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The hematological disorders therapy area, which covers indications such as sickle cell disease, paroxysmal nocturnal hemoglobinuria, anemia, thalassemia and hereditary hemochromatosis, is experiencing a significant level of first-in-class innovation, with 92 of its 327 pipeline programs with a disclosed molecular target identified as first-in-class, according to business intelligence provider GBI Research.
The company’s latest report states that the hematological disorders market is small with substantial unmet need and as such represents a significant investment opportunity. Due to few treatment options, many patients have a poor quality of life and poor prognosis, especially those with more severe disease states. The lack of pharmacotherapy treatment options means that alternative treatment approaches such as chronic blood transfusions and bone marrow transplants are used.
Investment in first-in-class development is particularly important within hematological disorders, it is reported, as it holds the potential of achieving clinical breakthroughs through radical pharmaceutical innovation. As such, it represents the most likely method of discovering new and effective drugs for a therapy area in which there has been a lack of funding and few recent breakthrough treatment discoveries. GBI Research states that a successful first-in-class product has the potential to gain a large market share due to the significant level of unmet need with regard to effective marketed treatment options.