OpportunityAnalyzer: Fabry Disease – Opportunity Analysis and Forecast to 2024

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.

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 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.

Key Highlights

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?

Companies mentioned

Genzyme

Shire

Amicus Therapeutics

Sanofi

Table of Contents

1Table of Contents

1.1List of Tables

1.2List of Figures

2Introduction

2.1Catalyst

2.2Related Reports

3Disease Overview

3.1Etiology and Pathophysiology

3.1.1Etiology

3.1.2Pathophysiology

3.2Symptoms

4Epidemiology

4.1Disease Background

4.2Risk Factors and Comorbidities/Manifestations

4.3Global Trends

4.3.1US

4.3.25EU

4.3.3Japan

4.4Forecast Methodology

4.4.1Sources Used

4.4.2Sources Not Used

4.4.3Forecast Assumptions and Methods – Diagnosed Prevalent Cases

4.5Epidemiological Forecast for Fabry Disease (2014–2024)

4.5.1Diagnosed Prevalent Cases of Fabry Disease

4.5.2Age-Specific Diagnosed Prevalent Cases of Fabry Disease

4.5.3Sex-Specific Diagnosed Prevalent Cases of Fabry Disease

4.6Discussion

4.6.1Epidemiological Forecast Insight

4.6.2Limitations of the Analysis

4.6.3Strengths of the Analysis

5Current Treatment Options

5.1Overview

5.2Product Profiles

5.2.1Fabrazyme (Agalsidase Beta)

5.2.2Replagal (Agalsidase Alfa)

6Unmet Needs Assessment and Opportunity Analysis

6.1Overview

6.2Earlier Fabry Disease Diagnosis

6.2.1Unmet Need

6.2.2Gap Analysis

6.2.3Opportunity

6.3Fabry Disease Treatments with Improved Efficacy

6.3.1Unmet Need

6.3.2Gap Analysis

6.3.3Opportunity

6.4Lower Cost of Fabry Treatments

6.4.1Unmet Need

6.4.2Gap Analysis

6.4.3Opportunity

6.5Widespread Availability of Home-Based Infusion to Improve Compliance

6.5.1Unmet Need

6.5.2Gap Analysis

6.5.3Opportunity

7Research and Development Strategies

7.1Overview

7.1.1Chaperone Therapies and Their Drug Combinations

7.1.2Substrate Reduction Therapies

7.2Clinical Trial Design

7.2.1Efficacy Endpoints

7.2.2Clinical Trial Treatment Periods

7.2.3Challenges in Fabry Disease Clinical Trials

8Pipeline Assessment

8.1Overview

8.2Promising Drugs in Clinical Development

8.2.1Migalastat

8.3Innovative Early-Stage Approaches

8.4Biosimilars

9Pipeline Valuation Analysis

9.1Clinical Benchmark of Key Pipeline Drugs

9.2Commercial Benchmark of Key Pipeline Drugs

9.3Competitive Assessment

9.4Top-Line 10-Year Forecast

9.4.1US

9.4.25EU

9.4.3Japan

9.4.4Drivers and Barriers

10Appendix

10.1Bibliography

10.2Abbreviations

10.3Methodology

10.4Forecast Methodology

10.4.1Percent Diagnosed Patients

10.4.2Percent Drug-Treated Patients

10.4.3Drugs Included in Each Therapeutic Class

10.4.4Launch Dates

10.4.5General Pricing Assumptions

10.4.6Individual Drug Assumptions

10.5Physicians and Specialists Included in This Study

10.6About the Authors

10.6.1Analyst

10.6.2Therapy Area Director

10.6.3Epidemiologist

10.6.4Global Director of Therapy Analysis and Epidemiology

10.6.5Global Head of Healthcare

10.7About GlobalData

10.8Disclaimer

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

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