Hemoglobinopathy includes a range of disorders, which occur due to altered structure of hemoglobin. Hemoglobin is present in the red blood cells that transports oxygen from the lung to the body. Hemoglobinopathy disorder are inherited and are passed on from one generation to another. It is further classified into two categories – 1. Thalassemia Syndrome 2. Structural Hemoglobin Variants. Thalassemia syndrome includes disorder related to synthesis defect. The synthesis defect of alpha globin gives rise to alpha thalassemia and defect in beta globin give rise to beta thalassemia. The structural hemoglobin variants occur due to alteration of alpha and beta chains that causes defect in structure.
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Global Hemoglobinopathy Treatment Market Drivers
Increasing research and development activities are expected to support global hemoglobinopathy treatment market growth over the forecast period. For instance, in September 2018, AstraZeneca initiated its clinical trial for Ticagrelor. It is being studied for the treatment of sickle cell disease. The drug is being studied for the evaluation of its safety and efficacy. Currently, it is in the third phase of clinical trial.
Furthermore, in January 2019, Celgene Corporation and Acceleron Pharma Inc. started their clinical trial for luspatercept. It is being studied for the treatment of adult patients with beta-thalassemia. The drug is being tested for its safety, efficacy, and pharmacokinetics. In June 2019, the U.S. Food & Drug Administration (FDA) accepted biologics license application (BLA) of luspatercept. Hence, such developments are expected to drive the global hemoglobinopathy treatment market growth.
Furthermore, key players are indulged in obtaining approvals from regulatory bodies for their products, which is expected to impel the global hemoglobinopathy treatment market growth. For instance, June 2019, Bluebird Bio Inc. received the European Medicines Agency (EMA) approval for its product, Zynteglo. It is a gene therapy, which contains autologous CD34+ cells encoding βA-T87Q-globin gene. It is indicated for patients aged 12 years and above with transfusion-dependent β-thalassemia who do not have the β0/β0 genotype. Hence, such approvals are expected to impact positively on the global hemoglobinopathy treatment market growth.
Global Hemoglobinopathy Treatment Market Key Players
Some of the major players operating in the global hemoglobinopathy treatment market include, Novartis AG, AstraZeneca, Bluebird Bio Inc., Bristol-Myers Squibb, Emmaus Medical, Inc., Acceleron Pharma, Inc., HemaQuest Pharmaceuticals Inc., Eli Lilly and Company, Celgene Corporation, and Acceleron Pharma, Inc.
Global Hemoglobinopathy Treatment Market Taxonomy
Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene)
By Disease Type
Sickle Cell Diseases
Global Hemoglobinopathy Treatment Market Regional Analysis
North America is expected to hold dominant position in global hemoglobinopathy treatment market. Factors such as increase in prevalence of hemoglobinopathy disorders are expected to drive the global hemoglobinopathy treatment market growth. According to the data published by National Centre for Biotechnology Information (NCBI), in 2019, around 300,000 infants are born with sickle cell anemia annually and the population of sickle cell anemia is 100,000 in the U.S. Hence, higher prevalence of sickle cell anemia may increase the demand for treatment, thereby expected to support the global hemoglobinopathy treatment market growth.
Global Hemoglobinopathy Treatment Market Restraints
Rejection of approvals related to hemoglobinopathy treatment by the regulatory bodies is expected to hamper the global hemoglobinopathy treatment market growth. For instance, in May 2019, European Medical Agency (EMA) rejected an approval of a drug, Xyndari. Xyandri was manufactured by Emmaus Medical, Inc. It was being developed for the treatment of patients with sickle cell disease. Hence, this factor may have a negative impact on the global Hemoglobinopathy Treatment Market growth.
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