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Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need
Date:1/29/2013

NEW YORK, Jan. 29, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

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

http://www.reportlinker.com/p01089007/Inherited-Orphan-Blood-Disorders-Therapeutics-Market-to-2019---Breakthrough-Drugs-Remain-Elusive-Against-Backdrop-of-High-Unmet-Need.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Pathology

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

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

 

To order this report:

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

Nicolas Bombourg
Reportlinker
Email: nicolasbombourg@reportlinker.com
US: (805)652-2626
Intl: +1 805-652-2626

 


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