脆弱X症候群治療薬の世界市場機会分析・予測 2026年

◆英語タイトル:OpportunityAnalyzer: Fragile X Syndrome - Opportunity Analysis and Forecasts to 2026
◆商品コード:GDHC081POA
◆発行会社(リサーチ会社):GlobalData
◆発行日:2018年1月12日
◆ページ数:150
◆レポート言語:英語
◆レポート形式:PDF
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【レポートの概要】

OpportunityAnalyzer: Fragile X Syndrome – Opportunity Analysis and Forecasts to 2026
Summary

In this report, GlobalData analyzes the nuances of the Fragile X Syndrome (FXS) treatment landscape and provides detailed insights into the market dynamics of this highly competitive space. The analysis also includes the evaluation of the commercial and clinical profiles of pipeline drugs, and their sales forecasts for the Fragile X Syndrome indication over the 2016-2026 period.

Although a relatively rare disease, Fragile X Syndrome has a distinct underlying genetic cause. Early intervention is key to the prognosis of the patient, yet there are currently no approved therapies specifically for Fragile X Syndrome. Instead, symptoms are managed on a case-by-case basis. In addition, a high rate of late-stage pipeline drugs have failed in clinical trials, meaning there are currently only four drugs with the potential to be launched during the forecast period. However, these drugs have a high potential for fast uptake in the market, as they would be the only approved therapies for Fragile X syndrome available and they each target their unique set of symptoms associated with the disease; the fast uptake of these pipeline drugs will be a principal driver of growth over the forecast period. However, lucrative opportunities remain for companies and products that improve upon one or more of unmet needs that will remain after the forecast period.

Scope

- Overview of FXS, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
- Annualized FXS therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in three patient segments (0-9 Year Olds, 10-19 Year Olds, 20+ Year Olds), forecast from 2016 to 2026.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the FXS therapeutics market.
- Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for FXS therapy. The most promising candidates in Phase IIb and Phase III developments are profiled.
- Analysis of the current and future market competition in the global FXS therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to –
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global FXS therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global FXS therapeutics market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

【レポートの目次】

1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 7
1.2 List of Figures 8
2 Executive Summary 9
2.1 The FXS Market Will Experience Double-Digit Growth from 2016 to 2026 10
2.2 R&D Within the FXS Market 11
2.3 The First Therapies Approved Specifically for Fragile X Are on the Horizon 12
2.4 Market Opportunities for New Entrants 12
2.5 The Launch of Late-Stage Pipeline Drugs Will Be the Principal Driver of Sales Growth 13
2.6 What Do Physicians Think? 14
3 Introduction 16
3.1 Catalyst 16
3.2 Related Reports 16
4 Disease Overview 17
4.1 Etiology and Pathophysiology 17
4.1.1 Etiology 17
4.1.2 Pathophysiology 21
4.2 Classification or Staging Systems 24
4.3 Symptoms 25
4.4 Quality of Life 29
5 Epidemiology 31
5.1 Disease Background 31
5.2 Risk Factors and Comorbidities 31
5.3 Global and Historical Trends 32
5.4 Forecast Methodology 33
5.4.1 Sources 33
5.4.2 Forecast Assumptions and Methods 34
5.5 Epidemiological Forecast for FXS (2016-2026) 35
5.5.1 Diagnosed Prevalent Cases of FXS 35
5.5.2 Age-Specific Diagnosed Prevalent Cases of FXS 36
5.5.3 Sex-Specific Diagnosed Prevalent Cases of FXS 37
5.6 Discussion 38
5.6.1 Epidemiological Forecast Insight 38
5.6.2 Limitations of Analysis 38
5.6.3 Strengths of Analysis 39
6 Current Treatment Options 40
6.1 Overview 40
6.2 Diagnosis and Treatment 40
6.2.1 Diagnosis 40
6.2.2 Treatment Guidelines and Leading Prescribed Drugs 43
6.3 Clinical Practice 47
6.4 Selective Serotonin Reuptake Inhibitors 49
6.4.1 Overview 49
6.4.2 Efficacy and Safety 50
6.4.3 SWOT Analysis 51
6.5 Other Antidepressants 51
6.5.1 Overview 51
6.5.2 Efficacy and Safety 53
6.5.3 SWOT Analysis 55
6.6 Psychostimulants 55
6.6.1 Overview 55
6.6.2 Efficacy 57
6.6.3 Safety 58
6.6.4 SWOT Analysis 59
6.7 Atypical Antipsychotics 59
6.7.1 Overview 59
6.7.2 Efficacy 60
6.7.3 Safety 62
6.7.4 SWOT Analysis 63
6.8 Anticonvulsants 63
6.8.1 Overview 63
6.8.2 Efficacy 66
6.8.3 Safety 67
6.8.4 SWOT Analysis 69
6.9 Lithium 70
6.9.1 Overview 70
6.9.2 Efficacy 71
6.9.3 Safety 72
6.9.4 SWOT Analysis 73
6.10 Other Therapeutic Classes 73
7 Unmet Needs and Opportunity Assessment 74
7.1 Overview 74
7.2 Medication Specifically Approved for FXS Treatment 75
7.2.1 Unmet Need 75
7.2.2 Gap Analysis 76
7.2.3 Opportunity 76
7.3 Improved Efficacy and Safety Profile of Current Treatments 77
7.3.1 Unmet Need 77
7.3.2 Gap Analysis 78
7.3.3 Opportunity 79
7.4 Improved Clinical Trial Design 79
7.4.1 Unmet Need 79
7.4.2 Gap Analysis 82
7.4.3 Opportunity 82
7.5 Early Diagnosis Capability for Early Intervention and Improved Prognosis 82
7.5.1 Unmet Need 82
7.5.2 Gap Analysis 83
7.5.3 Opportunity 84
8 R&D Strategies 85
8.1 Overview 85
8.1.1 Modulating Inhibitory Neurotransmission 86
8.1.2 Modulating Excitatory Neurotransmission 87
8.1.3 Other Therapeutic Approaches 87
8.2 Clinical Trial Design 88
8.2.1 Lack of High-Quality Outcome Measures 89
8.2.2 Biomarkers for FXS 89
8.2.3 Consensus on Outcome Measures 90
9 Pipeline Assessment 92
9.1 Overview 92
9.2 Acamprosate Calcium 95
9.2.1 Overview 95
9.2.2 Efficacy 97
9.2.3 Safety 98
9.2.4 SWOT Analysis 99
9.3 Cannabidiol (ZYN002) 99
9.3.1 Overview 99
9.3.2 Efficacy 101
9.3.3 Safety 102
9.3.4 SWOT Analysis 103
9.4 Ganaxolone 104
9.4.1 Overview 104
9.4.2 Efficacy 105
9.4.3 Safety 106
9.4.4 SWOT Analysis 107
9.5 Trofinetide 107
9.5.1 Overview 107
9.5.2 Efficacy 109
9.5.3 Safety 110
9.5.4 SWOT Analysis 111
9.6 Innovative Early-Stage Approaches 111
9.6.1 Bryostatin-1 112
9.6.2 Gaboxadol 112
9.6.3 AMO-01 112
10 Pipeline Valuation Analysis 113
10.1 Clinical Benchmark of Key Pipeline Drugs 113
10.2 Commercial Benchmark of Key Pipeline Drugs 114
10.3 Competitive Assessment 115
10.4 Top-Line 10-Year Forecast 116
10.4.1 US 120
10.4.2 5EU 121
10.4.3 Japan 123
11 Appendix 125
11.1 Bibliography 125
11.2 Abbreviations 131
11.3 Methodology 133
11.4 Forecasting Methodology 134
11.4.1 Diagnosed Patients 134
11.4.2 Percent Drug-Treated Patients 134
11.4.3 Drugs Included in Each Therapeutic Class 135
11.4.4 Launch Dates 135
11.4.5 General Pricing Assumptions 136
11.4.6 Individual Drug Assumptions 137
11.4.7 Generic Erosion 142
11.4.8 Pricing of Pipeline Agents 142
11.5 Primary Research – KOLs Interviewed for This Report 143
11.6 Primary Research – Prescriber Survey 144
11.7 About the Authors 145
11.7.1 Analyst 145
11.7.2 Therapy Area Director 145
11.7.3 Epidemiologist 146
11.7.4 Managing Epidemiologist 146
11.7.5 Global Director of Therapy Analysis and Epidemiology 147
11.7.6 Global Head and EVP of Healthcare Operations and Strategy 148
11.8 About GlobalData 149
11.9 Contact Us 149
11.10 Disclaimer 150

1.1 List of Tables
Table 1: Fragile X Syndrome: Key Metrics in the 7MM 9
Table 2: Expansion Risk from Permutation to Full Mutation in Women 21
Table 3: Symptoms and Characteristics Associated with Fragile X Syndrome Description 28
Table 4: Comorbidities for FXS 32
Table 5: 7MM, Diagnosed Prevalent Cases of FXS, All Ages, Both Sexes, N, 2016-2026 35
Table 6: Treatment Guidelines for FXS 45
Table 7: Most Prescribed Drug Classes for FXS in the Global Markets, 2017 47
Table 8: Product Profile - SSRIs 50
Table 9: SSRIs SWOT Analysis, 2017 51
Table 10: Product Profile - Other Antidepressants 53
Table 11: Antidepressant SWOT Analysis, 2017 55
Table 12: Product Profile - Psychostimulants 57
Table 13: Psychostimulants SWOT Analysis, 2017 59
Table 14: Product Profile - Atypical Antipsychotics 60
Table 15: Atypical Antipsychotic SWOT Analysis, 2017 63
Table 16: Product Profile - Anticonvulsants 66
Table 17: Anticonvulsant SWOT Analysis, 2017 69
Table 18: Product Profile - Lithium 71
Table 19: Lithium SWOT Analysis, 2017 73
Table 20: Summary of Minor Therapeutic Classes, 2017 73
Table 21: Key Target Therapy Strategies Pursued by FXS Drug Developers 86
Table 22: Key Phase II/III Trials for the Promising Pipeline Agents that GlobalData Expects be Licensed for FXS in the 7MM During the Forecast Period 95
Table 23: Product Profile - Acamprosate Calcium 97
Table 24: Ongoing Phase III Study of Acamprosate Calcium in the US 98
Table 25: Acamprosate Calcium SWOT Analysis, 2017 99
Table 26: Product Profile - Cannabidiol 101
Table 27: Ongoing Phase I/II FAB-C Study of Cannabidiol in Australia 102
Table 28: Cannabidiol SWOT Analysis, 2017 103
Table 29: Product Profile - Ganaxolone 105
Table 30: Key Results from the Phase II Study of Ganaxolone in Belgium and the US 106
Table 31: Ganaxolone SWOT Analysis, 2017 107
Table 32: Product Profile - Trofinetide 109
Table 33: Trofinetide SWOT Analysis, 2017 111
Table 34: Innovative Early-Stage Approaches for FXS, 2017 111
Table 35: Clinical Benchmark of Key Pipeline Drugs - FXS 113
Table 36: Commercial Benchmark of Key Pipeline Drugs - FXS 114
Table 37: Key Events Impacting Sales for FXS, 2016-2026 119
Table 38: FXS Market - Global Drivers and Barriers, 2016-2026 119
Table 39: Key Historical and Projected Patent Expiry Dates for FXS 135
Table 40: Key Launch Dates for FXS in the 7MM 135
Table 41: High-Prescribing Physicians (non-KOLs) Surveyed, By Country 144

1.2 List of Figures
Figure 1: Global Sales Forecast by Country for Fragile X Syndrome in 2016 and 2026 11
Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against Methylphenidate 14
Figure 3: Genetic Location of FM1 Gene With Locus Xq27.3 18
Figure 4: Illustration of mGluR Theory of FXS 23
Figure 5: Typical Phenotypic Characteristics of FXS 26
Figure 6: 7MM, Sources Used, Diagnosed Prevalent Cases of FXS 33
Figure 7: 7MM, Age-Specific Diagnosed Prevalent Cases of FXS, Both Sexes, N, 2016 36
Figure 8: 7MM, Sex-Specific Diagnosed Prevalent Cases of FXS, All Ages, N, 2016 37
Figure 9: Unmet Need and Opportunity in Fragile X Syndrome, 2017 75
Figure 10: Overview of the Development Pipeline in FXS 94
Figure 11: Competitive Assessment of the FXS Pipeline Drugs Benchmarked Against Methylphenidate 116
Figure 12: Global (7MM) Sales Forecast by Country for FXS in 2016 and 2026 117
Figure 13: Global Sales Forecast by Class for FXS in 2016 and 2026 118
Figure 14: Sales Forecast by Class for FXS in the US in 2016 and 2026 121
Figure 15: Sales Forecast by Class for FXS in the 5EU in 2016 and 2026 122
Figure 16: Sales Forecast by Class for FXS in Japan in 2016 and 2026 124

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