Orphan Autoimmune Disorders to 2017 - Strong Performance by Nplate and Promacta and Future Launches of Immunoglobulin Therapies will Drive the ITP Market

Summary

Company’s report, “Orphan Autoimmune Disorders to 2017 - Strong Performance by Nplate and Promacta and Future Launches of Immunoglobulin Therapies will Drive the ITP Market” provides in-depth analysis of the drivers and barriers that affect the global orphan autoimmune disorders. The report analyzes the markets for orphan autoimmune disorders therapeutics in the US, the top five countries in Europe (the UK, Germany, France, Italy and Spain) and Japan. Treatment usage patterns, sales, price and volume are forecast until 2017 for the leading therapeutic segments. The report provides competitive benchmarking for the leading companies and analyzes the mergers, acquisitions and licensing agreements that shape the global markets.

It is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by Company’s team of industry experts.

Company finds that the orphan autoimmune disorders market will show steady growth during the period 2010-2017. This growth will primarily be driven by the growth of the Idiopathic Thrombocytopenic Purpura (ITP) market, which has shown tremendous growth in terms of revenue between 2008 and 2010. Moreover, other key markets, such as myasthenia gravis and amyotrophic lateral sclerosis (ALS), will continue to grow steadily in the presence of very few molecules in the late stage of the product pipeline. The global orphan autoimmune disorders market is a niche market characterized by stiff competition from generics and off-label drugs and very few licensing agreements, co-development and M&A deals.

Scope

- Annualized market data for the orphan autoimmune disorders therapeutics market from 2002 to 2010, and forecast to 2017.
- Analysis of the leading therapeutic segments. These include ITP, myasthenia gravis and ALS.
- Analysis of the orphan autoimmune disorders therapeutic markets in the leading geographies of the world, which include the US, top five countries of Europe and Japan.
- Market characterization of the orphan autoimmune disorders (ITP, myasthenia gravis and ALS) market including market size, revenue analysis by top seven geographies, generic share, annual cost of therapy, and treatment usage patterns.
- Key drivers and barriers that have a significant impact on the market.
- Coverage of the pipeline molecules in various phases of drug development.
- Competitive benchmarking of the leading companies. The key companies studied in this report are Amgen Inc., GlaxoSmithKline plc, CSL Behring LLC., Mitsubishi Tanabe Pharma Corporation and Biogen Idec.
- Key M&A activities and licensing agreements that have taken place between 2004 and 2011 in the global orphan autoimmune disorders therapeutics market.

Reasons to buy

- Align your product portfolio to the markets with high growth potential.
- Develop market-entry and market expansion strategies by identifying the leading therapeutic segments and geographic markets poised for strong growth.
- Reinforce R&D pipelines by identifying new target mechanisms which can produce first-in-class molecules with more efficiency and better safety.
- Develop key strategic initiatives by understanding the key focus areas of the leading companies.

1 Table of Contents 3
1.1 List of Tables 6
1.2 List of Figures 8
2 Orphan Autoimmune Disorders to 2017 - Introduction 9
2.1 Overview 9
2.2 GBI Research Report Guidance 10
3 Orphan Autoimmune Disorders to 2017 - Market Characterization 11
3.1 Market Forecasts 11
3.1.1 Revenue 11
3.1.2 Market Share of Orphan Diseases 13
3.1.3 Generic/Off-Label Share 14
3.2 Drivers and Restraints for the Global Orphan Autoimmune Disorders Therapeutics Market 15
3.2.1 Market Drivers 15
3.2.2 Market Restraints 15
4 Orphan Autoimmune Disorders to 2017 - Geographical Landscape 17
4.1 Revenue Analysis by Geography 17
4.2 The US 19
4.2.1 Revenue 19
4.3 Top Five Countries of Europe 20
4.3.1 Revenue 20
4.4 Japan 21
4.4.1 Revenue 21
5 Orphan Autoimmune Disorders to 2017 - Therapeutic Landscape 23
5.1 Global Idiopathic Thrombocytopenic Purpura (ITP) Therapeutics Market Forecasts 23
5.1.1 Disease Overview 23
5.1.2 Revenue 25
5.1.3 Generic Share 26
5.1.4 Revenue Analysis by Country 27
5.1.5 Annual Cost of Treatment 29
5.1.6 Treatment Usage Patterns 30
5.1.7 Major Marketed Products 32
5.2 Drivers and Restraints of the ITP Therapeutics Market 33
5.2.1 Market Drivers 33
5.2.2 Market Restraints 33
5.3 Global Myasthenia Gravis Therapeutics Market Forecasts 34
5.3.1 Disease Overview 34
5.3.2 Revenue 35
5.3.3 Generic Share 36
5.3.4 Revenue Analysis by Country 37
5.3.5 Annual Cost of Treatment 39
5.3.6 Treatment Usage Patterns 40
5.3.7 Major Marketed Products 42
5.4 Drivers and Restraints of the Myasthenia Gravis Therapeutics Market 42
5.4.1 Market Drivers 42
5.4.2 Market Restraints 42
5.5 Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market Forecasts 43
5.5.1 Disease Overview 43
5.5.2 Revenue 44
5.5.3 Revenue Analysis by Country 46
5.5.4 Annual Cost of Treatment 49
5.5.5 Treatment Usage Patterns 50
5.5.6 Major Marketed Products 52
5.6 Drivers and Restraints of the ALS Therapeutics Market 52
5.6.1 Market Drivers 52
5.6.2 Market Restraints 52
6 Orphan Autoimmune Disorders to 2017 - Pipeline Analysis 54
6.1 Research and Development Pipeline by Phase 54
6.2 Research and Development Pipeline by Indication 55
6.3 Idiopathic Thrombocytopenic Purpura (ITP): Research and Development Pipeline 56
6.3.1 Overview 56
6.3.2 Pipeline by Clinical Phases of Development 57
6.4 Myasthenia Gravis: Research and Development Pipeline 58
6.4.1 Overview 58
6.4.2 Pipeline by Clinical Phases of Development 59
6.5 Amyotrophic Lateral Sclerosis (ALS): Research and Development Pipeline 60
6.5.1 Overview 60
6.5.2 Pipeline by Clinical Phases of Development 61
6.6 Promising Drugs under Clinical Development for Orphan Autoimmune Disorders 64
6.6.1 Venoglobulin-IH 64
6.6.2 Gammaplex 65
6.6.3 IGIV3I Grifols 65
6.6.4 Olesoxime (TRO19622) 66
7 Orphan Autoimmune Disorders to 2017 - Competitive Profiling 67
7.1 Amgen Inc. 67
7.1.1 Company Overview 67
7.1.2 Marketed Products for Orphan Autoimmune Disorders 67
7.1.3 SWOT Analysis 67
7.2 GlaxoSmithKline 68
7.2.1 Company Overview 68
7.2.2 Marketed Products for Orphan Autoimmune Disorders 68
7.2.3 SWOT Analysis 68
7.3 CSL Behring LLC 69
7.3.1 Company Overview 69
7.3.2 Marketed Products for Orphan Autoimmune Disorders 69
7.3.3 SWOT Analysis 69
7.4 Mitsubishi Tanabe Pharma Corporation 70
7.4.1 Company Overview 70
7.4.2 Orphan Autoimmune Disorders- R&D Pipeline 70
7.4.3 SWOT Analysis 70
7.5 Biogen Idec. 71
7.5.1 Company Overview 71
7.5.2 Orphan Autoimmune Disorders- R&D Pipeline 71
7.5.3 SWOT Analysis 72
8 Orphan Autoimmune Disorders to 2017 - Strategic Consolidations 73
8.1 Overview 73
8.2 Segmentation by Year 74
8.3 Segmentation by Indication 75
8.4 Segmentation by Geography 76
8.5 Segmentation by Deal Value 77
8.6 Major Mergers and Acquisitions (M&A) 78
8.7 Major Licensing Agreements 79
8.8 Major Co-Development Deals 80
9 Orphan Autoimmune Disorders to 2017 - Appendix 81
9.1 Market Definitions 81
9.2 Abbreviations 81
9.3 Research Methodology 82
9.3.1 Coverage 82
9.3.2 Secondary Research 83
9.3.3 Primary Research 83
9.3.4 Therapeutic Landscape 84
9.3.5 Epidemiology-based Forecasting 84
9.3.6 Market Size by Geography 86
9.3.7 Geographical Landscape 87
9.3.8 Pipeline Analysis 87
9.3.9 Competitive Landscape 87
9.3.10 Expert Panel Validation 87
9.4 Contact Us 87
9.5 Disclaimer 87
9.6 Sources 88 1.1

 List of Tables

Table 1: Orphan Autoimmune Disorders, Global, Revenue ($m), 2002- 2010 11
Table 2: Orphan Autoimmune Disorders, Global, Revenue ($m), 2010- 2017 12
Table 3: Orphan Autoimmune Disorders, Market Share of Orphan Diseases, (%), 2010 13
Table 4: Orphan Autoimmune Disorders, Global, Revenue Analysis by Geography ($m), 2002-2010 18
Table 5: Orphan Autoimmune Disorders, Global, Revenue Analysis by Geography ($m), 2010-2017 18
Table 6: Orphan Autoimmune Disorders, The US, Revenue ($m), 2002-2010 19
Table 7: Orphan Autoimmune Disorders, The US, Revenue ($m), 2010-2017 19
Table 8: Orphan Autoimmune Disorders, Top Five Countries of Europe, Revenue ($m), 2002-2010 20
Table 9: Orphan Autoimmune Disorders, Top Five Countries of Europe, Revenue ($m), 2010-2017 20
Table 10: Orphan Autoimmune Disorders, Japan, Revenue ($m), 2002-2010 21
Table 11: Orphan Autoimmune Disorders, Japan, Revenue ($m), 2010-2017 21
Table 12: ITP Therapeutics Market, Global, Revenue ($m), 2002-2010 25
Table 13: ITP Therapeutics Market, Global, Revenue ($m), 2010-2017 25
Table 14: ITP Therapeutics Market, Global, Revenue Analysis by Country ($m), 2002-2010 27
Table 15: ITP Therapeutics Market, Global, Revenue Analysis by Country ($m), 2010-2017 28
Table 16: ITP Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2010 29
Table 17: ITP Therapeutics Market, Global, Annual Cost of Treatment ($), 2010-2017 29
Table 18: ITP Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2002-2010 30
Table 19: ITP Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2010-2017 31
Table 20: Myasthenia Gravis Therapeutics Market, Global, Revenue ($m), 2002-2010 35
Table 21: Myasthenia Gravis Therapeutics Market, Global, Revenue ($m), 2010-2017 35
Table 22: Myasthenia Gravis Therapeutics Market, Global, Revenue Analysis by Country ($m), 2002-2010 37
Table 23: Myasthenia Gravis Therapeutics Market, Global, Revenue Analysis by Country ($m), 2010-2017 38
Table 24: Myasthenia Gravis Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2010 39
Table 25: Myasthenia Gravis Therapeutics Market, Global, Annual Cost of Treatment ($), 2010-2017 39
Table 26: Myasthenia Gravis Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2002-2010 40
Table 27: Myasthenia Gravis Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2010-2017 41
Table 28: ALS Therapeutics Market, Global, Revenue Forecasts ($m), 2002-2010 45
Table 29: ALS Therapeutics Market, Global, Revenue Forecasts ($m), 2010-2017 45
Table 30: ALS Therapeutics Market, Global, Revenue by Country ($m), 2002-2010 46
Table 31: ALS Therapeutics Market, Global, Revenue by Country ($m), 2010-2017 47
Table 32: ALS Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2010 49
Table 33: ALS Therapeutics Market, Annual Cost of Treatment, Global, ($), 2010-2017 49
Table 34: ALS Therapeutics Market, Global Treatment Usage Pattern (hundred), 2002-2010 50
Table 35: ALS Therapeutics Market, Global Treatment Usage Pattern (hundred), 2010-2017 51
Table 36: Orphan Autoimmune Disorders, Global, Pipeline Molecules by Phase, (%), 2011 54
Table 37: Orphan Autoimmune Disorders, Global, Pipeline Molecules by Indication, (%), 2011 55
Table 38: ITP Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 56
Table 39: ITP Therapeutics Market, Global, Preclinical Pipeline Molecules, 2011 57
Table 40: ITP Therapeutics Market, Global, Phase I Pipeline Molecules, 2011 57
Table 41: ITP Therapeutics Market, Global, Phase II Pipeline Molecules, 2011 57
Table 42: ITP Therapeutics Market, Global, Phase III Pipeline Molecules, 2011 57
Table 43: Myasthenia Gravis Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 58
Table 44: Myasthenia Gravis Therapeutics Market, Global, IND Filed Pipeline Molecules, 2011 59
Table 45: Myasthenia Gravis Therapeutics Market, Global, Phase II Pipeline Molecules, 2011 59
Table 46: Myasthenia Gravis Therapeutics Market, Global, Phase III/NDA Filed Pipeline Molecules, 2011 59
Table 47: ALS Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 60
Table 48: ALS Therapeutics Market, Global, Discovery Pipeline Molecules, 2011 61
Table 49: ALS Therapeutics Market, Global, Preclinical Pipeline Molecules, 2011 62
Table 50: ALS Therapeutics Market, Global, Phase I Pipeline Molecules, 2011 63
Table 51: ALS Therapeutics Market, Global, Phase II Pipeline Molecules, 2011 63
Table 52: ALS Therapeutics Market, Global, Phase III Pipeline Molecules, 2011 64
Table 53: Orphan Autoimmune Disorder Therapeutics Market, Mitsubishi Tanabe, Pipeline Products for Orphan Autoimmune Disorders, 2011 70
Table 54: Orphan Autoimmune Disorder Therapeutics Market, Biogen Idec, Pipeline Products for Orphan Autoimmune Disorders, 2011 71
Table 55: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Type, (%), 2004-2011 73
Table 56: Orphan Autoimmune Disorder Therapeutics Market, Global, Number of Deals by Year, 2004-2011 74
Table 57: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Indication, (%), 2004-2011 75
Table 58: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Geography, (%), 2004-2011 76
Table 59: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Value, (%), 2004-2011 77
Table 60: Orphan Autoimmune Disorder Therapeutics Market, Global, Major M&A Deals, 2007-2011 78
Table 61: Orphan Autoimmune Disorder Therapeutics Market, Global, Major Licensing Agreements, 2007-2011 79
Table 62: Orphan Autoimmune Disorder Therapeutics Market, Global, Major Co-Development Deals, 2006-2011 80 1.2

 List of Figures

Figure 1: Orphan Autoimmune Disorders, Global, Revenue ($m), 2002- 2017 11
Figure 2: Orphan Autoimmune Disorders, Market Share of Orphan Diseases (%), 2010 13
Figure 3: Orphan Autoimmune Disorders, Global, Generic/Off-Label Revenue Share (%), 2010 14
Figure 4: Orphan Autoimmune Disorders, Global, Revenue Analysis by Geography ($m), 2002-2017 17
Figure 5: Orphan Autoimmune Disorders, The US, Revenue ($m), 2002-2017 19
Figure 6: Orphan Autoimmune Disorders, Top Five Countries of Europe, Revenue ($m), 2002-2017 20
Figure 7: Orphan Autoimmune Disorders, Japan, Revenue ($m), 2002-2017 21
Figure 8: ITP Therapeutics Market, Global, Revenue ($m), 2002-2017 25
Figure 9: ITP Therapeutics Market, Global, Generic Share(%), 2010, 2017 26
Figure 10: ITP Therapeutics Market, Global, Revenue Analysis by Country ($m), 2002-2017 27
Figure 11: ITP Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2017 29
Figure 12: ITP Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2002-2017 30
Figure 13: Myasthenia Gravis Therapeutics Market, Global, Revenue ($m), 2002-2017 35
Figure 14: Myasthenia Gravis Therapeutics Market, Global, Generic Share (%), 2010, 2017 36
Figure 15: Myasthenia Gravis Therapeutics Market, Global, Revenue Analysis by Country ($m), 2002-2017 37
Figure 16: Myasthenia Gravis Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2017 39
Figure 17: Myasthenia Gravis Therapeutics Market, Global, Treatment Usage Patterns (‘000), 2002-2017 40
Figure 18: ALS Therapeutics Market, Global, Revenue Forecasts ($m), 2002-2017 44
Figure 19: ALS Therapeutics Market, Global, Revenue by Country ($m), 2002-2017 46
Figure 20: ALS Therapeutics Market, Global, Generic Share (%), 2010, 2017 48
Figure 21: ALS Therapeutics Market, Global, Annual Cost of Treatment ($), 2002-2017 49
Figure 22: ALS Therapeutics Market, Global Treatment Usage Pattern (hundred), 2002-2017 50
Figure 23: Orphan Autoimmune Disorders, Global, Pipeline Molecules by Phase, (%), 2011 54
Figure 24: Orphan Autoimmune Disorders, Global, Pipeline Molecules by Indication, (%), 2011 55
Figure 25: ITP Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 56
Figure 26: Myasthenia Gravis Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 58
Figure 27: ALS Therapeutics Market, Global, Pipeline Molecules by Phase, (%), 2011 60
Figure 28: Orphan Autoimmune Disorders, Amgen Inc, SWOT Analysis, 2011 67
Figure 29: Orphan Autoimmune Disorders, GlaxoSmithKline plc., SWOT Analysis, 2011 68
Figure 30: Orphan Autoimmune Disorders, CSL Behring LLC, SWOT Analysis, 2011 69
Figure 31: Orphan Autoimmune Disorders, Mitsubishi Tanabe Pharma Corporation, SWOT Analysis, 2011 70
Figure 32: Orphan Autoimmune Disorders, Biogen Idec, SWOT Analysis, 2011 72
Figure 33: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Type (%), 2004-2011 73
Figure 34: Orphan Autoimmune Disorder Therapeutics Market, Global, Number of Deals by Year (%), 2004-2011 74
Figure 35: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Indication, (%), 2004-2011 75
Figure 36: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Geography, (%), 2004-2011 76
Figure 37: Orphan Autoimmune Disorder Therapeutics Market, Global, Deals by Value (%), 2004-2011 77
Figure 38: GBI Research Market Forecasting Model 86

Companies Mentioned

Amgen Inc.
GlaxoSmithKline
CSL Behring LLC
Mitsubishi Tanabe Pharma Corporation
Biogen Idec