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 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 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

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



List of Tables

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 ?- and ?-Thalassemia (Hemoglobin H and ?-Thalassemia Intermedia and Major only) 35
Table 9: Thalassemia, Total Market, CAGR of Average Annual Cost of Therapy 43
Table 10: Thalassemia, Pipeline, Discovery 48
Table 11: Thalassemia, Pipeline, Pre-Clinical 48
Table 12: Thalassemia, Pipeline, Phase I 48
Table 13: Thalassemia, Pipeline, Phase II 50
Table 14: Thalassemia, Pipeline, Phase III 51
Table 15: Global Distribution of Hereditary Angioedema 55
Table 16: Hereditary Angioedema, Total Market, CAGR of Average Annual Cost of Therapy 61
Table 17: Hereditary Angioedema, Pipeline, Discovery 66
Table 18: Hereditary Angioedema, Pipeline, Pre-Clinical 66
Table 19: Hereditary Angioedema, Pipeline, Phase I 67
Table 20: Hereditary Angioedema, Pipeline, Phase II 67
Table 21: Hereditary Angioedema, Pipeline, Phase III 68



List of Figures

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 ($m), 2011-2019 14
Figure 5: Sickle Cell Anemia, US, Revenue Forecast ($m), 2011-2019 14
Figure 6: Sickle Cell Anemia, UK, Revenue Forecast ($m), 2011-2019 15
Figure 7: Sickle Cell Anemia, France, Revenue Forecast ($m), 2011-2019 15
Figure 8: Sickle Cell Anemia, Germany, Revenue Forecast ($m), 2011-2019 16
Figure 9: Sickle Cell Anemia, Italy, Revenue Forecast ($m), 2011-2019 16
Figure 10: Sickle Cell Anemia, Spain, Revenue Forecast ($m), 2011-2019 17
Figure 11: Sickle Cell Anemia, Total Market, Average Annual Cost of Therapy, 2011-2019 18
Figure 12: Sickle Cell Anemia, Total Market, Treatment Usage Pattern (‘000), 2011-2019 19
Figure 13: Hydroxyurea Factsheet 20
Figure 14: Sickle Cell Anemia, Pipeline, Overview 22
Figure 15: Sickle Cell Anemia, Pipeline, Pre-Clinical Molecule Comparison 23
Figure 16: Sickle Cell Anemia, Pipeline, Phase I Molecule Comparison 24
Figure 17: Sickle Cell Anemia, Pipeline, Phase II Molecule Comparison 26
Figure 18: Sickle Cell Anemia, Pipeline, Phase III Molecule Comparison 27
Figure 19: Sickle Cell Anemia, Pipeline by Therapeutic Class 28
Figure 20: Sickle Cell Anemia, Pipeline by Molecule Type 28
Figure 21: Sickle Cell Anemia, Pipeline by Mechanism of Action 29
Figure 22: Hemoglobin Tetramer with ?- and ?-Globulin Chains 33
Figure 23: Global Distribution of ?- and ?-Thalassemia 35
Figure 24: Thalassemia, Total Market, Revenue Forecast ($m), 2011-2019 38
Figure 25: Thalassemia, US, Revenue Forecast ($m), 2011-2019 39
Figure 26: Thalassemia, UK, Revenue Forecast ($m), 2011-2019 39
Figure 27: Thalassemia, France, Revenue Forecast ($m), 2011-2019 40
Figure 28: Thalassemia, Germany, Revenue Forecast ($m), 2011-2019 40
Figure 29: Thalassemia, Italy, Revenue Forecast ($m), 2011-2019 41
Figure 30: Thalassemia, Spain, Revenue Forecast ($m), 2011-2019 41
Figure 31: Thalassemia, Japan, Revenue Forecast ($m), 2011-2019 42
Figure 32: Thalassemia, Total Market, Average Annual Cost of Therapy, 2011-2019 43
Figure 33: Thalassemia, Total Market, Treatment Usage Pattern 44
Figure 34: Thalassemia, Pipeline, Overview 47
Figure 35: Thalassemia: Pipeline, Phase I Molecule Comparison 49
Figure 36: Thalassemia, Pipeline, Phase II Molecule Comparison 50
Figure 37: Thalassemia, Pipeline by Therapeutic Class 51
Figure 38: Thalassemia, Pipeline by Molecule Type 51
Figure 39: Thalassemia, Pipeline by Mechanism of Action 52
Figure 40: Hereditary Angioedema, Total Market, Revenue Forecast($m), 2011-2019 56
Figure 41: Hereditary Angioedema, US, Revenue Forecast ($m), 2011-2019 57
Figure 42: Hereditary Angioedema, UK, Revenue Forecast ($m), 2011-2019 57
Figure 43: Hereditary Angioedema, France, Revenue Forecast ($m), 2011-2019 58
Figure 44: Hereditary Angioedema, Germany, Revenue Forecast ($m), 2011-2019 58
Figure 45: Hereditary Angioedema, Italy, Revenue Forecast ($m), 2011-2019 59
Figure 46: Hereditary Angioedema, Spain, Revenue Forecast ($m), 2011-2019 59
Figure 47: Hereditary Angioedema, Japan, Revenue Forecast ($m), 2011-2019 60
Figure 48: Hereditary Angioedema, Total Market, Average Annual Cost of Therapy, 2011-2019 61
Figure 49: Hereditary Angioedema, Total Market, Treatment Usage Pattern 62
Figure 50: Hereditary Angioedema, Pipeline, Overview 65
Figure 51: Hereditary Angioedema, Pipeline, Pre-Clinical Molecule Comparison 66
Figure 52: Hereditary Angioedema Pipeline, Phase II Molecule Comparison 67
Figure 53: Hereditary Angioedema, Pipeline, Phase III Molecule Comparison 68
Figure 54: Hereditary Angioedema, Pipeline by Therapeutic Class 69
Figure 55: Hereditary Angioedema, Pipeline by Molecule Type 69
Figure 56: Hereditary Angioedema, Pipeline by Mechanism of Action 70
Figure 57: Inherited Orphan Blood Diseases Market, Deal Segmentation by Year, 2000-2012 72
Figure 58: Inherited Orphan Blood Diseases Market, Deal Segmentation by Indication 72
Figure 59: Inherited Orphan Blood Diseases Market, Deal Segmentation by Geography 73
Figure 60: Inherited Orphan Blood Diseases Market, Deal Segmentation by Value 73
Figure 61: Inherited Orphan Blood Diseases Market, Deal Segmentation by Deal Type 74
Figure 62: GBI Research Market Forecasting Model 80

View This Report »

Find all the market research you need - instantly, in one place.

+1.2 Million Research Documents & Statistics +200,000 Trusted Public Sources 350 Industries With Global Coverage

24/7 Customer Support

Talk to Amrita

+1 718 303 2019

Purchase Reports From Reputable Market Research Publishers

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2023

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2023

  • $ 10 995
  • Industry report
  • July 2014
  • by Global Data

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2023 Summary GlobalData estimates that the total market size for the 7MM in 2013 was $12.3bn, comprised of $5.0bn in branded ...

Analysis of the Global Infectious Disease Diagnostics Market

Analysis of the Global Infectious Disease Diagnostics Market

  • $ 10 000
  • Industry report
  • September 2014
  • by Frost & Sullivan

Molecular Methods Present Significant Opportunities This deliverable provides research and analysis of the global infectious disease diagnostics market. It includes an in-depth analysis of revenue forecast ...

Frontier Pharma: Breast Cancer - Identifying and Commercializing First-in-Class Innovation

Frontier Pharma: Breast Cancer - Identifying and Commercializing First-in-Class Innovation

  • $ 6 995
  • Industry report
  • July 2014
  • by GBI Research

Frontier Pharma: Breast Cancer - Identifying and Commercializing First-in-Class Innovation Summary Exceptionally Large and Innovative Pipeline The breast cancer pipeline is the largest in the pharmaceutical ...

Bcc Research Bitopertin Forecast

August 2014 $ 5 500

1 Companies

Company Profiles

Pfizer Inc.

United States

ReportLinker is an award-winning market research solution that finds, filters and organizes the latest industry data so you get all the market research you need - quickly, in one place.