Aplastic Anemia – Pipeline Review, 2019

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.

Report Description

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.

Report Scope

• Thorough assessment of the pipeline products by areas such as development stage; route of administration; drug class; indication; sponsor; molecule type and drug target
• Comprehensive profiles of the pipeline products with details such as company overview; product description; R&D status; development activities; mechanism of action; molecule type; development stage; indications; funding and route of administration
• Overview of dormant and discontinued pipeline products
• Key insights in relation to the epidemiology of conditions being treated by the pipeline products and overview of the addressable or current market for the pipeline products
• Overview of the latest developments; news articles, press releases and relevant conferences

Report Methodology

• All pipeline reports are built through the analysis of data primarily collected through credible desk research sources. Secondary research is supplemented by interviews conducted with key opinion leaders.
• Desk research sources includes global and regional clinical trials databases; annual reports, websites, press releases & investor presentations of companies; white papers; news articles; reports published by industry associations; articles/reports published on databases such as NCBI, ResearchGate; internal databases

Reasons to Buy this Report

• Develop effective growth strategies based on comprehensive overview of the R&D activity and pipeline products for Aplastic Anemia
• Identify emerging players or competition in the market based on pipeline products and develop strategies to counter the emergence of these players
• Identify the focus of leading players in relation to R&D for Aplastic Anemia
• Identify potential companies from a partnership or acquisition point of view based on current synergy in R&D activities or strategies to diversify R&D focus to drive growth in business
• Analyse the reasons behind dormant and discontinued products to make changes in the R&D focus if necessary