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


#160071

81pages

GBI Research

$ 3500

In Stock


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

Summary

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 Researchs 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 but slowest growing market, expected to reach $1.89 billion by 2019 at a CAGR of 3%.

Scope


  • Current and future treatment trends for three rare diseases
  • Market forecasts for three rare diseases across seven major markets
  • Analysis of the developmental pipelines for three rare diseases
  • Information on recent industry deals

Reasons to buy


  • Understand how the inherited orphan blood diseases market has developed and is continuing to develop within the featured markets
  • Predict which products are likely to be most successful in the future
  • Gain an understanding of the possible market available for specific products
Table of Contents

1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 6
1.2 List of Figures 6

2 Inherited Orphan Blood Diseases - Introduction 8
2.1 Overview 8

3 Inherited Orphan Blood Disorders - Therapeutic Landscape 9
3.1 Sickle Cell Anemia 9
3.1.1 Disease Overview 9
3.1.2 Revenues 14
3.1.3 Revenue Analysis by Country 14
3.1.4 Annual Cost of Therapy 18
3.1.5 Treatment Usage Patterns 19
3.1.6 Marketed Products 20
3.1.7 Market Drivers 21
3.1.8 Market Restraints 21
3.2 Sickle Cell Anemia: Research and Development Pipeline 22
3.2.1 Overview 22
3.2.2 Pipeline by Clinical Phase of Development 23
3.2.3 Trends in Sickle Cell Anemia Pipeline 28
3.2.4 Promising Pipeline Molecules 30
3.2.5 Key Takeaway 32
3.3 Thalassemia 33
3.3.1 Disease Overview 33
3.3.2 Revenues 38
3.3.3 Revenue Analysis by Country 39
3.3.4 Annual Cost of Therapy 43
3.3.5 Treatment Usage Patterns 44
3.3.6 Marketed Products 44
3.3.7 Market Drivers 46
3.3.8 Market Restraints 46
3.4 Thalassemia - Research and Development Pipeline 47
3.4.1 Overview 47
3.4.2 Pipeline by Clinical Phase of Development 48
3.4.3 Trends in Thalassemia Pipeline 51
3.4.4 Promising Pipeline Molecules 53
3.4.5 Key Takeaway 54
3.5 Hereditary Angioedema 55
3.5.1 Disease Overview 55
3.5.2 Revenues 56
3.5.3 Revenue Analysis by Country 57
3.5.4 Annual Cost of Therapy 61
3.5.5 Treatment Usage Patterns 62
3.5.6 Marketed Products 63
3.5.7 Market Drivers 64
3.5.8 Market Restraints 64
3.6 Hereditary Angioedema: Research and Development Pipeline 65
3.6.1 Overview 65
3.6.2 Pipeline by Clinical Phase of Development 66
3.6.3 Trends in Hereditary Angioedema Pipeline 69
3.6.4 Promising Pipeline Molecules 71
3.6.5 Key Takeaway 71

4 Inherited Orphan Blood Disorders Therapeutics - Strategic Consolidations 72
4.1 Segmentation by Year 72
4.2 Segmentation by Indication 72
4.3 Segmentation by Geography 73
4.4 Segmentation by Deal Value 73
4.5 Segmentation by Deal Type 74
4.6 Major Mergers and Acquisitions 74
4.7 Major Licensing Agreements 75

5 Inherited Orphan Blood Disorders Therapeutics - Appendix 76
5.1 Market Definitions 76
5.2 Abbreviations 76
5.3 Sources 76
5.4 Research Methodology 77
5.4.1 Coverage 77
5.4.2 Secondary Research 77
5.4.3 Primary Research 78
5.4.4 Therapeutic Landscape 78
5.4.5 Geographical Landscape 81
5.4.6 Pipeline Analysis 81
5.4.7 Competitive Landscape 81
5.4.8 Expert Panel Validation 81
5.5 Contact Us 81
5.6 Disclaimer 81

Table 1: Probability of HbSS Genotype by Region 11
Table 2: Sickle Cell Anemia, Total Market, CAGR of Average Annual Cost of Therapy 18
Table 3: Sickle Cell Anemia, Pipeline, Discovery 23
Table 4: Sickle Cell Anemia, Pipeline, Pre-Clinical 23
Table 5: Sickle Cell Anemia, Pipeline, Phase I 24
Table 6: Sickle Cell Anemia, Pipeline, Phase II 25
Table 7: Sickle Cell Anemia, Pipeline, Phase III 27
Table 8: Prevalence of
Figure 1: Comparison of Normal Human Hemoglobin and Hemoglobin S 9
Figure 2: Comparison of Shape and Flow of Normal Erythrocytes and Sickle Cells 10
Figure 3: Global Distribution of Sickle Cell Gene (HbS) and Malaria 11
Figure 4: Sickle Cell Anemia, Total Market, Revenue Forecast ($m), 20112019 14
Figure 5: Sickle Cell Anemia, US, Revenue Forecast ($m), 20112019 14
Figure 6: Sickle Cell Anemia, UK, Revenue Forecast ($m), 20112019 15
Figure 7: Sickle Cell Anemia, France, Revenue Forecast ($m), 20112019 15
Figure 8: Sickle Cell Anemia, Germany, Revenue Forecast ($m), 20112019 16
Figure 9: Sickle Cell Anemia, Italy, Revenue Forecast ($m), 20112019 16
Figure 10: Sickle Cell Anemia, Spain, Revenue Forecast ($m), 20112019 17
Figure 11: Sickle Cell Anemia, Total Market, Average Annual Cost of Therapy, 20112019 18
Figure 12: Sickle Cell Anemia, Total Market, Treatment Usage Pattern (000), 20112019 19
Figure 13: Hydroxyurea Factsheet 20
Figure 14: Sickle Cell Anemia, Pipeline, Overview 22
Figure 15: Sickle Cell Anemia, Pipeline, Pre-Clinical Molecule Comparison 23
Figure 16: Sickle Cell Anemia, Pipeline, Phase I Molecule Comparison 24
Figure 17: Sickle Cell Anemia, Pipeline, Phase II Molecule Comparison 26
Figure 18: Sickle Cell Anemia, Pipeline, Phase III Molecule Comparison 27
Figure 19: Sickle Cell Anemia, Pipeline by Therapeutic Class 28
Figure 20: Sickle Cell Anemia, Pipeline by Molecule Type 28
Figure 21: Sickle Cell Anemia, Pipeline by Mechanism of Action 29
Figure 22: Hemoglobin Tetramer with