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Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need - Product Image

Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need

  • ID: 2393098
  • January 2013
  • 81 Pages
  • GBI Research

GBI Research, the leading business intelligence provider, has released its latest research, “Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need”. The report provides insights into the up-and-coming trends of a portion of the inherited orphan blood disorder pharmaceutical market by examining sickle cell anemia, thalassemia and Hereditary Angioedema (HAE) in seven major markets: the US, the top five European countries (France, Germany, Italy, Spain and the UK) and Japan. The report includes market forecasts to 2019, detailed pipeline analysis, in-depth profiling of major products and details of recent industry deals.

The report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research’s team of industry experts.

The sickle cell anemia market will be the fastest growing of the three markets during the forecast period with a Compound Annual Growth Rate (CAGR) of 9% seeing it reach $70m in 2019. The thalassemia market will grow at a lower CAGR of 7% to reach $59m in 2019. HAE represents the largest READ MORE >

1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Inherited Orphan Blood Diseases - Introduction
2.1 Overview
3 Inherited Orphan Blood Disorders - Therapeutic Landscape
3.1 Sickle Cell Anemia
3.1.1 Disease Overview
3.1.2 Revenues
3.1.3 Revenue Analysis by Country
3.1.4 Annual Cost of Therapy
3.1.5 Treatment Usage Patterns
3.1.6 Marketed Products
3.1.7 Market Drivers
3.1.8 Market Restraints
3.2 Sickle Cell Anemia: Research and Development Pipeline
3.2.1 Overview
3.2.2 Pipeline by Clinical Phase of Development
3.2.3 Trends in Sickle Cell Anemia Pipeline
3.2.4 Promising Pipeline Molecules
3.2.5 Key Takeaway
3.3 Thalassemia
3.3.1 Disease Overview
3.3.2 Revenues
3.3.3 Revenue Analysis by Country
3.3.4 Annual Cost of Therapy
3.3.5 Treatment Usage Patterns
3.3.6 Marketed Products
3.3.7 Market Drivers
3.3.8 Market Restraints
3.4 Thalassemia - Research and Development Pipeline
3.4.1 Overview
3.4.2 Pipeline by Clinical Phase of Development
3.4.3 Trends in Thalassemia Pipeline
3.4.4 Promising Pipeline Molecules
3.4.5 Key Takeaway
3.5 Hereditary Angioedema
3.5.1 Disease Overview
3.5.2 Revenues
3.5.3 Revenue Analysis by Country
3.5.4 Annual Cost of Therapy
3.5.5 Treatment Usage Patterns
3.5.6 Marketed Products
3.5.7 Market Drivers
3.5.8 Market Restraints
3.6 Hereditary Angioedema: Research and Development Pipeline
3.6.1 Overview
3.6.2 Pipeline by Clinical Phase of Development
3.6.3 Trends in Hereditary Angioedema Pipeline
3.6.4 Promising Pipeline Molecules
3.6.5 Key Takeaway
4 Inherited Orphan Blood Disorders Therapeutics - Strategic Consolidations
4.1 Segmentation by Year
4.2 Segmentation by Indication
4.3 Segmentation by Geography
4.4 Segmentation by Deal Value
4.5 Segmentation by Deal Type
4.6 Major Mergers and Acquisitions
4.7 Major Licensing Agreements
5 Inherited Orphan Blood Disorders Therapeutics - Appendix
5.1 Market Definitions
5.2 Abbreviations
5.3 Sources
5.4 Research Methodology
5.4.1 Coverage
5.4.2 Secondary Research
5.4.3 Primary Research
5.4.4 Therapeutic Landscape
5.4.5 Geographical Landscape
5.4.6 Pipeline Analysis
5.4.7 Competitive Landscape
5.4.8 Expert Panel Validation
5.5 Contact Us
5.6 Disclaimer

1.1 List of Tables
Table 1: Probability of HbSS Genotype by Region
Table 2: Sickle Cell Anemia, Total Market, CAGR of Average Annual Cost of Therapy
Table 3: Sickle Cell Anemia, Pipeline, Discovery
Table 4: Sickle Cell Anemia, Pipeline, Pre-Clinical
Table 5: Sickle Cell Anemia, Pipeline, Phase I
Table 6: Sickle Cell Anemia, Pipeline, Phase II
Table 7: Sickle Cell Anemia, Pipeline, Phase III
Table 8: Prevalence of a- and ß-Thalassemia (Hemoglobin H and ß-Thalassemia Intermedia and Major only)
Table 9: Thalassemia, Total Market, CAGR of Average Annual Cost of Therapy
Table 10: Thalassemia, Pipeline, Discovery
Table 11: Thalassemia, Pipeline, Pre-Clinical
Table 12: Thalassemia, Pipeline, Phase I
Table 13: Thalassemia, Pipeline, Phase II
Table 14: Thalassemia, Pipeline, Phase III
Table 15: Global Distribution of Hereditary Angioedema
Table 16: Hereditary Angioedema, Total Market, CAGR of Average Annual Cost of Therapy
Table 17: Hereditary Angioedema, Pipeline, Discovery
Table 18: Hereditary Angioedema, Pipeline, Pre-Clinical
Table 19: Hereditary Angioedema, Pipeline, Phase I
Table 20: Hereditary Angioedema, Pipeline, Phase II
Table 21: Hereditary Angioedema, Pipeline, Phase III

1.2 List of Figures
Figure 1: Comparison of Normal Human Hemoglobin and Hemoglobin S
Figure 2: Comparison of Shape and Flow of Normal Erythrocytes and Sickle Cells
Figure 3: Global Distribution of Sickle Cell Gene (HbS) and Malaria
Figure 4: Sickle Cell Anemia, Total Market, Revenue Forecast ($m), 2011–2019
Figure 5: Sickle Cell Anemia, US, Revenue Forecast ($m), 2011–2019
Figure 6: Sickle Cell Anemia, UK, Revenue Forecast ($m), 2011–2019
Figure 7: Sickle Cell Anemia, France, Revenue Forecast ($m), 2011–2019
Figure 8: Sickle Cell Anemia, Germany, Revenue Forecast ($m), 2011–2019
Figure 9: Sickle Cell Anemia, Italy, Revenue Forecast ($m), 2011–2019
Figure 10: Sickle Cell Anemia, Spain, Revenue Forecast ($m), 2011–2019
Figure 11: Sickle Cell Anemia, Total Market, Average Annual Cost of Therapy, 2011–2019
Figure 12: Sickle Cell Anemia, Total Market, Treatment Usage Pattern (‘000), 2011–2019
Figure 13: Hydroxyurea Factsheet
Figure 14: Sickle Cell Anemia, Pipeline, Overview
Figure 15: Sickle Cell Anemia, Pipeline, Pre-Clinical Molecule Comparison
Figure 16: Sickle Cell Anemia, Pipeline, Phase I Molecule Comparison
Figure 17: Sickle Cell Anemia, Pipeline, Phase II Molecule Comparison
Figure 18: Sickle Cell Anemia, Pipeline, Phase III Molecule Comparison
Figure 19: Sickle Cell Anemia, Pipeline by Therapeutic Class
Figure 20: Sickle Cell Anemia, Pipeline by Molecule Type
Figure 21: Sickle Cell Anemia, Pipeline by Mechanism of Action
Figure 22: Hemoglobin Tetramer with a- and ß-Globulin Chains
Figure 23: Global Distribution of a- and ß-Thalassemia
Figure 24: Thalassemia, Total Market, Revenue Forecast ($m), 2011–2019
Figure 25: Thalassemia, US, Revenue Forecast ($m), 2011–2019
Figure 26: Thalassemia, UK, Revenue Forecast ($m), 2011–2019
Figure 27: Thalassemia, France, Revenue Forecast ($m), 2011–2019
Figure 28: Thalassemia, Germany, Revenue Forecast ($m), 2011–2019
Figure 29: Thalassemia, Italy, Revenue Forecast ($m), 2011–2019
Figure 30: Thalassemia, Spain, Revenue Forecast ($m), 2011–2019
Figure 31: Thalassemia, Japan, Revenue Forecast ($m), 2011–2019
Figure 32: Thalassemia, Total Market, Average Annual Cost of Therapy, 2011–2019
Figure 33: Thalassemia, Total Market, Treatment Usage Pattern
Figure 34: Thalassemia, Pipeline, Overview
Figure 35: Thalassemia: Pipeline, Phase I Molecule Comparison
Figure 36: Thalassemia, Pipeline, Phase II Molecule Comparison
Figure 37: Thalassemia, Pipeline by Therapeutic Class
Figure 38: Thalassemia, Pipeline by Molecule Type
Figure 39: Thalassemia, Pipeline by Mechanism of Action
Figure 40: Hereditary Angioedema, Total Market, Revenue Forecast($m), 2011–2019
Figure 41: Hereditary Angioedema, US, Revenue Forecast ($m), 2011–2019
Figure 42: Hereditary Angioedema, UK, Revenue Forecast ($m), 2011–2019
Figure 43: Hereditary Angioedema, France, Revenue Forecast ($m), 2011–2019
Figure 44: Hereditary Angioedema, Germany, Revenue Forecast ($m), 2011–2019
Figure 45: Hereditary Angioedema, Italy, Revenue Forecast ($m), 2011–2019
Figure 46: Hereditary Angioedema, Spain, Revenue Forecast ($m), 2011–2019
Figure 47: Hereditary Angioedema, Japan, Revenue Forecast ($m), 2011–2019
Figure 48: Hereditary Angioedema, Total Market, Average Annual Cost of Therapy, 2011–2019
Figure 49: Hereditary Angioedema, Total Market, Treatment Usage Pattern
Figure 50: Hereditary Angioedema, Pipeline, Overview
Figure 51: Hereditary Angioedema, Pipeline, Pre-Clinical Molecule Comparison
Figure 52: Hereditary Angioedema Pipeline, Phase II Molecule Comparison
Figure 53: Hereditary Angioedema, Pipeline, Phase III Molecule Comparison
Figure 54: Hereditary Angioedema, Pipeline by Therapeutic Class
Figure 55: Hereditary Angioedema, Pipeline by Molecule Type
Figure 56: Hereditary Angioedema, Pipeline by Mechanism of Action
Figure 57: Inherited Orphan Blood Diseases Market, Deal Segmentation by Year, 2000–2012
Figure 58: Inherited Orphan Blood Diseases Market, Deal Segmentation by Indication
Figure 59: Inherited Orphan Blood Diseases Market, Deal Segmentation by Geography
Figure 60: Inherited Orphan Blood Diseases Market, Deal Segmentation by Value
Figure 61: Inherited Orphan Blood Diseases Market, Deal Segmentation by Deal Type
Figure 62: GBI Research Market Forecasting Model

Inherited Orphan Blood Diseases Patients Face Stagnant Treatment Future

Generally unimpressive treatment line-ups combined with weak product pipelines will lead to modest growth for several inherited orphan blood disease markets, states business intelligence provider GBI Research.

According to the firm’s new report*, the global therapeutic markets for sickle cell anemia, thalassemia and hereditary angioedema (HAE) are all expected to exhibit uninspired growth. The greatest value increase is expected from the sickle cell anemia market, which is forecast to climb at a Compound Annual Growth Rate (CAGR) of 9%, from US$36m in 2012 to US$70m in 2019.

Meanwhile, the global HAE therapeutics market is predicted to increase at a miserly CAGR of 3% during the same period, from US$1.5 billion to US$1.9 billion.

Kimberley Carter, Associate Analyst at GBI Research states: “Even though orphan blood disorders are rare, the three diseases featured in this report are hereditary and have a large impact on the lives of the families and ethnic groups afflicted by these diseases. Many of these diseases cause debilitating health issues and limit lifespan. Because of this there is a great need for curative and disease modifying therapies.

“While this may require significant R&D investment, any drugs that are successful in treating these diseases can be expected to be priced at the highest premium range.”

All three ailments studied in the new report have weak product pipelines, with most upcoming treatments either too similar to currently available options, or insufficiently promising to make any real impact.

At present, only the HAE market can be considered strong, thanks largely to the dominance of Cinryze – the only branded therapy taken according to a regular dosing regimen. Cinryze will continue to dominate the HAE market as long as it remains unique, and an exceptionally weak pipeline will most likely ensure this remains the case.

* Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need

This report provides insights into the up-and-coming trends of a portion of the inherited orphan blood disorder pharmaceutical market by examining sickle cell anemia, thalassemia and Hereditary Angioedema (HAE) in seven major markets.

This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by GBI Research’s team of industry experts.

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