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遺伝病の希少疾患治療(〜2018年):嚢胞性線維症(CF)の新物質がもたらす強い投資の機会およびライセンシング活動

Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity

発行 GBI Research 商品コード 240081
出版日 ページ情報 英文 138 Pages
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遺伝病の希少疾患治療(〜2018年):嚢胞性線維症(CF)の新物質がもたらす強い投資の機会およびライセンシング活動 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity
出版日: 2012年05月15日 ページ情報: 英文 138 Pages
概要

世界における遺伝病の希少疾患治療市場額は、2004年の5億5900万米ドルからCAGR17.3%で拡大し、2010年に15億米ドルとなりました。同市場額は2010年〜2018年の期間中、CAGR13.8%で拡大し、2018年までに41億米ドルへ拡大すると見られています。

当レポートでは、世界および主要地域における遺伝病の希少疾患治療市場について調査し、2002年〜2010年の年間市場データ実績と2018年までの予測を提供しており、治療情勢に関する市場データ、主な市場成長促進因子と阻害因子、R&Dパイプライン分析、競合情勢、、市場機会の分析、主要企業のプロファイルを盛り込んで、概略下記の構成でお届けいたします。

第1章 目次

第2章 エグゼクティブサマリー

第3章 イントロダクション

第4章 市場概要

  • イントロダクション
  • 希少疾患治療市場の収益予測
  • 世界の治療利用パターン
  • 希少疾患治療市場の成長促進因子・阻害因子

第5章 地域別情勢

  • 米国
  • 欧州の主要5ヶ国
  • 日本

第6章 治療情勢

  • 嚢胞性線維症(CF)
  • デュシェンヌ型筋ジストロフィー(DMD)
  • ファブリー病
  • ポンペ病

第7章 パイプライン分析

  • イントロダクション
  • R&Dパイプライン:嚢胞性線維症(CF)
  • R&Dパイプライン:デュシェンヌ型筋ジストロフィー(DMD)
  • R&Dパイプライン:ファブリー病
  • R&Dパイプライン:ポンペ病

第8章 競合情勢

  • 競合プロファイル

第9章 戦略的整理統合

  • M&A情勢
  • 主なライセンシング合意
  • 共同開発合意

第10章 付録

第11章 付録

図表

目次

Summary

GBI Research, the leading business intelligence provider, has released its latest research, "Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity", which provides insights into the global orphan diseases in genetic disorders therapeutics market, including market forecasts until 2018. The report provides an in-depth analysis of major orphan genetic disease indications, covering Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Fabry disease and Pompe disease. The report also includes insights into the orphan genetic disease therapeutics R&D pipeline. It analyzes the competitive landscape, including M&As, and licensing and co-development 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.

GBI Research found that the global orphan disease therapeutics in genetic disorders market was worth $1.5 billion in 2010, having grown from $559m in 2004 at a Compound Annual Growth Rate (CAGR) of 17.3%. It is expected to increase to $4.1 billion by 2018, growing at a CAGR of 13.8% over the period from 2010. This high rate of growth in the historic period can be attributed to the entry of novel, highly priced therapies into markets with high unmet need. These include Myozyme for Pompe disease and enzyme replacement therapies for Fabry disease, which have high costs of over $200,000 per patient per year. More highly-priced therapies are expected in the forecast period, to further drive growth. The recent approval of Kalydeco in CF and the expected approval of exon-skipping therapies for DMD are examples of this. Competitive activity is also increasing as larger companies realize the rewards of investing in orphan therapeutics, and the high unmet need and low competition in many markets is expected to attract more into the space in the future.

Scope

  • Data and analysis on the global orphan disease therapeutics in genetic disorders in the leading geographies of the world- the US, the UK, Germany, France, Italy, Spain, and Japan.
  • Annualized market data for the orphan disease therapeutics in genetic disorders market from 2002-2010 with forecasts to 2018.
  • Market data on the therapeutic landscape, covering CF, DMD, Fabry disease and Pompe disease. This includes market size, market share, annual cost of therapy, treatment flow algorithm and branded and generic share.
  • Key drivers and restraints that have had a significant impact on the market and on each indication.
  • The competitive landscape of the global orphan disease therapeutics in genetic disorders, including companies such as Genzyme, GlaxoSmithKline, Novartis, Santhera, and Shire.
  • Key M&A activities and licensing and co-development agreements that took place from 2006-2011 in the global orphan disease therapeutics in genetic disorders 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 potential region and global orphan disease therapeutics in genetic disorders market segments poised for strong growth.
  • Create a more tailored country strategy through the understanding of key drivers and barriers of the global oncology therapeutics market.
  • Develop key strategic initiatives by understanding the key focus areas and top selling therapeutics of leading companies.
  • Accelerate and strengthen your market position by identifying key companies for mergers, acquisitions and strategic partnerships.

Table of Contents

1 Table of Contents 5

  • 1.1 List of Tables 8
  • 1.2 List of Figures 11

2 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Introduction 13

3 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Market Overview 14

  • 3.1 Introduction 14
    • 3.1.1 Orphan Disease Regulation and Incentives 14
  • 3.2 Revenue Forecasts for Orphan Disease Therapeutics in Genetic Disorders 16
    • 3.2.1 Revenue 16
    • 3.2.2 Annual Cost of Treatment 18
  • 3.3 Global Treatment Usage Patterns 19
    • 3.3.1 Diseased Population 19
    • 3.3.2 Treatment Seeking Population 20
    • 3.3.3 Diagnosed Population 21
    • 3.3.4 Prescription Population 22
  • 3.4 Drivers and Restraints for Orphan Genetic Disease Therapeutics Market 23
    • 3.4.1 Drivers for Orphan Genetic Disease Therapeutics Market 23
    • 3.4.2 Restraints for Orphan Genetic Disease Therapeutics Market 25
    • 3.4.3 Unmet Needs 25
    • 3.4.4 Opportunities 25

4 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Geographical Landscape 26

  • 4.1 The US 26
    • 4.1.1 Revenue 26
    • 4.1.2 Annual Cost of Treatment 27
    • 4.1.3 Treatment Usage Patterns 28
  • 4.2 Top Five Countries of Europe 32
    • 4.2.1 Revenue 32
    • 4.2.2 Annual Cost of Treatment 33
    • 4.2.3 Treatment Usage Patterns 34
  • 4.3 Japan 38
    • 4.3.1 Revenue 38
    • 4.3.2 Annual Cost of Treatment 39
    • 4.3.3 Treatment Usage Patterns 40

5 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Therapeutic Landscape 44

  • 5.1 Cystic Fibrosis 44
    • 5.1.1 Introduction 44
    • 5.1.2 Symptoms 45
    • 5.1.3 Diagnosis 45
    • 5.1.4 Treatment Flow 46
    • 5.1.5 Revenue 47
    • 5.1.6 Annual Cost of Treatment 49
    • 5.1.7 Treatment Usage Patterns 50
    • 5.1.8 Marketed Products 54
    • 5.1.9 Drivers and Restraints for the Cystic Fibrosis Therapeutics Market 58
  • 5.2 Duchenne Muscular Dystrophy 61
    • 5.2.1 Introduction 61
    • 5.2.2 Symptoms 61
    • 5.2.3 Treatment Algorithm 62
    • 5.2.4 Revenue 63
    • 5.2.5 Annual Cost of Treatment 66
    • 5.2.6 Treatment Usage Patterns 67
    • 5.2.7 Marketed Therapies 71
    • 5.2.8 Drivers and Restraints for Duchenne Muscular Dystrophy Therapeutics Market 71
  • 5.3 Fabry Disease 73
    • 5.3.1 Introduction 73
    • 5.3.2 Symptoms 73
    • 5.3.3 Treatment Flow 74
    • 5.3.4 Revenue 74
    • 5.3.5 Annual Cost of Treatment 76
    • 5.3.6 Treatment Usage Patterns 78
    • 5.3.7 Drivers and Restarints for Fabry Disease Therapeutics Market 82
    • 5.3.8 Restraints for Fabry Disease Therapeutics Market 83
    • 5.3.9 Marketed Products 83
  • 5.4 Pompe Disease 85
    • 5.4.1 Introduction 85
    • 5.4.2 Symptoms 86
    • 5.4.3 Treatment Flow Algorithm 86
    • 5.4.4 Revenue 87
    • 5.4.5 Annual Cost of Treatment 88
    • 5.4.6 Treatment Usage Patterns 89
    • 5.4.7 Marketed Products 94

6 Orphan Disease Therapeutics in Genetic Disorders to 2018 -Pipeline Analysis 96

  • 6.1 Introduction 96
  • 6.2 R&D Pipeline - Cystic Fibrosis 97
    • 6.2.1 Pre-Clinical Stage 97
    • 6.2.2 Phase I 98
    • 6.2.3 Phase II 99
    • 6.2.4 Phase III 102
    • 6.2.5 Promising Drugs in the Pipeline 102
  • 6.3 R&D Pipeline - Duchenne Muscular Dystrophy 104
    • 6.3.1 Pre-Clinical 104
    • 6.3.2 Phase I 105
    • 6.3.3 Phase II 106
    • 6.3.4 Phase III 106
    • 6.3.5 Promising Molecules in the Pipeline 107
  • 6.4 R&D Pipeline - Fabry Disease 108
    • 6.4.1 Pre-Clinical 108
    • 6.4.2 Phase II 109
    • 6.4.3 Phase III 109
  • 6.5 R&D Pipeline - Pompe Disease 110
    • 6.5.1 Pre-Clinical 110
    • 6.5.2 Phase II 110

7 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Competitive Landscape 111

  • 7.1 Competitive Profiling 111
    • 7.1.1 GlaxoSmithKline 111
    • 7.1.2 Prosensa 112
    • 7.1.3 Santhera 112
    • 7.1.4 AVI BioPharma 113
    • 7.1.5 Genzyme (Sanofi Subsidiary) 114
    • 7.1.6 Shire 114
    • 7.1.7 PTC Therapeutics 115
    • 7.1.8 Novartis 116
    • 7.1.9 Genentech (Roche) 117
    • 7.1.10 Gilead 117

8 Orphan Disease Therapeutics in Genetic Disorders to 2018 - Strategic Consolidations 119

  • 8.1 M&A Landscape 119
    • 8.1.1 Key Deals 119
    • 8.1.2 M&A Deals by Year 121
    • 8.1.3 M&A Deals by Geography 121
    • 8.1.4 M&A Deals by Value 122
  • 8.2 R&D Licensing Agreements 122
    • 8.2.1 Key Deals 122
    • 8.2.2 Deals by Year 126
    • 8.2.3 Deals by Geography 127
  • 8.3 Co-Development Agreements 127
    • 8.3.2 Deals by Year 132
    • 8.3.3 Deals by Geography 132

9 Appendix 133

  • 9.1 Market Definitions 133
  • 9.2 Abbreviations 133
  • 9.3 Sources 134
  • 9.4 Research Methodology 135
    • 9.4.1 Coverage 135
    • 9.4.2 Secondary Research 135
    • 9.4.3 Primary Research 136
    • 9.4.4 Forecasting 136
    • 9.4.5 Geographical Landscape 137
    • 9.4.6 Pipeline Analysis 137
    • 9.4.7 Competitive Landscape 137
    • 9.4.8 Expert Panel Validation 138
  • 9.5 Contact Us 138
  • 9.6 Disclaimer 138

List of Tables

  • Table 1: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue ($m), 2004-2010 16
  • Table 2: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts ($m), 2010-2018 16
  • Table 3: Orphan Disease Therapeutics in Genetic Disorders, Global, Average ACT ($), 2004-2010 18
  • Table 4: Orphan Disease Therapeutics in Genetic Disorders, Global, Average ACT ($), 2010-2018 18
  • Table 5: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2004-2010 19
  • Table 6: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2010-2018 19
  • Table 7: Orphan Disease Therapeutics in Genetic Disorders, Global, Treatment Seeking Population, 2004-2010 20
  • Table 8: Orphan Disease Therapeutics in Genetic Disorders, Global, Treatment Seeking Population, 2010-2018 20
  • Table 9: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2004-2010 21
  • Table 10: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2010-2018 21
  • Table 11: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2004-2010 22
  • Table 12: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2010-2018 22
  • Table 13: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue ($m), 2004-2010 26
  • Table 14: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue Forecasts ($m), 2010-2018 26
  • Table 15: Orphan Disease Therapeutics in Genetic Disorders, The US, Average ACT ($), 2004-2010 27
  • Table 16: Orphan Disease Therapeutics in Genetic Disorders, The US, Average ACT ($), 2010-2018 27
  • Table 17: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2004-2010 28
  • Table 18: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2010-2018 28
  • Table 19: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2004-2010 29
  • Table 20: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2010-2018 29
  • Table 21: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2004-2010 30
  • Table 22: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2010-2018 30
  • Table 23: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2004-2010 31
  • Table 24: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2010-2018 31
  • Table 25: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue ($m), 2004-2010 32
  • Table 26: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue Forecasts ($m), 2010-2018 32
  • Table 27: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Average ACT ($), 2004-2010 33
  • Table 28: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Average ACT ($), 2010-2018 33
  • Table 29: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe Diseased Population, 2004-2010 34
  • Table 30: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diseased Population, 2010-2018 34
  • Table 31: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2004-2010 35
  • Table 32: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2010-2018 35
  • Table 33: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2004-2010 36
  • Table 34: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2010-2018 36
  • Table 35: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2004-2010 37
  • Table 36: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2010-2018 37
  • Table 37: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue ($m), 2004-2010 38
  • Table 38: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue Forecasts ($m), 2010-2018 38
  • Table 39: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2004-2010 39
  • Table 40: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2010-2018 39
  • Table 41: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2004-2010 40
  • Table 42: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2010-2018 40
  • Table 43: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2004-2010 41
  • Table 44: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2010-2018 41
  • Table 45: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2004-2010 42
  • Table 46: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2010-2018 42
  • Table 47: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2004-2010 43
  • Table 48: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2010-2018 43
  • Table 49: Cystic Fibrosis Therapeutics Market, Global, Revenue ($m), 2004-2010 47
  • Table 50: Cystic Fibrosis Therapeutics Market, Global, Revenue Forecasts ($m), 2010-2018 47
  • Table 51: Cystic Fibrosis Therapeutics Market, Global, Average ACT ($), 2004-2010 49
  • Table 52: Cystic Fibrosis Therapeutics Market, Global, Average ACT ($), 2010-2018 49
  • Table 53: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2004-2010 50
  • Table 54: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2010-2018 50
  • Table 55: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population , 2004-2010 51
  • Table 56: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population, 2010-2018 51
  • Table 57: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2004-2010 52
  • Table 58: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2010-2018 52
  • Table 59: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2004-2010 53
  • Table 60: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2010-2018 53
  • Table 61: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenue ($m), 2004-2010 63
  • Table 62: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenues Forecasts ($m), 2010-2018 63
  • Table 63: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2004-2010 66
  • Table 64: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2010-2018 66
  • Table 65: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2004-2010 67
  • Table 66: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2010-2018 67
  • Table 67: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2004-2010 68
  • Table 68: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2010-2018 68
  • Table 69: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diagnosed Population, 2004-2010 69
  • Table 70: Duchenne Muscular Dystrophy Therapeutics Market, Japan, Diagnosed Population, 2010-2018 69
  • Table 71: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2004-2010 70
  • Table 72: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2010-2018 70
  • Table 73: Fabry Disease Therapeutics Market, Global, Revenue ($m), 2004-2010 74
  • Table 74: Fabry Disease Therapeutics Market, Global, Revenue Forecasts ($m), 2010-2018 74
  • Table 75: Fabry Disease Therapeutics Market, Global, Average ACT ($), 2004-2010 76
  • Table 76: Fabry Disease Therapeutics Market, Global, Average ACT ($), 2010-2018 76
  • Table 77: Fabry Disease Therapeutics Market, Global, Diseased Population, 2004-2010 78
  • Table 78: Fabry Disease Therapeutics Market, Global, Diseased Population, 2010-2018 78
  • Table 79: Fabry Disease Therapeutics Market, Global, Treatment Seeking Population, 2004-2010 79
  • Table 80: Fabry Disease Therapeutics Market, Global,Treatment Seeking Population, 2010-2018 79
  • Table 81: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2004-2010 80
  • Table 82: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2010-2018 80
  • Table 83: Fabry Disease Therapeutics Market, Global, Prescription Population, 2004-2010 81
  • Table 84: Fabry Disease Therapeutics Market, Global, Prescription Population, 2010-2018 81
  • Table 85: Pompe Disease Therapeutics Market, Global, Revenue ($m), 2004-2010 87
  • Table 86: Pompe Disease Therapeutics Market, Global, Revenues Forecasts ($m), 2010-2018 87
  • Table 87: Pompe Disease Therapeutics Market, Global, Average ACT ($), 2004-2010 88
  • Table 88: Pompe Disease Therapeutics Market, Global, Average ACT ($), 2010-2018 88
  • Table 89: Pompe Disease Therapeutics Market, Global, Diseased Population, 2004-2010 89
  • Table 90: Pompe Disease Therapeutics Market, Global, Diseased Population, 2010-2018 89
  • Table 91: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2004-2010 90
  • Table 92: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2010-2018 90
  • Table 93: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2004-2010 91
  • Table 94: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2010-2018 91
  • Table 95: Pompe Disease Therapeutics Market, Global, Prescription Population, 2004-2010 92
  • Table 96: Pompe Disease Therapeutics Market, Global, Prescription Population, 2010-2018 92
  • Table 97: Cystic Fibrosis Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 97
  • Table 98: Cystic Fibrosis Therapeutics Market, Global, Phase I Pipeline, 2011 98
  • Table 99: Cystic Fibrosis Therapeutics Market, Global, Phase II Pipeline, 2011 99
  • Table 100: Cystic Fibrosis Therapeutics Market, Global, Phase III Pipeline, 2011 102
  • Table 101: Duchenne Muscular Dystrophy Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 104
  • Table 102: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase I Pipeline, 2011 105
  • Table 103: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase II Pipeline, 2011 106
  • Table 104: Duchenne Muscular Dystrophy Therapeutics Market, Global, Phase II Pipeline, 2011 106
  • Table 105: Fabry Disease Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 108
  • Table 106: Fabry Disease Therapeutics Market, Global, Phase II Pipeline, 2011 109
  • Table 107: Fabry Disease Therapeutics Market, Global, Phase III Pipeline, 2011 109
  • Table 108: Pompe Disease Therapeutics Market, Global, Pre-Clinical Pipeline, 2011 110
  • Table 109: Pompe Disease Therapeutics Market, Global, Phase II Pipeline, 2011 110

List of Figures

  • Figure 1: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts ($m), 2004-2018 16
  • Figure 2: Orphan Disease Therapeutics in Genetic Disorders, Global, Revenue Forecasts by Region ($m), 2004-2018 17
  • Figure 3: Orphan Disease Therapeutics in Genetic Disorders, Global, ACT ($), 2004-2018 18
  • Figure 4: Orphan Disease Therapeutics in Genetic Disorders, Global, Diseased Population, 2004-2018 19
  • Figure 5: Orphan Disease Therapeutics i`n Genetic Disorders Market, Global, Treatment Seeking Population, 2004-2018 20
  • Figure 6: Orphan Disease Therapeutics in Genetic Disorders, Global, Diagnosed Population, 2004-2018 21
  • Figure 7: Orphan Disease Therapeutics in Genetic Disorders, Global, Prescription Population, 2004-2018 22
  • Figure 8: Orphan Disease Therapeutics in Genetic Disorders, Global, Drivers and Restraints 23
  • Figure 9: Orphan Disease Therapeutics in Genetic Disorders, The US, Revenue ($m), 2004-2018 26
  • Figure 10: Orphan Disease Therapeutics in Genetic Disorders, The US, ACT ($), 2004-2018 27
  • Figure 11: Orphan Disease Therapeutics in Genetic Disorders, The US, Diseased Population, 2004-2018 28
  • Figure 12: Orphan Disease Therapeutics in Genetic Disorders, The US, Treatment Seeking Population, 2004-2018 29
  • Figure 13: Orphan Disease Therapeutics in Genetic Disorders, The US, Diagnosed Population, 2004-2018 30
  • Figure 14: Orphan Disease Therapeutics in Genetic Disorders, The US, Prescription Population, 2004-2018 31
  • Figure 15: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Revenue ($m), 2004-2018 32
  • Figure 16: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, ACT ($), 2004-2018 33
  • Figure 17: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diseased Population, 2004-2018 34
  • Figure 18: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Treatment Seeking Population, 2004-2018 35
  • Figure 19: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Diagnosed Population, 2004-2018 36
  • Figure 20: Orphan Disease Therapeutics in Genetic Disorders, Top Five Countries of Europe, Prescription Population, 2004-2018 37
  • Figure 21: Orphan Disease Therapeutics in Genetic Disorders, Japan, Revenue ($m), 2004-2018 38
  • Figure 22: Orphan Disease Therapeutics in Genetic Disorders, Japan, ACT ($), 2004-2018 39
  • Figure 23: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diseased Population, 2004-2018 40
  • Figure 24: Orphan Disease Therapeutics in Genetic Disorders, Japan, Treatment Seeking Population, 2004-2018 41
  • Figure 25: Orphan Disease Therapeutics in Genetic Disorders, Japan, Diagnosed Population, 2004-2018 42
  • Figure 26: Orphan Disease Therapeutics in Genetic Disorders, Japan, Prescription Population, 2004-2018 43
  • Figure 27: Cystic Fibrosis Therapeutics Market, Global, Treatment Flow Algorithm, 2011 46
  • Figure 28: Cystic Fibrosis Therapeutics Market, Global, Revenue ($m), 2004-2018 47
  • Figure 29: Cystic Fibrosis Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2018 48
  • Figure 30: Cystic Fibrosis Therapeutics Market, Global, ACT ($), 2004-2018 49
  • Figure 31: Cystic Fibrosis Therapeutics Market, Global, Diseased Population, 2004-2018 50
  • Figure 32: Cystic Fibrosis Therapeutics Market, Global, Treatment Seeking Population, 2004-2018 51
  • Figure 33: Cystic Fibrosis Therapeutics Market, Global, Diagnosed Population, 2004-2018 52
  • Figure 34: Cystic Fibrosis Therapeutics Market, Global, Prescription Population, 2004-2018 53
  • Figure 35: Cystic Fibrosis Therapeutics Market, Global, Drivers and Restraints 58
  • Figure 36: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Flow Algorithm 62
  • Figure 37: Duchenne Muscular Dystrophy Therapeutics Market, Global, Revenues ($m), 2004-2018 63
  • Figure 38: Duchenne Muscular Dystrophy Therapeutics Market, Global, Branded and Generic Market Share (%), 2010 and 2018 64
  • Figure 39: Duchenne Muscular Dystrophy Therapeutics Market, Global, ACT ($), 2004-2018 66
  • Figure 40: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diseased Population, 2004-2018 67
  • Figure 41: Duchenne Muscular Dystrophy Therapeutics Market, Global, Treatment Seeking Population, 2004-2018 68
  • Figure 42: Duchenne Muscular Dystrophy Therapeutics Market, Global, Diagnosed Population, 2004-2018 69
  • Figure 43: Duchenne Muscular Dystrophy Therapeutics Market, Global, Prescription Population, 2004-2018 70
  • Figure 44: Duchenne Muscular Dystrophy Therapeutics Market, Global, Drivers and Restraints 71
  • Figure 45: Fabry Disease Therapeutics Market, Global, Treatment Flow Algorithm, 2011 74
  • Figure 46: Fabry Disease Therapeutics Market, Global, Revenue ($m), 2004-2018 74
  • Figure 47: Fabry Disease Therapeutics Market, Global, ACT ($), 2004-2018 76
  • Figure 48: Fabry Disease Therapeutics Market, Global, Diseased Population, 2004-2018 78
  • Figure 49: Fabry Disease Therapeutics Market, Global, Treatment Seeking Population, 2004-2018 79
  • Figure 50: Fabry Disease Therapeutics Market, Global, Diagnosed Population, 2004-2018 80
  • Figure 51: Fabry Disease Therapeutics Market, Global, Prescription Population, 2004-2018 81
  • Figure 52: Fabry Disease Therapeutics Market, Global, Drivers and Restraints 82
  • Figure 53: Pompe Disease Therapeutics Market, Global, Treatment Flow Algorithm 86
  • Figure 54: Pompe Disease Therapeutics Market, Global, Revenue ($m), 2004-2018 87
  • Figure 55: Pompe Disease Therapeutics Market, Global, ACT ($), 2004-2018 88
  • Figure 56: Pompe Disease Therapeutics Market, Global, Diseased Population, 2004-2018 89
  • Figure 57: Pompe Disease Therapeutics Market, Global, Treatment Seeking Population, 2004-2018 90
  • Figure 58: Pompe Disease Therapeutics Market, Global, Diagnosed Population, 2004-2018 91
  • Figure 59: Pompe Disease Therapeutics Market, Global, Prescription Population, 2004-2018 92
  • Figure 60: Pompe Disease Therapeutics Market, Global, Drivers and Restraints 93
  • Figure 61: Orphan Disease Therapeutics in Genetic Disorders, GSK, SWOT, 2011 111
  • Figure 62: Orphan Disease Therapeutics in Genetic Disorders, Prosensa, SWOT, 2011 112
  • Figure 63: Orphan Disease Therapeutics in Genetic Disorders, Santhera, SWOT, 2011 113
  • Figure 64: Orphan Disease Therapeutics in Genetic Disorders, Avi BioPharma, SWOT, 2011 113
  • Figure 65: Orphan Disease Therapeutics in Genetic Disorders, Genzyme (Sanofi Subsidiary), SWOT, 2011 114
  • Figure 66: Orphan Disease Therapeutics in Genetic Disorders, Shire, SWOT, 2011 115
  • Figure 67: Orphan Disease Therapeutics in Genetic Disorders, PTC Therapeutics, SWOT, 2011 115
  • Figure 68: Orphan Disease Therapeutics in Genetic Disorders, Novartis, SWOT, 2011 116
  • Figure 69: Orphan Disease Therapeutics in Genetic Disorders, Genentech (Roche), SWOT, 2011 117
  • Figure 70: Orphan Disease Therapeutics in Genetic Disorders, Gilead, SWOT, 2011 118
  • Figure 71: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Year, 2006-2011 121
  • Figure 72: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Geography (%), 2006-2011 121
  • Figure 73: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of M&A Deals by Value (%), 2006-2011 122
  • Figure 74: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Licensing Deals by Year, 2007-2011 126
  • Figure 75: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Licensing Deals by Geography (%), 2006-2011 127
  • Figure 76: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Co-Development Deals by Year, 2006-2011 132
  • Figure 77: Orphan Disease Therapeutics in Genetic Disorders, Global, Number of Co-Development Deals by Geography (%), 2006-2011 132
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