Product Code: GDHC039POA
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.
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?
- 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-naive 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.
- 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
1. Table of Contents
- 1.1. List of Tables
- 1.2. List of Figures
- 2.1. Catalyst
- 2.2. Related Reports
3. Disease Overview
- 3.1. Etiology and Pathophysiology
- 3.1.1. Etiology
- 3.1.2. Pathophysiology
- 3.2. Symptoms
- 4.1. Disease Background
- 4.2. Risk Factors and Comorbidities/Manifestations
- 4.3. Global Trends
- 4.3.1. US
- 4.3.2. 5EU
- 4.3.3. Japan
- 4.4. Forecast Methodology
- 4.4.1. Sources Used
- 4.4.2. Sources Not Used
- 4.4.3. Forecast Assumptions and Methods - Diagnosed Prevalent Cases
- 4.5. Epidemiological Forecast for Fabry Disease (2014-2024)
- 4.5.1. Diagnosed Prevalent Cases of Fabry Disease
- 4.5.2. Age-Specific Diagnosed Prevalent Cases of Fabry Disease
- 4.5.3. Sex-Specific Diagnosed Prevalent Cases of Fabry Disease
- 4.6. Discussion
- 4.6.1. Epidemiological Forecast Insight
- 4.6.2. Limitations of the Analysis
- 4.6.3. Strengths of the Analysis
5. Current Treatment Options
- 5.1. Overview
- 5.2. Product Profiles
- 5.2.1. Fabrazyme (Agalsidase Beta)
- 5.2.2. Replagal (Agalsidase Alfa)
6. Unmet Needs Assessment and Opportunity Analysis
- 6.1. Overview
- 6.2. Earlier Fabry Disease Diagnosis
- 6.2.1. Unmet Need
- 6.2.2. Gap Analysis
- 6.2.3. Opportunity
- 6.3. Fabry Disease Treatments with Improved Efficacy
- 6.3.1. Unmet Need
- 6.3.2. Gap Analysis
- 6.3.3. Opportunity
- 6.4. Lower Cost of Fabry Treatments
- 6.4.1. Unmet Need
- 6.4.2. Gap Analysis
- 6.4.3. Opportunity
- 6.5. Widespread Availability of Home-Based Infusion to Improve Compliance
- 6.5.1. Unmet Need
- 6.5.2. Gap Analysis
- 6.5.3. Opportunity
7. Research and Development Strategies
- 7.1. Overview
- 7.1.1. Chaperone Therapies and Their Drug Combinations
- 7.1.2. Substrate Reduction Therapies
- 7.2. Clinical Trial Design
- 7.2.1. Efficacy Endpoints
- 7.2.2. Clinical Trial Treatment Periods
- 7.2.3. Challenges in Fabry Disease Clinical Trials
8. Pipeline Assessment
- 8.1. Overview
- 8.2. Promising Drugs in Clinical Development
- 8.3. Innovative Early-Stage Approaches
- 8.4. Biosimilars
9. Pipeline Valuation Analysis
- 9.1. Clinical Benchmark of Key Pipeline Drugs
- 9.2. Commercial Benchmark of Key Pipeline Drugs
- 9.3. Competitive Assessment
- 9.4. Top-Line 10-Year Forecast
- 9.4.1. US
- 9.4.2. 5EU
- 9.4.3. Japan
- 9.4.4. Drivers and Barriers
- 10.1. Bibliography
- 10.2. Abbreviations
- 10.3. Methodology
- 10.4. Forecast Methodology
- 10.4.1. Percent Diagnosed Patients
- 10.4.2. Percent Drug-Treated Patients
- 10.4.3. Drugs Included in Each Therapeutic Class
- 10.4.4. Launch Dates
- 10.4.5. General Pricing Assumptions
- 10.4.6. Individual Drug Assumptions
- 10.5. Physicians and Specialists Included in This Study
- 10.6. About the Authors
- 10.6.1. Analyst
- 10.6.2. Therapy Area Director
- 10.6.3. Epidemiologist
- 10.6.4. Global Director of Therapy Analysis and Epidemiology
- 10.6.5. Global Head of Healthcare
- 10.7. About GlobalData
- 10.8. Disclaimer
List of Tables
- Table 1: Pathophysiological Findings in Fabry Disease Tissue Specimens
- Table 2: Typical Signs of Fabry Disease According to Patient Age
- Table 3: 7MM, Sources of Fabry Disease Diagnosed Prevalence Data
- Table 4: 7MM, Diagnosed Prevalent Cases of Fabry Disease, All Ages, Both Sexes, N, Selected Years 2014-2024
- Table 5: 7MM, Age-Specific Diagnosed Prevalent Cases of Fabry Disease, Both Sexes, N (Row %), 2014
- Table 6: 7MM, Sex-Specific Diagnosed Prevalent Cases of Fabry Disease, All Ages, N (Row %), 2014
- Table 7: Key Marketed Products for Fabry Disease, 7MM
- Table 8: Guidelines for the Initiation of ERT in Fabry Disease Patients
- Table 9: Product Profile - Fabrazyme
- Table 10: Efficacy of Fabrazyme at Reducing GL-3 in the Capillary Endothelium of the Tissues
- Table 11: Efficacy of Fabrazyme at Reducing Clinical Events
- Table 12: Adverse Reactions Occurring in the 20-week Phase III Fabrazyme Study
- Table 13: Summary of Adverse Reactions in the Phase IV Post-marketing Fabrazyme Study
- Table 14: Fabrazyme SWOT Analysis, 2015
- Table 15: Product Profile - Replagal
- Table 16: Efficacy of Replagal at Reducing Overall Neuropathic Pain
- Table 17: Replagal SWOT Analysis, 2015
- Table 18: Unmet Need in Fabry Disease
- Table 19: Late-Stage Pipeline Products for Fabry Disease, 7MM
- Table 20: Product Profile - Migalastat
- Table 21: Efficacy of Fabrazyme at Reducing GL-3 and Plasma Lyso-GL-3 in the Capillary Endothelium of the Tissues
- Table 22: Summary of Migalastat Safety in Fabry Patients (Study 012)
- Table 23: Migalastat SWOT Analysis, 2015
- Table 24: Early-Stage Pipeline Products for Fabry Disease, 7MM
- Table 25: Pipeline Agalsidase Biosimilars for Fabry Disease, 7MM
- Table 26: Clinical Benchmark of Key Pipeline Drugs - Fabry Disease Treatments
- Table 27: Commercial Benchmark of Key Pipeline Drugs - Fabry Disease Treatments
- Table 28: Top-Line Sales Forecasts ($m) for the Fabry Disease Market in the 7MM, 2014-2024
- Table 29: Key Events Impacting Sales in the Fabry Disease Market, 2014-2024
- Table 30: Fabry Disease Market - Drivers and Barriers, 2014-2024
- Table 31: Launch Dates in the Fabry disease Market, 2014-2024
List of Figures
- Figure 1: 7MM, Diagnosed Prevalent Cases of Fabry Disease, All Ages, Both Sexes, N, 2014-2024
- Figure 2: 7MM, Age-Specific Diagnosed Prevalent Cases of Fabry Disease, Both Sexes, N, 2014
- Figure 3: 7MM, Sex-Specific Diagnosed Prevalent Cases of Fabry Disease, All Ages, N, 2014
- Figure 4: Competitive assessment of Late Stage Pipeline Agents for Fabry Disease, 2014-2024
- Figure 5: Sales for the Fabry Disease Market in the 7MM, 2014-2024