Hemoglobinopathy is a genetic disorder that causes structural abnormalities in hemoglobin molecules, particularly defective globin chains. Hemoglobinopathies are inherited single-gene disorders of which sickle cell disease is the most common kind. There is a high prevalence of Hemoglobinopathies in Africa, Southeast Asia and the Mediterranean basin. If left untreated, critical cases of hemoglobinopathies lead to anemia, organ dysfunction or death. Gel electrophoresis and complete blood count are used as diagnostic tests to determine the presence of hemoglobinopathies. The disease does not have a permanent cure; this has resulted in a growing demand for new product innovations in the pharmaceutical sector. Additionally, the high prevalence of the disease and the national control program are driving the need for drug therapy in developing economies. Alnylam Pharmaceuticals, Gamilda Cell, Sangamo Biosciences, Cancer Therapeutics CRC Pty Ltd and Biogen Idec are some of the key players in this market. These companies have invested huge sums of money in research and development of hemoglobinopathies to develop a permanent cure for this disease. NiCord, ZFP and ALN-TMP are some of the current products available in the market. Companies have adopted the strategy of collaboration and licensing to supplement their research activities.
Global hemoglobinopathies market is segmented on the basis therapeutics and geography. The therapeutic segment includes various drugs such as NiCord, ZFP Transcription Factors, ALN-TMP, Drug Targeting PRMT5 and Drug Targeting Protein Arginine Methyltransferase 5. ALN-TMP is a subcutaneous RNAi therapeutic that targets Tmprss6 and is used in the treatment of β-thalassemia. NiCord®, based on the novel NAM technology, is a potential solution for a number of diseases such as thalassemia, sickle cell disease, hematological malignancies, severe rare genetic metabolic diseases and autoimmune diseases. Geographically, the global hemoglobinopathies market is segmented into North America, Europe, Asia-Pacific and LAMEA. Africa is the leading market for these drugs due to the high prevalence of hemoglobinopathies in the region.
KEY MARKET SEGMENTS
The global hemoglobinopathies market is segmented into two major categories: pipeline therapeutics and geography.
MARKET BY PIPELINE THERAPEUTICS
MARKET BY GEOGRAPHY