デュシェンヌ型筋ジストロフィー治療薬の世界市場機会分析...市場調査レポートについてご紹介

1 Table of Contents
1 Table of Contents 8
1.1 List of Tables 12
1.2 List of Figures 15
2 Introduction 16
2.1 Catalyst 16
2.2 Related Reports 17
3 Disease Overview 18
3.1 Etiology and Pathophysiology 18
3.1.1 Etiology 18
3.1.2 Pathophysiology 19
3.2 Prognosis and Symptoms 20
3.3 Diagnosis 21
4 Epidemiology 23
4.1 Disease Background 23
4.2 Risk Factors and Comorbidities 24
4.3 Global Trends 25
4.4 Forecast Methodology 27
4.4.1 Sources Used 33
4.4.2 Sources Not Used 34
4.4.3 Forecast Assumptions and Methods 35
4.5 Epidemiological Forecast for DMD (2013-2023) 40
4.5.1 Diagnosed Prevalent Cases of DMD (All Ages) 40
4.5.2 Diagnosed Prevalent Cases of DMD (Ages 5-13 Years) 42
4.5.3 Age-Specific Diagnosed Prevalent Cases of DMD (All Ages) 44
4.5.4 Age-Standardized Diagnosed Prevalence of DMD 45
4.6 Epidemiological Forecast for Nonsense Mutations in DMD (2013-2023) 46
4.7 Epidemiological Forecast for the Applicability of Exon-Skipping Therapies in DMD (2013-2023) 48
4.7.1 Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy (All Ages) 48
4.7.2 Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy (All Ages) 49
4.7.3 Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy (All Ages) 51
4.7.4 Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy (All Ages) 52
4.8 Epidemiological Forecast for BMD (2013-2023) 54
4.8.1 Diagnosed Prevalent Cases of BMD (All Ages) 54
4.8.2 Age-Specific Diagnosed Prevalent Cases of BMD (All Ages) 55
4.8.3 Age-Standardized Diagnosed Prevalence of BMD 57
4.9 Discussion 58
4.9.1 Epidemiological Forecast Insight 58
4.9.2 Limitations of the Analysis 59
4.9.3 Strengths of the Analysis 60
5 Current Treatment Options 61
5.1 Overview 61
5.1.1 Duchenne Muscular Dystrophy 61
5.1.2 Becker Muscular Dystrophy 62
5.2 Product Profiles – Major Brands 63
5.2.1 Corticosteroids (Prednisone and Deflazacort – Numerous Generic and Brand Names) 63
5.2.2 Translarna (ataluren) 66
6 Unmet Needs Assessment and Oppportunity Analysis 72
6.1 Overview 72
6.2 Need for Disease-Modifying Therapies 73
6.2.1 Unmet Need 73
6.2.2 Gap Analysis 74
6.2.3 Opportunity 74
6.3 Delayed Diagnosis 75
6.3.1 Unmet Need 75
6.3.2 Gap Analysis 76
6.3.3 Opportunity 76
6.4 High Variation in Design of DMD Clinical Trials 77
6.4.1 Unmet Need 77
6.4.2 Gap Analysis 78
6.4.3 Opportunity 78
6.5 Need for Regulatory Pathway for Approval of Follow-On Exon-Skipping Molecules 79
6.5.1 Unmet Need 79
6.5.2 Gap Analysis 79
6.5.3 Opportunity 80
7 R&D Strategies 81
7.1 Overview 81
7.1.1 Follow-On and Multi Exon-Skipping Molecules 81
7.1.2 Forming Alliances with Patient Advocacy Groups 82
7.1.3 Partnering with Pharmaceutical Companies to Increase Marketing Resources 83
7.2 Current Clinical Trial Design 84
7.2.1 Current Methods for Assessing Clinical Efficacy and Their Limitations 85
7.2.2 Current Trial Design of Key Pipeline Products 87
7.2.3 Patient Exclusion Issues in Current Trial Designs 88
7.3 Future Clinical Trial Design 89
7.3.1 Dystrophin-Positive Biomarkers as Surrogate Clinical Endpoints 89
7.3.2 Need for Measuring Clinical Outcome and Endpoints Based on Disease Onset 90
7.3.3 Maximizing Inclusion of Patient Populations in Studies 91
8 Pipeline Assessment 92
8.1 Overview 92
8.2 Promising Drugs in Clinical Development 93
8.2.1 Drisapersen 94
8.2.2 Eteplirsen 97
8.2.3 Catena/Raxone (idebenone) 101
8.2.4 Tadalafil 105
8.3 Innovative Early-Stage Approaches 108
8.3.1 Follow-On Exon-Skipping Therapies 110
8.3.2 Utrophin Activation 111
8.3.3 Myostatin Inhibition 112
8.3.4 Gene Therapy 112
8.3.5 Targeting Cellular Pathways and Other Early-Stage Approaches 113
9 Pipeline Valuation Analysis 115
9.1 Clinical Benchmark of Key Pipeline Drugs 115
9.2 Commercial Benchmark of Key Pipeline Drugs 117
9.3 Competitive Assessment 119
9.4 Top-Line Five-Year Forecast 122
9.4.1 US 126
9.4.2 5EU 127
10 Appendix 128
10.1 Bibliography 128
10.2 Abbreviations 137
10.3 Methodology 140
10.4 Forecasting Methodology 141
10.4.1 Diagnosed DMD Patients 141
10.4.2 Percent Drug-Treated Patients 142
10.4.3 Drugs Included in Each Therapeutic Class 142
10.4.4 Launch and Patent Expiry Dates 143
10.4.5 General Pricing Assumptions 143
10.4.6 Individual Drug Assumptions 144
10.4.7 Generic Erosion 148
10.4.8 Pricing of Pipeline Agents 148
10.5 Physicians and Specialists Included in this Study 150
10.6 About the Authors 151
10.6.1 Author 151
10.6.2 Reviewers 151
10.6.3 Epidemiologist 152
10.6.4 Global Head of Healthcare 152
10.7 About GlobalData 153
10.8 Disclaimer 153