Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington's Disease Segment

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Market Research Report Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington's Disease Segment
Published by	GBI Research
Published	April 2, 2012	Product code	235831
Content info	Pages: 124
Price	USD 3500 PDF by E-mail (Single user license) USD 7000 PDF by E-mail (Site license) USD 10500 PDF by E-mail (Global license)

Introduction

Introduction

Abstract

Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington's Disease Segment

Summary

GBI Research, the leading business intelligence provider, has released its latest research "Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington's Disease Segment", which provides insights into the CNS orphan disease therapeutics market until 2017. 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 report provides an in-depth analysis of amyotrophic lateral sclerosis, huntington's disease and myasthenia gravis. It also provides the share of generics in global CNS orphan disease therapeutics and in each indication market. The report examines the global orphan diseases therapeutics in CNS usage patterns. It includes the geographical distribution of amyotrophic lateral sclerosis, huntington's disease and myasthenia gravis markets across the US, the top five countries of Europe and in Japan. The report also includes insights into the orphan diseases therapeutics in CNS Research and Development (R&D) product pipeline and explores the competitive landscape, including major players in the CNS orphan disease therapeutics market. Finally, the report includes analysis on Mergers and Acquisitions (M&A) and licensing agreements that took place in the orphan diseases therapeutics in CNS market.

GBI Research's analysis shows that the overall global CNS orphan disease market is set to increase over the forecast period. It grew from $359.6m in 2002 to $665.5m in 2010 at a Compound Annual Growth Rate (CAGR) of 8%. This is projected to increase to a CAGR of 17.6% over the forecast period to $2.1 billion in 2017. The global ALS therapeutics market generated approximately $163.2m in 2010, growing from $109.7m in 2002 at a CAGR of 5.1%. By 2017, this is expected to have increased to $210.5m at a CAGR of 3.7%. ALS was one of the biggest orphan CNS disease markets globally in 2010. The global HD therapeutics market generated approximately $126.7m in 2010, growing from $34.3m in 2002 at a CAGR of 17.8%. By 2017, this is expected to have increased to $786.5m at a CAGR of 29.8%. Globally, the MG therapeutics market increased from $42.5m in 2002 to $57.7m in 2010 at a CAGR of 3.9%. Over the forecast period it is expected to grow to $88.6m in 2017 with a higher CAGR of 6.3%.

Scope

The report analyzes treatment usage patterns, market characterization, pipeline analysis, competitive landscape and key M&A trends in the global orphan diseases therapeutics in CNS market for three indications, amyotrophic lateral sclerosis, huntington's disease and myasthenia gravis.
Data and analysis on the orphan diseases therapeutics in CNS market in the leading geographies of the world - the US, the UK, Germany, France, Italy, Spain and Japan.
Annualized market data for the orphan diseases therapeutics in CNS market from 2004 to 2010, with forecasts to 2017.
Market data on the geographical landscape and therapeutic landscape, including market size, market share, annual cost of therapy, sales volume and treatment usage patterns such as disease population, treatment seeking population, diagnosis population and prescription population.
Share of the generics in global orphan diseases therapeutics in CNS market and for the market of each indication that is covered in the report.
Key drivers and restraints that have had a significant impact on the market.
The competitive landscape of the global market including top companies benchmarking. The key companies studied in this report are Biogen Idec, Avanir Pharmaceuticals, Neuraltus Pharmaceuticals, Cytokinetics, The Avicena Group, NeuroSearch, Trophos, Amarin Corporation, Siena Biotech, Sanofi.
Key M&A activities and licensing agreements that took place between 2004 and 2011 in the orphan diseases therapeutics in CNS market.

Reasons to buy

The report will assist business development and marketing executives to strategize their product launches, by allowing them to -

Align your product portfolio to the markets with high growth potential.
Build effective strategies to launch their pipeline products by identifying potential geographies.
Exploit in-licensing and out-licensing opportunities by identifying products that might fill their portfolio gaps.
Develop key strategic initiatives by studying the key strategies of top competitors.
Create a more tailored country strategy through the understanding of key drivers and barriers and market potential of each indication.
Develop market entry and market expansion strategies by identifying the geographic markets poised for strong growth.
Reinforce R&D pipelines by identifying new target mechanisms which can produce first-in-class molecules which are safer and more efficacious.

1 Table of Contents

1 Table of Contents 3

1.1 List of Tables 5
1.2 List of Figures 7

2 Orphan Diseases Therapeutics in CNS - Introduction 9

2.1 GBI Research Report Guidance 10

3 Orphan Diseases Therapeutics in CNS - Market Overview 11

3.1 Introduction 11
3.2 Orphan Disease Regulation and Incentives 11
3.3 Global Revenue Forecasts for Orphan Diseases Therapeutics in CNS 13

3.3.1 Global Revenue and Forecast 13
3.3.2 Geographical Segmentation 15
3.3.3 Branded and Generic Market Share 17
3.3.4 Revenue Share by Indication 18
3.3.5 Market Drivers 20
3.3.6 Market Restraints 21
3.3.7 Unmet Needs 21
3.3.8 Opportunities 22

4 Orphan Diseases Therapeutics in CNS - Geographical Landscape 23

4.1 The US 23

4.1.1 Revenue 23

4.2 Top Five Countries of Europe 25

4.2.1 Revenue 25

4.3 Japan 26

4.3.1 Revenue 26

5 Orphan Diseases in CNS Disorders - Therapeutic Landscape 27

5.1 Amyotrophic Lateral Sclerosis 27

5.1.1 Disease Overview 27
5.1.2 Revenue 27
5.1.3 Average Annual Cost of Treatment 31
5.1.4 Branded and Generic Market Share 33
5.1.5 Treatment Algorithm 34
5.1.6 Treatment Usage Patterns 34
5.1.7 Approved Molecules 41
5.1.8 Rilutek 41
5.1.9 Nuedexta (Dextromethorphan Hydrobromide and Quinidine Sulfate) 42

5.2 Huntington's Disease 43

5.2.1 Disease Overview 43
5.2.2 Total Revenue 44
5.2.3 Average Annual Cost of Treatment (ACT) 47
5.2.4 Branded and Generic Market Share 48
5.2.5 Treatment Algorithm 49
5.2.6 Treatment Usage Patterns 50

5.3 Approved Molecules 54

5.3.1 Xenazine (tetrabenazine) 54

5.4 Myasthenia Gravis 55

5.4.1 Disease Overview 55
5.4.2 Total Revenue 56
5.4.3 Branded and Generic Market Share 59
5.4.4 Average Annual Cost of Treatment 60
5.4.5 Treatment Algorithm 61
5.4.6 Treatment Usage Patterns 62
5.4.7 Approved Molecules 66

5.5 Other Indications 67

5.5.1 Total Revenue 67
5.5.2 Product Profiles 68

6 Global Orphan Diseases in CNS Disorders - Pipeline Analysis 71

6.1 Introduction 71
6.2 Research and Development Pipeline 72

6.2.1 Overview 72
6.2.2 Pipeline by Clinical Phases of Development 75
6.2.3 Profiles of Promising Drugs in Orphan Diseases in CNS Disorders 82

7 Global Orphan Diseases in CNS Disorders - Competitive Landscape 85

7.1 Major Players 85
7.2 Major Company Profiles 85

7.2.1 Biogen Idec 85
7.2.2 Avanir 86
7.2.3 Neuraltus 87
7.2.4 Cytokinetics 88
7.2.5 Avicena 89
7.2.6 NeuroSearch 90
7.2.7 Trophos 91
7.2.8 Amarin 92
7.2.9 Siena Biotech 93
7.2.10 Sanofi 94

8 Global Orphan Diseases in CNS Disorders - Strategic Consolidations 95

8.1 Mergers and Acquisitions 95

8.1.1 Overview 95
8.1.2 Major Mergers and Acquisitions Deals 97
8.1.3 Segmentation by Indication 98
8.1.4 Segmentation by Geography 99
8.1.5 Segmentation by Deal Value 100
8.1.6 Segmentation by Year 101

8.2 Licensing Agreements 102

8.2.1 Overview 102
8.2.2 Major Licensing Deals 105
8.2.3 Segmentation by Indication 107
8.2.4 Segmentation by Geography 108
8.2.5 Segmentation by Deal Value 109
8.2.6 Segmentation by Year 110

8.3 Co Development Deals 111

8.3.1 Segmentation by Geography 113
8.3.2 Segmentation by Value 114
8.3.3 Segmentation by Year 115

9 CNS Orphan Diseases - Appendix 116

9.1 Market Definition 116
9.2 Abbreviations 116
9.3 Research Methodology 117

9.3.1 Coverage 118
9.3.2 Secondary Research 118
9.3.3 Primary Research 118

9.4 Forecasting 119

9.4.1 Epidemiology-based Forecasting 119
9.4.2 Expert Panels Validation 120

9.5 Contact Us 120
9.6 Disclaimer 120
9.7 Sources 120

List of Tables

1.1 List of Tables

Table 1: Orphan Diseases Therapeutics in CNS, Global, Revenue ($m), 2002-2010 13
Table 2: Orphan Diseases Therapeutics in CNS, Global, Revenue Forecasts ($m), 2010-2017 13
Table 3: Orphan Diseases Therapeutics in CNS, Global, Revenue Segmentation by Geography, ($m), 2002-2010 15
Table 4: Orphan Diseases Therapeutics in CNS, Global, Revenue Segmentation by Geography, ($m), 2010-2017 16
Table 5: Orphan Diseases Therapeutics in CNS, Global, Revenues by Indication ($m), 2002-2010 18
Table 6: Orphan Diseases Therapeutics in CNS, Global, Revenues by Indication ($m), 2010-2017 18
Table 7: Orphan Diseases Therapeutics in CNS, The US, Revenues, ($m), 2002-2010 23
Table 8: Orphan Diseases Therapeutics in CNS, The US, Revenue Forecasts, ($m), 2010-2017 23
Table 9: Orphan Diseases Therapeutics in CNS, Top Five Countries of Europe, Revenues ($m), 2010-2017 25
Table 10: Orphan Diseases Therapeutics in CNS, Top Five Countries of Europe, Revenue Forecasts ($m), 2010-2017 25
Table 11: Orphan Diseases Therapeutics in CNS, Japan, Revenues ($m), 2002-2010 26
Table 12: Orphan Diseases Therapeutics in CNS, Japan, Revenue Forecasts ($m), 2010-2017 26
Table 13: ALS Therapeutics, Global, Revenues ($m), 2002-2010 28
Table 14: ALS Therapeutics, Global, Revenue Forecasts ($m), 2010-2017 28
Table 15: ALS Therapeutics, Global, Revenue by Geographical Segmentation ($m), 2002-2010 29
Table 16: ALS Therapeutics, Global, Revenue by Geographical Segmentation ($m), 2010-2017 30
Table 17: ALS Therapeutics, Global, Annual Cost of Therapy ($), 2002-2010 31
Table 18: ALS Therapeutics, Global, Annual Cost of Therapy ($m), 2010-2017 31
Table 19: ALS Therapeutics Market, Global, Generic and Branded Revenues ($m), 2010 and 2017 33
Table 20: ALS Therapeutics Market, Global, Diseased Population ('000), 2002-2010 34
Table 21: ALS Therapeutics Market, Global, Diseased Population ('000), 2010-2017 35
Table 22: ALS Therapeutics Market, Global, Treatment Seeking Population ('000), 2002-2010 36
Table 23: ALS Therapeutics Market, Global, Treatment Seeking Population ('000), 2010-2017 36
Table 24: ALS Therapeutics, Global, Diagnosed Population ('000), 2002-2010 38
Table 25: ALS Therapeutics, Global, Diagnosed Population ('000), 2010-2017 38
Table 26: ALS Therapeutics, Global, Prescription Population ('000), 2002-2010 40
Table 27: ALS Therapeutics, Global, Prescription Population ('000), 2010-2017 40
Table 28: HD Therapeutics Market, Global, Revenues ($m), 2002-2010 44
Table 29: HD Therapeutics Market, Global, Revenue Forecasts ($m), 2010-2017 44
Table 30: HD Therapeutics, Global, Revenue Segmentation by Country ($m), 2002-2010 46
Table 31: HD Therapeutics, Global, Revenue Forecasts Segmentation by Country ,($m), 2010-2017 46
Table 32: HD Therapeutics, Global, Annual Cost of Therapy ($), 2002-2010 47
Table 33: HD Therapeutics, Global, Annual Cost of Therapy ($), 2010-2017 47
Table 34: HD Therapeutics Market, Global, Generic and Branded Revenues ($m), 2010 and 2017 48
Table 35: HD Therapeutics, Global, Diseased Population ('000), 2002-2010 50
Table 36: HD Therapeutics, Global, Diseased Population ('000), 2010-2017 50
Table 37: HD Therapeutics, Global, Treatment Seeking Population ('000), 2002-2010 51
Table 38: HD Therapeutics, Global, Treatment Seeking Population ('000), 2010-2017 51
Table 39: HD Therapeutics, Global, Diagnosed Population ('000), 2002-2010 52
Table 40: HD Therapeutics, Global, Diagnosed Population ('000), 2010-2017 52
Table 41: HD Therapeutics, Global, Prescription Population ('000), 2002-2010 53
Table 42: HD Therapeutics, Global, Prescription Population ('000), 2010-2017 53
Table 43: MG Therapeutics, Global, Revenues ($m), 2002-2010 56
Table 44: MG Therapeutics, Global, Revenue Forecasts ($m), 2010-2017 56
Table 45: MG Therapeutics, Global, Revenue Segmentation by Geography, ($m), 2002-2010 57
Table 46: MG Therapeutics, Global, Revenue Forecasts Segmentation by Geography, ($m), 2010-2017 58
Table 47: MG Therapeutics Market, Global, Branded and Generic Revenues ($m), 2010 and 2017 59
Table 48: MG Therapeutics, Global, Annual Cost of Therapy ($m), 2002-2010 60
Table 49: MG Therapeutics, Global, Annual Cost of Therapy ($m), 2010-2017 60
Table 50: MG Therapeutics, Global, Diseased Population ('000), 2002-2010 62
Table 51: MG Therapeutics, Global, Diseased Population ('000), 2010-2017 62
Table 52: MG Therapeutics, Global, Treatment Seeking Population ('000), 2002-2010 63
Table 53: MG Therapeutics, Global, Treatment Seeking Population ('000), 2010-2017 63
Table 54: MG Therapeutics, Global, Diagnosed Population ('000), 2002-2010 64
Table 55: MG Therapeutics, Global, Diagnosed Population ('000), 2010-2017 65
Table 56: MG Therapeutics, Global, Prescription Population ('000), 2002-2010 65
Table 57: MG Therapeutics, Global, Prescription Population ('000), ($m), 2010-2017 66
Table 58: Other CNS Orphan Disease Therapeutics, Global, Revenues ($m), 2002-2010 67
Table 59: Other CNS Orphan Disease Therapeutics, Global, Revenue Forecasts ($m), 2010-2017 67
Table 60: Orphan Diseases Therapeutics in CNS, Global, Discovery Pipeline Molecules, 2011 75
Table 61: Orphan Diseases Therapeutics in CNS, Global, Pre-clinical Pipeline Molecules, 2011 76
Table 62: Orphan Diseases Therapeutics in CNS, Global, IND Filed Molecules, 2011 78
Table 63: Orphan Diseases Therapeutics in CNS, Global, Phase I Pipeline Molecules, 2011 79
Table 64: Orphan Diseases Therapeutics in CNS, Global, Phase II Pipeline Molecules, 2011 80
Table 65: Orphan Diseases Therapeutics in CNS, Global, Phase III Pipeline Molecules, 2011 81
Table 66: Orphan Diseases Therapeutics in CNS, Global, M&A Deal, 2004-2011 96
Table 67: Orphan Diseases Therapeutics in CNS, Global, Licensing Agreements, by Year, 2004-2012 102
Table 68: Orphan Diseases Therapeutics in CNS, Global, Co-development Deals by Year, 2009-2011 111
Table 69: Orphan Diseases Therapeutics in CNS, Global, Co-development Deals by Year, 2009-2011 112

List of Figures

1.2 List of Figures

Figure 1: Orphan Diseases Therapeutics in CNS, Global, Revenue , ($m), 2002-2017 13
Figure 2: Orphan Diseases Therapeutics in CNS, Global, Revenue Segmentation by Geography, ($m), 2002-2017 15
Figure 3: Orphan Diseases Therapeutics in CNS, Global, Branded and Generic Market Share (%), 2010 17
Figure 4: Orphan Diseases Therapeutics in CNS, Global, Revenue by Indication, ($m) 2002-2017 18
Figure 5: Orphan Diseases Therapeutics in CNS Market, Drivers and Restraints 20
Figure 6: Orphan Diseases Therapeutics in CNS, The US, Revenue, ($m), 2002-2017 23
Figure 7: Orphan Diseases Therapeutics in CNS, Top Five Countries of Europe, Revenues ($m), 2002-2017 25
Figure 8: Orphan Diseases Therapeutics in CNS, Japan, Revenue ($m), 2002-2017 26
Figure 9: ALS Therapeutics Market, Global, Revenue ($m), 2002-2017 27
Figure 10: ALS Therapeutics, Global, Revenue by Geography ($m), 2002-2017 29
Figure 11: ALS Therapeutics, Global, Annual Cost of Treatment ($m), 2002-2017 31
Figure 12: ALS Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2017 33
Figure 13: ALS Therapeutics Market, Global, Diseased Population (Hundreds), 2002-2017 34
Figure 14: ALS Therapeutics, Global, Treatment Seeking Population (Hundreds), 2002-2017 36
Figure 15: ALS Therapeutics, Global, Diagnosed Population (Hundreds), 2002-2017 38
Figure 16: ALS Therapeutics, Global, Prescription Population (Hundreds), 2002-2017 40
Figure 17: HD Therapeutics Market, Global, Revenues ($m), 2002-2017 44
Figure 18: HD Therapeutics Market, Global, Revenues by Country ($m), 2002-2017 45
Figure 19: HD Therapeutics Market, Global, Annual Cost of Therapy ($), 2002-2017 47
Figure 20: HD Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2017 48
Figure 21: HD Therapeutics, Treatment Algorithm, 2010 49
Figure 22: HD Therapeutics, Global, Diseased Population ('000), 2002-2017 50
Figure 23: HD Therapeutics, Global, Treatment Seeking Population ('000), 2002-2017 51
Figure 24: HD Therapeutics, Global, Diagnosed Population ('000), 2002-2017 52
Figure 25: HD Therapeutics, Global, Prescription Population ('000), 2002-2017 53
Figure 26: MG Therapeutics, Global, Revenues ($m), 2002-2017 56
Figure 27: MG Therapeutics, Global, Revenues by Country ($m), 2002-2017 57
Figure 28: MG Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2017 59
Figure 29: MG Therapeutics, Global, Annual Cost of Therapy ($m), 2002-2017 60
Figure 30: MG Therapeutics, Treatment Algorithm, 2010 61
Figure 31: MG Therapeutics, Global, Diseased Population ('000), 2002-2017 62
Figure 32: MG Therapeutics, Global, Treatment Seeking Population ('000), 2002-2017 63
Figure 33: MG Therapeutics, Global, Diagnosed Population ('000), 2002-2017 64
Figure 34: MG Therapeutics, Global, Prescription Population ('000), 2002-2017 65
Figure 35: Other Orphan CNS Therapeutics, Global, Revenues ($m), 2002-2017 67
Figure 36: Orphan Diseases Therapeutics in CNS, Global, R&D Pipeline by Stage of Development (%), 2011 72
Figure 37: ALS Therapeutics Market, Global, R&D Pipeline by Stage of Development (%), 2011 72
Figure 38: HD Therapeutics Market, Global, R&D Pipeline by Stage of Development (%), 2011 73
Figure 39: MG Therapeutics Market, Global, R&D Pipeline by Clinical Stage of Development (%), 2011 73
Figure 40: Orphan Diseases Therapeutics in CNS, Global, SWOT -Biogen, 2011 85
Figure 41: Orphan Diseases Therapeutics in CNS, Global, SWOT -Avinar, 2011 86
Figure 42: Orphan Diseases Therapeutics in CNS, Global, SWOT -Neuraltus, 2011 87
Figure 43: Orphan Diseases Therapeutics in CNS, Global, SWOT -Cytokinetics, 2011 88
Figure 44: Orphan Diseases Therapeutics in CNS, Global, SWOT -Avicena, 2011 89
Figure 45: Orphan Diseases Therapeutics in CNS, Global, SWOT -NeuroSearch, 2011 90
Figure 46: Orphan Diseases Therapeutics in CNS, Global, SWOT -Trophos, 2011 91
Figure 47: Orphan Diseases Therapeutics in CNS, Global, SWOT -Amarin, 2011 92
Figure 48: Orphan Diseases Therapeutics in CNS, Global, SWOT -Siena Biotech, 2011 93
Figure 49: Orphan Diseases Therapeutics in CNS, Global, SWOT -Sanofi, 2011 94
Figure 50: Orphan Diseases Therapeutics in CNS, Global, M&As by Indication, 2004-2011 98
Figure 51: Orphan Diseases Therapeutics in CNS Global, M&As by Geography, 2004-2011 99
Figure 52: Orphan Diseases Therapeutics in CNS, Global, M&As by Value, 2004-2011 100
Figure 53: Orphan Diseases Therapeutics in CNS, Global, M&As by Year, 2004-2011 101
Figure 54: Orphan Diseases Therapeutics in CNS, Global, Licensing Deals by Indication, 2004-2011 107
Figure 55: Orphan Diseases Therapeutics in CNS, Global, Licensing Deals by Geography, 2004-2011 108
Figure 56: Orphan Diseases Therapeutics in CNS, Global, Licensing Deals by Value, 2005-2011 109
Figure 57: Orphan Diseases Therapeutics in CNS, Global, Licensing Deals by Year, 2004-2011 110
Figure 58: Orphan Diseases Therapeutics in CNS, Global, Co-Developments by Geography, 2004-2011 113
Figure 59: Orphan Diseases Therapeutics in CNS, Global, Co-Developments by Value, 2005-2011 114
Figure 60: Orphan Diseases Therapeutics in CNS, Global, Co-Developments by Year, 2005-2011 115

Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington's Disease Segment published by GBI Research in April 2, 2012. This report consists of Pages: 124 and the price starts from US $ 3500.