well as proliferation of blood cells. The mutations in JAK2, MPL, CALR, and TET2 genes are most often associated with primary myelofibrosis. Afterwards, there is an over-production of megakaryocytes that stimulates another type of a cell that releases collagen, excess of collagen can lead to the formation of a scar tissue in the bone marrow. Some of the symptoms observed in patients with myelofibrosis are fever, bone pain, easy bleeding, excessive sweating, shortness of breath, and tiredness. The complications associated with myelofibrosis are painful bones and joints, acute leukemia, and portal hypertension.
Significant growth has been observed in the therapeutics pipeline of myelofibrosis, attributable to the increasing collaborations between companies, educational institutes and health organizations. It has been observed that the companies with these collaborations are focusing on development of combination therapies that are believed to show positive clinical trial results. The myelofibrosis therapeutics pipeline comprises of approximately 29 drug candidates in different stages of development.
Some of the key players developing drugs for the treatment of myelofibrosis include CTI BioPharma Corporation, Geron Corporation, Gilead Sciences, Inc and others.
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