Table of Contents
1 Table of Contents 1
1.1 List of Tables 3
1.2 List of Figures 4
2 Inherited Orphan Blood Diseases - Executive Summary 5
3 Inherited Orphan Blood Disorders - Introduction 5
3.1 Overview 5
3.2 GBI Research Report Guidance 5
4 Inherited Orphan Blood Disorders - Therapeutic Landscape 6
4.1 Sickle Cell Anemia 6
4.1.1 Disease Overview 6
4.2 Thalassemia 12
4.2.1 Disease Overview 12
4.3 Hereditary Angioedema 17
4.3.1 Disease Overview 17
5 Inherited Orphan Blood Disorders - Market Forecasts 19
5.1 Sickle Cell Anemia: Revenues 19
5.1.1 Revenue Analysis by Country 20
5.1.2 Annual Cost of Therapy 23
5.1.3 Treatment Usage Patterns 24
5.1.4 Marketed Products 25
5.1.5 Market Drivers 26
5.1.6 Market Restraints 26
5.2 Thalassemia: Revenues 27
5.2.1 Revenue Analysis by Country 28
5.2.2 Annual Cost of Therapy 31
5.2.3 Treatment Usage Patterns 32
5.2.4 Marketed Products 33
5.2.5 Market Drivers 34
5.2.6 Market Restraints 35
5.3 Hereditary Angiedema: Revenues 36
5.3.1 Revenue Analysis by Country 36
5.3.2 Annual Cost of Therapy 40
5.3.3 Treatment Usage Patterns 41
5.3.4 Marketed Products 41
5.3.5 Market Drivers 42
5.3.6 Market Restraints 43
6 Inherited Orphan Blood Disorders - Research and Development Pipeline 43
6.1 Sickle Cell Anemia: Research and Development Pipeline 48
6.1.1 Overview 48
6.1.2 Pipeline by Clinical Phase of Development 49
6.1.3 Trends in Sickle Cell Anemia Pipeline 50
6.1.4 Promising Pipeline Molecules 50
6.1.5 Key Takeaway 52
6.2 Thalassemia - Research and Development Pipeline 53
6.2.1 Overview 53
6.2.2 Pipeline by Clinical Phase of Development 54
6.2.3 Promising Pipeline Molecules 54
6.2.4 Key Takeaway 56
6.3 Hereditary Angioedema: Research and Development Pipeline 57
6.3.1 Overview 57
6.3.2 Pipeline by Clinical Phase of Development 58
6.3.3 Promising Pipeline Molecules 58
6.3.4 Key Takeaway 59
7 Inherited Orphan Blood Disorders Therapeutics - Strategic Consolidations 59
7.1 Segmentation by Year 59
7.2 Segmentation by Indication 59
7.3 Segmentation by Geography 61
7.4 Segmentation by Deal Value 61
7.5 Segmentation by Deal Type 62
7.6 Major Mergers and Acquisitions 62
7.7 Major Licensing Agreements 63
8 Inherited Orphan Blood Disorders Therapeutics - Appendix 63
8.1 Market Definitions 63
8.2 Abbreviations 63
8.3 Sources 64
8.4 Research Methodology 65
8.4.1 Coverage 65
8.4.2 Secondary Research 65
8.4.3 Primary Research 66
8.4.4 Therapeutic Landscape 67
8.4.5 Geographical Landscape 69
8.4.6 Pipeline Analysis 70
8.4.7 Competitive Landscape 70
8.4.8 Expert Panel Validation 70
8.5 Contact Us 70
8.6 Disclaimer 70