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Opportunity Analyzer: Fabry Disease - Opportunity Analysis and Forecast to 2024

Summary

GlobalData estimates the 2014 sales for the Fabry disease market at approximately $682m across the 7MM. The US contributed the majority of these sales, generating an estimated $295m. By the end of the forecast period in 2024, Fabry disease sales are expected to grow to $1.25 billion at a Compound Annual Growth Rate (CAGR) of 6.3% over the 10-year period. The majority of sales in the 7MM in 2024 will come from the US, which will represent 44.2% of the market. GlobalData expects an increase in the number of newly diagnosed cases of Fabry disease, and consequently in the number of treatable Fabry patients, as a result of increasing awareness of Fabry disease among physicians. ERT is now well established in the treatment of Fabry disease; however, there still remains concern about its efficacy, tissue penetrance, and intravenous administration. As a result, alternative approaches are being investigated to advance new treatments for Fabry disease, which focus on three main areas of research: chaperone therapies, substrate reduction therapies (SRTs), and combinations of their use with ERT.

Highlights

Key Questions Answered

- At present, patient registries demonstrate a long delay between onset of initial symptoms and a diagnosis, which can span between 10 to 20 years. This is due the condition being very rare, the lack of awareness of the disease among physicians, and the diverse range of symptoms that a patient may have when initially presenting with the disease. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs in this market?
- Since the approval of Fabrazyme and Replagal in the EU in 2001, no other drugs have been approved for the treatment of Fabry disease. Will the pipeline drugs in development change the treatment landscape for Fabry disease and attain high sales revenues during 2014-2024?
- Key opinion leaders interviewed by GlobalData believe the biggest opportunity lies with combination therapies, to improve drug delivery and increase drugs’ efficacy,. How will these changes impact the growth of the future market?

Key Findings

- One of the main drivers for the Fabry disease market growth will be an increase in the number of newly diagnosed cases of Fabry disease, and consequently in the number of treatable Fabry patients, as a result of increasing awareness of Fabry disease among physicians.
- The first pharmacological chaperone for the treatment of Fabry disease, Amicus Therapeutics’ migalastat, will launch in the US and 5EU (France, Germany, Italy, Spain, and UK) in 2017, followed by its launch in Japan in 2020. Migalastat patient share is not only expected to be captured from existing patients receiving enzyme replacement therapy (ERT), but also treatment-naïve patients who have not previously been considered suitable for ERT.
- There is a lack of suitable treatments available for young children with Fabry disease. ERT requires intravenous infusions every two weeks; hence, until orally available treatment options become available, such as Amicus’ migalastat, it is expected that expansion of the Fabry disease market to include younger children will not occur.

Scope

- Overview of Fabry disease, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
- Annualized Fabry disease therapeutics market revenue, annual cost of therapy and treatment usage pattern data from from 2014 and forecast for ten years to 2024.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the Fabry disease therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
- Analysis of the current and future market competition in the global Fabry disease 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

- 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. Additionally a list of acquisition targets included in the pipeline product company list.
- Develop business strategies by understanding the trends shaping and driving the Fabry disease therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Fabry disease therapeutics market in 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.
- Track drug sales in the 7MM Fabry disease therapeutics market from 2014-2024.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table Of Contents

Opportunity Analyzer: Fabry Disease - Opportunity Analysis and Forecast to 2024
1 Table of Contents
1 Table of Contents 8
1.1 List of Tables 12
1.2 List of Figures 13
2 Introduction 14
2.1 Catalyst 14
2.2 Related Reports 14
3 Disease Overview 15
3.1 Etiology and Pathophysiology 15
3.1.1 Etiology 15
3.1.2 Pathophysiology 15
3.2 Symptoms 18
4 Epidemiology 19
4.1 Disease Background 19
4.2 Risk Factors and Comorbidities/Manifestations 20
4.3 Global Trends 20
4.3.1 US 20
4.3.2 5EU 20
4.3.3 Japan 21
4.4 Forecast Methodology 21
4.4.1 Sources Used 22
4.4.2 Sources Not Used 23
4.4.3 Forecast Assumptions and Methods - Diagnosed Prevalent Cases 24
4.5 Epidemiological Forecast for Fabry Disease (2014-2024) 26
4.5.1 Diagnosed Prevalent Cases of Fabry Disease 26
4.5.2 Age-Specific Diagnosed Prevalent Cases of Fabry Disease 27
4.5.3 Sex-Specific Diagnosed Prevalent Cases of Fabry Disease 29
4.6 Discussion 31
4.6.1 Epidemiological Forecast Insight 31
4.6.2 Limitations of the Analysis 32
4.6.3 Strengths of the Analysis 32
5 Current Treatment Options 34
5.1 Overview 34
5.2 Product Profiles 40
5.2.1 Fabrazyme (Agalsidase Beta) 40
5.2.2 Replagal (Agalsidase Alfa) 50
6 Unmet Needs Assessment and Opportunity Analysis 57
6.1 Overview 57
6.2 Earlier Fabry Disease Diagnosis 57
6.2.1 Unmet Need 57
6.2.2 Gap Analysis 59
6.2.3 Opportunity 61
6.3 Fabry Disease Treatments with Improved Efficacy 62
6.3.1 Unmet Need 62
6.3.2 Gap Analysis 64
6.3.3 Opportunity 65
6.4 Lower Cost of Fabry Treatments 66
6.4.1 Unmet Need 66
6.4.2 Gap Analysis 67
6.4.3 Opportunity 69
6.5 Widespread Availability of Home-Based Infusion to Improve Compliance 69
6.5.1 Unmet Need 69
6.5.2 Gap Analysis 70
6.5.3 Opportunity 72
7 Research and Development Strategies 73
7.1 Overview 73
7.1.1 Chaperone Therapies and Their Drug Combinations 73
7.1.2 Substrate Reduction Therapies 75
7.2 Clinical Trial Design 77
7.2.1 Efficacy Endpoints 77
7.2.2 Clinical Trial Treatment Periods 78
7.2.3 Challenges in Fabry Disease Clinical Trials 79
8 Pipeline Assessment 82
8.1 Overview 82
8.2 Promising Drugs in Clinical Development 83
8.2.1 Migalastat 83
8.3 Innovative Early-Stage Approaches 98
8.4 Biosimilars 99
9 Pipeline Valuation Analysis 103
9.1 Clinical Benchmark of Key Pipeline Drugs 103
9.2 Commercial Benchmark of Key Pipeline Drugs 104
9.3 Competitive Assessment 104
9.4 Top-Line 10-Year Forecast 107
9.4.1 US 110
9.4.2 5EU 110
9.4.3 Japan 111
9.4.4 Drivers and Barriers 113
10 Appendix 114
10.1 Bibliography 114
10.2 Abbreviations 121
10.3 Methodology 123
10.4 Forecast Methodology 123
10.4.1 Percent Diagnosed Patients 123
10.4.2 Percent Drug-Treated Patients 124
10.4.3 Drugs Included in Each Therapeutic Class 124
10.4.4 Launch Dates 124
10.4.5 General Pricing Assumptions 124
10.4.6 Individual Drug Assumptions 125
10.5 Physicians and Specialists Included in This Study 127
10.6 About the Authors 129
10.6.1 Analyst 129
10.6.2 Therapy Area Director 129
10.6.3 Epidemiologist 130
10.6.4 Global Director of Therapy Analysis and Epidemiology 130
10.6.5 Global Head of Healthcare 131
10.7 About GlobalData 132
10.8 Disclaimer 132

1.1 List of Tables
Table 1: Pathophysiological Findings in Fabry Disease Tissue Specimens 16
Table 2: Typical Signs of Fabry Disease According to Patient Age 18
Table 3: 7MM, Sources of Fabry Disease Diagnosed Prevalence Data 22
Table 4: 7MM, Diagnosed Prevalent Cases of Fabry Disease, All Ages, Both Sexes, N, Selected Years 2014-2024 26
Table 5: 7MM, Age-Specific Diagnosed Prevalent Cases of Fabry Disease, Both Sexes, N (Row %), 2014 28
Table 6: 7MM, Sex-Specific Diagnosed Prevalent Cases of Fabry Disease, All Ages, N (Row %), 2014 30
Table 7: Key Marketed Products for Fabry Disease, 7MM 34
Table 8: Guidelines for the Initiation of ERT in Fabry Disease Patients 38
Table 9: Product Profile — Fabrazyme 43
Table 10: Efficacy of Fabrazyme at Reducing GL-3 in the Capillary Endothelium of the Tissues 44
Table 11: Efficacy of Fabrazyme at Reducing Clinical Events 45
Table 12: Adverse Reactions Occurring in the 20-week Phase III Fabrazyme Study 46
Table 13: Summary of Adverse Reactions in the Phase IV Post-marketing Fabrazyme Study 47
Table 14: Fabrazyme SWOT Analysis, 2015 49
Table 15: Product Profile - Replagal 52
Table 16: Efficacy of Replagal at Reducing Overall Neuropathic Pain 53
Table 17: Replagal SWOT Analysis, 2015 56
Table 18: Unmet Need in Fabry Disease 57
Table 19: Late-Stage Pipeline Products for Fabry Disease, 7MM 83
Table 20: Product Profile - Migalastat 89
Table 21: Efficacy of Fabrazyme at Reducing GL-3 and Plasma Lyso-GL-3 in the Capillary Endothelium of the Tissues 91
Table 22: Summary of Migalastat Safety in Fabry Patients (Study 012) 95
Table 23: Migalastat SWOT Analysis, 2015 97
Table 24: Early-Stage Pipeline Products for Fabry Disease, 7MM 98
Table 25: Pipeline Agalsidase Biosimilars for Fabry Disease, 7MM 99
Table 26: Clinical Benchmark of Key Pipeline Drugs - Fabry Disease Treatments 103
Table 27: Commercial Benchmark of Key Pipeline Drugs - Fabry Disease Treatments 104
Table 28: Top-Line Sales Forecasts ($m) for the Fabry Disease Market in the 7MM, 2014-2024 108
Table 29: Key Events Impacting Sales in the Fabry Disease Market, 2014-2024 109
Table 30: Fabry Disease Market - Drivers and Barriers, 2014-2024 113
Table 31: Launch Dates in the Fabry disease Market, 2014-2024 124

1.2 List of Figures
Figure 1: 7MM, Diagnosed Prevalent Cases of Fabry Disease, All Ages, Both Sexes, N, 2014-2024 27
Figure 2: 7MM, Age-Specific Diagnosed Prevalent Cases of Fabry Disease, Both Sexes, N, 2014 29
Figure 3: 7MM, Sex-Specific Diagnosed Prevalent Cases of Fabry Disease, All Ages, N, 2014 31
Figure 4: Competitive assessment of Late Stage Pipeline Agents for Fabry Disease, 2014-2024 105
Figure 5: Sales for the Fabry Disease Market in the 7MM, 2014?2024 109

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