Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential

Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential

Summary

GBI Research has released its latest research, “Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential”. The report provides insights into the up-and-coming trends of a portion of the acquired orphan blood disorder pharmaceutical market by examining Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF), Paroxysmal Nocturnal Hemoglobinuria (PNH) and Polycythemia Vera (PV) 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 MF market will be the fastest growing of the five markets during the forecast period, at a Compound Annual Growth Rate (CAGR) of 34% and reaching a total market size of $1.1bn in 2019. MDS represents the largest market, expected to reach $2.2bn by 2019.

Scope

- Current and future treatment trends for three rare diseases
- Market forecasts for five rare diseases across seven major markets
- Analysis of the developmental pipelines for five 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

1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 6
1.2 List of Figures 7
2 Acquired Orphan Blood Diseases - Introduction 9
2.1 Overview 9
3 Acquired Orphan Blood Diseases - Therapeutic Landscape 10
3.1 Paroxysmal Nocturnal Hemoglobinuria 10
3.1.1 Disease Overview 10
3.1.2 Revenues 12
3.1.3 Revenue Analysis by Country 13
3.1.4 Annual Cost of Therapy 17
3.1.5 Treatment Usage Patterns 18
3.1.6 Marketed Products 19
3.1.7 Market Drivers 20
3.1.8 Market Restraints 20
3.2 Paroxysmal Nocturnal Hemoglobinuria: Research and Development Pipeline 21
3.2.1 Overview 21
3.2.2 Trends in Paroxysmal Nocturnal Hemoglobinuria Pipeline 23
3.2.3 Promising Pipeline Molecules 24
3.2.4 Key Takeaway 24
3.3 Idiopathic Thrombocytopenic Purpura 25
3.3.1 Disease Overview 25
3.3.2 Revenues 27
3.3.3 Revenue Analysis by Country 28
3.3.4 Annual Cost of Therapy 32
3.3.5 Treatment Usage Patterns 33
3.3.6 Marketed Products 34
3.3.7 Market Drivers 34
3.3.8 Market Restraints 34
3.4 Idiopathic Thrombocytopenic Purpura: Research and Development Pipeline 35
3.4.1 Overview 35
3.4.2 Pipeline by Clinical Phase of Development 36
3.4.3 Trends in Idiopathic Thrombocytopenic Purpura Pipeline 40
3.4.4 Promising Pipeline Molecules 41
3.4.5 Key Takeaway 43
3.5 Myelodysplastic Syndrome 44
3.5.1 Disease Overview 44
3.5.2 Revenues 45
3.5.3 Revenue Analysis by Country 46
3.5.4 Annual Cost of Therapy 50
3.5.5 Treatment Usage Patterns 51
3.5.6 Marketed Products 52
3.5.7 Market Drivers 53
3.5.8 Market Restraints 53
3.6 Myelodysplastic Syndrome: Research and Development Pipeline 54
3.6.1 Overview 54
3.6.2 Pipeline by Clinical Phase of Development 55
3.6.3 Trends in Myelodysplastic Syndrome Pipeline 80
3.6.4 Promising Pipeline Molecules 83
3.6.5 Key Takeaway 83
3.7 Myelofibrosis 84
3.7.1 Disease Overview 84
3.7.2 Revenues 85
3.7.3 Revenue Analysis by Country 86
3.7.4 Annual Cost of Therapy 90
3.7.5 Treatment Usage Patterns 91
3.7.6 Marketed Products 92
3.7.7 Market Drivers 92
3.7.8 Market Restraints 92
3.8 Myelofibrosis: Research and Development Pipeline 93
3.8.1 Overview 93
3.8.2 Pipeline by Clinical Phase of Development 94
3.8.3 Trends in Myelofibrosis Pipeline 99
3.8.4 Promising Pipeline Molecules 101
3.8.5 Key Takeaway 101
3.9 Polycythemia Vera 102
3.9.1 Disease Overview 102
3.9.2 Revenues 103
3.9.3 Revenue Analysis by Country 104
3.9.4 Annual Cost of Therapy 108
3.9.5 Treatment Usage Patterns 109
3.9.6 Marketed Products 110
3.9.7 Market Drivers 111
3.9.8 Market Restraints 111
3.10 Polycythemia Vera: Research and Development Pipeline 112
3.10.1 Overview 112
3.10.2 Pipeline by Clinical Phase of Development 113
3.10.3 Trends in Polycythemia Vera Pipeline 116
3.10.4 Promising Pipeline Molecules 118
3.10.5 Key Takeaway 118
4 Acquired Orphan Blood Diseases - Strategic Consolidations 119
4.1 Overview 119
4.1.1 Segmentation by Year 119
4.1.2 Segmentation by Indication 119
4.1.3 Segmentation by Geography 120
4.1.4 Segmentation by Deal Value 120
4.1.5 Segmentation by Deal Type 121
4.2 Major Mergers and Acquisitions 121
4.3 Major Licensing Agreements 122
5 Acquired Orphan Blood Diseases - Appendix 123
5.1 Abbreviations 123
5.2 Sources 123
5.3 Research Methodology 124
5.3.1 Coverage 124
5.3.2 Secondary Research 124
5.3.3 Primary Research 125
5.3.4 Therapeutic Landscape 125
5.3.5 Geographical Landscape 128
5.3.6 Pipeline Analysis 128
5.3.7 Competitive Landscape 128
5.3.8 Expert Panel Validation 128
5.4 Contact Us 128
5.5 Disclaimer 128

1.1 List of Tables
Table 1: Approximate PNH Patient Population, by Country, 2011 10
Table 2: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019 12
Table 3: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%), 2011-2019 17
Table 4: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019 18
Table 5: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Molecule Details, 2012 22
Table 6: Idiopathic Thrombocytopenic Purpura, Approximate Patient Population by Country (‘000), 2011 25
Table 7: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019 27
Table 8: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%), 2011-2019 32
Table 9: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019 33
Table 10: Idiopathic Thrombocytopenic Purpura, Pipeline, Discovery, 2012 36
Table 11: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I, 2012 36
Table 12: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II, by Therapeutic Class, 2012 37
Table 13: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III, 2012 38
Table 14: Myelodysplastic Syndrome, Patient Population by Country (‘000), 2011 44
Table 15: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019 45
Table 16: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019 50
Table 17: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019 51
Table 18: Myelodysplastic Syndrome, Pipeline Overview, Phase I, 2012 55
Table 19: Myelodysplastic Syndrome, Pipeline Overview, Phase II 61
Table 20: Myelodysplastic Syndrome, Pipeline Overview, Phase III 77
Table 21: Myelodysplastic Syndrome, Pipeline Overview, Other 80
Table 22: Myelofibrosis, Patient Population by Country (‘000), 2011 84
Table 23: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019 85
Table 24: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019 90
Table 25: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019 91
Table 26: Myelofibrosis, Pipeline, Phase I, 2012 94
Table 27: Myelofibrosis, Pipeline, Phase II, 2012 95
Table 28: Myelofibrosis, Pipeline, Phase III, 2012 98
Table 29: Polycythemia Vera, Approximate Patient Population by Country (‘000), 2011 102
Table 30: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019 103
Table 31: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019 108
Table 32: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019 109
Table 33: Polycythemia Vera, Pipeline, Preclinical and Phase I, 2012 113
Table 34: Polycythemia Vera, Pipeline, Phase II, 2012 114
Table 35: Polycythemia Vera, Pipeline, Phase III, 2012 115

1.2 List of Figures
Figure 1: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019 12
Figure 2: Paroxysmal Nocturnal Hemoglobinuria, US, Revenue Forecast ($m), 2011-2019 13
Figure 3: Paroxysmal Nocturnal Hemoglobinuria, UK, Revenue Forecast ($m), 2011-2019 13
Figure 4: Paroxysmal Nocturnal Hemoglobinuria, France, Revenue Forecast ($m), 2011-2019 14
Figure 5: Paroxysmal Nocturnal Hemoglobinuria, Germany, Revenue Forecast ($m), 2011-2019 14
Figure 6: Paroxysmal Nocturnal Hemoglobinuria, Italy, Revenue Forecast ($m), 2011-2019 15
Figure 7: Paroxysmal Nocturnal Hemoglobinuria, Spain, Revenue Forecast ($m), 2011-2019 15
Figure 8: Paroxysmal Nocturnal Hemoglobinuria, Japan, Revenue Forecast ($m), 2011-2019 16
Figure 9: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%), 2011-2019 17
Figure 10: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019 18
Figure 11: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Overview 21
Figure 12: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Therapeutic Class 23
Figure 13: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Molecule Type 23
Figure 14: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Mechanism of Action 24
Figure 15: Idiopathic Thrombocytopenic Purpura, Underlying Mechanism 25
Figure 16: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019 27
Figure 17: Idiopathic Thrombocytopenic Purpura, US, Revenue Forecast ($m), 2011-2019 28
Figure 18: Idiopathic Thrombocytopenic Purpura, UK, Revenue Forecast ($m), 2011-2019 28
Figure 19: Idiopathic Thrombocytopenic Purpura, France, Revenue Forecast ($m), 2011-2019 29
Figure 20: Idiopathic Thrombocytopenic Purpura, Germany, Revenue Forecast ($m), 2011-2019 29
Figure 21: Idiopathic Thrombocytopenic Purpura, Italy, Revenue Forecast ($m), 2011-2019 30
Figure 22: Idiopathic Thrombocytopenic Purpura, Spain, Revenue Forecast ($m), 2011-2019 30
Figure 23: Idiopathic Thrombocytopenic Purpura, Japan, Revenue Forecast ($m), 2011-2019 31
Figure 24: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%), 2011-2019 32
Figure 25: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019 33
Figure 26: Idiopathic Thrombocytopenic Purpura, Pipeline Overview 35
Figure 27: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I by Therapeutic Class 36
Figure 28: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II Molecule Comparison 37
Figure 29: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III Molecule Comparison 39
Figure 30: Idiopathic Thrombocytopenic Purpura, Pipeline by Therapeutic Class 40
Figure 31: Idiopathic Thrombocytopenic Purpura, Pipeline by Molecule Type 40
Figure 32: Idiopathic Thrombocytopenic Purpura, Pipeline by Mechanism of Action 41
Figure 33: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019 45
Figure 34: Myelodysplastic Syndrome, US, Revenue Forecast ($m), 2011-2019 46
Figure 35: Myelodysplastic Syndrome, UK, Revenue Forecast ($m), 2011-2019 46
Figure 36: Myelodysplastic Syndrome, France, Revenue Forecast ($m), 2011-2019 47
Figure 37: Myelodysplastic Syndrome, Germany, Revenue Forecast ($m), 2011-2019 47
Figure 38: Myelodysplastic Syndrome, Italy, Revenue Forecast ($m), 2011-2019 48
Figure 39: Myelodysplastic Syndrome, Spain, Revenue Forecast ($m), 2011-2019 48
Figure 40: Myelodysplastic Syndrome, Japan, Revenue Forecast ($m), 2011-2019 49
Figure 41: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019 50
Figure 42: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019 51
Figure 43: Myelodysplastic Syndrome, Pipeline Overview 54
Figure 44: Myelodysplastic Syndrome, Pipeline, Phase I Molecule Comparison 60
Figure 45: Myelodysplastic Syndrome, Pipeline, Phase II Molecule Comparison 76
Figure 46: Myelodysplastic Syndrome, Pipeline, Phase III Molecule Comparison 79
Figure 47: Myelodysplastic Syndrome, Pipeline by Therapeutic Class 80
Figure 48: Myelodysplastic Syndrome, Pipeline by Molecule Type 81
Figure 49: Myelodysplastic Syndrome, Pipeline by Mechanism of Action 82
Figure 50: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019 85
Figure 51: Myelofibrosis, US, Revenue Forecast ($m), 2011-2019 86
Figure 52: Myelofibrosis, UK, Revenue Forecast ($m), 2011-2019 86
Figure 53: Myelofibrosis, France, Revenue Forecast ($m), 2011-2019 87
Figure 54: Myelofibrosis, Germany, Revenue Forecast ($m), 2011-2019 87
Figure 55: Myelofibrosis, Italy, Revenue Forecast ($m), 2011-2019 88
Figure 56: Myelofibrosis, Spain, Revenue Forecast ($m), 2011-2019 88
Figure 57: Myelofibrosis, Japan, Revenue Forecast ($m), 2011-2019 89
Figure 58: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019 90
Figure 59: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019 91
Figure 60: Myelofibrosis, Pipeline Overview 93
Figure 61: Myelofibrosis, Pipeline, Phase I Molecule Comparison 94
Figure 62: Myelofibrosis, Pipeline, Phase II Molecule Comparison 97
Figure 63: Myelofibrosis, Pipeline, Phase III Molecule Comparison 98
Figure 64: Myelofibrosis, Pipeline by Therapeutic Class 99
Figure 65: Myelofibrosis, Pipeline by Molecule Type 99
Figure 66: Myelofibrosis, Pipeline by Mechanism of Action 100
Figure 67: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019 103
Figure 68: Polycythemia Vera, US, Revenue Forecast ($m), 2011-2019 104
Figure 69: Polycythemia Vera, UK, Revenue Forecast ($m), 2011-2019 104
Figure 70: Polycythemia Vera, France, Revenue Forecast ($m), 2011-2019 105
Figure 71: Polycythemia Vera, Germany, Revenue Forecast ($m), 2011-2019 105
Figure 72: Polycythemia Vera, Italy, Revenue Forecast ($m), 2011-2019 106
Figure 73: Polycythemia Vera, Spain, Revenue Forecast ($m), 2011-2019 106
Figure 74: Polycythemia Vera, Japan, Revenue Forecast ($m), 2011-2019 107
Figure 75: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019 108
Figure 76: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019 109
Figure 77: Polycythemia Vera, Pipeline Overview 112
Figure 78: Polycythemia Vera, Pipeline, Phase I Molecule Comparison 113
Figure 79: Polycythemia Vera, Pipeline, Phase II Molecule Comparison 115
Figure 80: Polycythemia Vera, Pipeline by Therapeutic Class 116
Figure 81: Polycythemia Vera, Pipeline by Molecule Type 116
Figure 82: Polycythemia Vera, Pipeline by Mechanism of Action 117
Figure 83: Deal Segmentation by Year, 2004-2012 119
Figure 84: Deal Segmentation by Indication 119
Figure 85: Deal Segmentation by Geography, Total Market 120
Figure 86: Deal Segmentation by Value 120
Figure 87: Deal Segmentation by Deal Type 121
Figure 88: GBI Research Market Forecasting Model 127