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Aplastic Anemia is caused due to the destruction of pluripotent stem cells in bone marrow. The prominent symptoms of aplastic anemia are persistent tiredness, dizziness, paleness, frequent or repeated infections, and slow healing, due to a lack of normal white cells and red blood cells in the human body. According to the National Organization for Rare Disorders, Inc., the incidence of aplastic anemia in Israel and Europe is an estimated 2 per million people per year.
Current treatments for aplastic anemia include blood transfusions, stem cell transplant, immunosuppressant drugs, bone marrow stimulants, antibiotics, and others. Cyclosporines (Gengraf, Neoral, Sandimmune) and anti-thymocyte globulins are some of the immunosuppressant drugs used in combination for the treatment of the aplastic anemia. These drugs help in suppressing the activity of immune cells damaging bone marrow subsequently helping the recovery of bone marrow to generate new blood cells.
Various research institutes and different pharmaceutical industries have been focusing on studying and developing new treatment options for aplastic anemia. Currently, AMG531, which is being studied by Kyowa Hakko Kirin Korea Co., Ltd., is currently under phase 2 trials for as to evaluate the efficacy and safety of the drug and to determine the recommended initial dose of the drugs for the patients suffering from aplastic anemia.
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At present, around 60% of the pipeline candidates for aplastic anemia are in the phase-2 stage. More than half of the studies are sponsored by research institutions.
The report on ‘Aplastic Anemia– Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Aplastic Anemia. The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Aplastic Anemia.
The report on ‘Aplastic Anemia– Pipeline Review, 2019’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.