1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends
  4. > Opportunity Analyzer: Gaucher Disease - Opportunity Analysis and Forecast to 2024

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

Summary

GlobalData estimated the 2014 sales for the Gaucher Disease (GD) market at approximately $875m across the 7MM (US, France, Germany, Italy, Spain, UK, and Israel). The US market contributed the majority of these sales, generating an estimated $482m. By the end of the forecast period in 2024, GD sales are expected to increase to $1.16 billion at a Compound Annual Growth Rate (CAGR) of 2.9%. The majority of these sales will continue to come from the US, which will represent approximately 56.2% of the GD market in the 7MM in 2024. GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. Despite the anticipated increase in the number of diagnosed prevalent cases of GD over the forecast period, it is expected that a large portion of the GD population will remain undiagnosed. This is due to continued late diagnosis and misdiagnosis of GD as a result of the heterogeneous manifestations of the disease. The sparsely populated GD pipeline, with no drugs in late-stage development, will further hinder the growth of the GD market.

Highlights

Key Questions Answered

- Although the use of enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs) has significantly changed the natural history of GD, with a marked decrease in morbidity, especially in Type 1 GD patients, GD market is still marked by the presence of a number of unmet needs in current treatments. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the GD market?
- The late-stage GD pipeline is very sparsely populated and there no drugs in late-stage development. Nevertheless, the GD market will experience substantial growth. What are the main drivers of this growth during 2014-2024? Which of the marketed drugs will have the highest peak sales at the highest CAGR, and why?

Key Findings

- GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. This is largely attributable to Genzyme’s and Shire’s continued efforts to raise awareness of rare diseases through organized events and established websites, and by sponsoring patient registries.
- Genzyme’s recently approved oral substrate reduction therapy (SRT), Cerdelga (eliglustat), is more effective and safer than the other oral SRT, Zavesca (miglustat), and therefore will be used more commonly as an oral alternative to IV ERT in adult Type 1 GD patients. As Cerdelga is more expensive than the other GD therapies, it is expected to drive the growth of the GD market.
- None of the therapies fulfilled the greatest unmet need in GD treatment — need for the drugs that can treat the neurological manifestations of the disease. One of the main R&D strategies in the GD space is the focus on the development of new SRT formulations and pharmacological chaperones (PCs) that can cross the blood brain barrier and thereby improve the neurological manifestations of GD.

Scope

- Overview of GD, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
- Annualized GD 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 GD 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 GD 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. Additionally a list of acquisition targets included in the pipeline product company list.
- Develop business strategies by understanding the trends shaping and driving the GD therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the GD 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 GD 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: Gaucher Disease - Opportunity Analysis and Forecast to 2024
1 Table of Contents
1 Table of Contents 10
1.1 List of Tables 14
1.2 List of Figures 17
2 Introduction 18
2.1 Catalyst 18
2.2 Related Reports 19
3 Disease Overview 20
3.1 Etiology and Pathophysiology 23
3.1.1 Etiology 23
3.1.2 Pathophysiology 26
3.2 Symptoms 27
3.3 Prognosis 31
3.4 Quality of Life 32
4 Epidemiology 33
4.1 Disease Background 33
4.2 Risk Factors and Comorbidities 34
4.3 Global Trends 35
4.3.1 US 35
4.3.2 5EU 36
4.3.3 Israel 36
4.4 Forecast Methodology 37
4.4.1 Sources Used 37
4.4.2 Sources Not Used 38
4.4.3 Forecast Assumptions and Methods 39
4.5 Epidemiological Forecast for Gaucher Disease (2014-2024) 40
4.5.1 Total Prevalent Cases of Type 1 Gaucher Disease 40
4.5.2 Age-Specific Total Prevalent Cases of Type 1 Gaucher Disease 42
4.5.3 Sex-Specific Total Prevalent Cases of Type 1 Gaucher Disease 44
4.5.4 Age-Standardized Total Prevalence of Type 1 Gaucher Disease 46
4.5.5 Total Prevalent Cases of Type 2 Gaucher Disease 47
4.5.6 Sex-Specific Total Prevalent Cases of Type 2 Gaucher Disease 50
4.5.7 Total Prevalent Cases of Type 3 Gaucher Disease 50
4.5.8 Age-Specific Total Prevalent Cases of Type 3 Gaucher Disease 52
4.5.9 Sex-Specific Total Prevalent Cases of Type 3 Gaucher Disease 54
4.5.10 Age-Standardized Total Prevalence of Type 3 Gaucher Disease 55
4.6 Discussion 56
4.6.1 Epidemiological Forecast Insight 56
4.6.2 Limitations of the Analysis 57
4.6.3 Strengths of the Analysis 58
5 Current Treatment Options 59
5.1 Overview 59
5.1.1 Enzyme Replacement Therapy 59
5.1.2 Substrate Reduction Therapy 62
5.1.3 ERT Dosing Issues 64
5.1.4 Treatment Guidelines 70
5.2 Product Profiles 73
5.2.1 Enzyme Replacement Therapies 73
5.2.2 Substrate Reduction Therapies 102
6 Unmet Needs Assessment and Oppportunity Analysis 130
6.1 Overview 130
6.2 Treatment of the Neurological Manifestations of Gaucher Disease 132
6.2.1 Unmet Needs 132
6.2.2 Gap Analysis 134
6.2.3 Opportunity 136
6.3 Earlier Gaucher Disease Diagnosis and Disease Severity Assessment 138
6.3.1 Unmet Needs 138
6.3.2 Gap Analysis 145
6.3.3 Opportunity 149
6.4 Lower Cost of Gaucher Disease Treatment 152
6.4.1 Unmet Needs 152
6.4.2 Gap Analysis 153
6.4.3 Opportunity 156
6.5 Improved Route of Administration 158
6.5.1 Unmet Needs 158
6.5.2 Gap Analysis 159
6.5.3 Opportunity 161
7 RandD Strategies 162
7.1 Overview 162
7.1.1 Substrate Reduction Therapy 163
7.1.2 Pharmacological Chaperones and Their Combination with ERT 166
7.2 Clinical Trial Design 168
7.2.1 Efficacy Endpoints 168
7.2.2 Challenges Involved in Gaucher Disease Clinical Trials 171
8 Pipeline Assessment 173
8.1 Promising Drugs in Clinical Development 173
8.2 Innovative Early-Stage Approaches 173
8.2.1 Overview 173
9 Pipeline Valuation Analysis 184
9.1 Clinical Benchmarking of the Key Gaucher Disease Drugs 184
9.2 Commercial Benchmarking of the Key Gaucher Disease Drugs 185
9.3 Competitive Assessment 187
9.4 Top-Line 10-Year Forecast 190
9.4.1 US 193
9.4.2 5EU 194
9.4.3 Israel 195
9.4.4 Drivers and Barriers 198
10 Appendix 199
10.1 Bibliography 199
10.2 Abbreviations 225
10.3 Methodology 230
10.4 Forecasting Methodology 230
10.4.1 Percentage of Diagnosed Gaucher Disease Patients 230
10.4.2 Percentage of Drug-Treated Gaucher Disease Patients 231
10.4.3 Drugs Included in Each Therapeutic Class 232
10.4.4 Key Product Launch and Patent Expiry Dates 232
10.4.5 General Pricing Assumptions 233
10.4.6 Individual Drug Assumptions 235
10.4.7 Generic Erosion 237
10.5 Physicians and Specialists Included in This Study 238
10.6 About the Authors 240
10.6.1 Author 240
10.6.2 Therapy Area Director 240
10.6.3 Epidemiologist 241
10.6.4 Global Head of Healthcare 241
10.6.5 Global Director of Therapy Analysis and Epidemiology 242
10.7 About GlobalData 243
10.8 Disclaimer 243

1.1 List of Tables
Table 1: Types of Gaucher Disease and Their Main Characteristics 22
Table 2: Organ-Specific Clinical Presentation of Gaucher Disease 30
Table 3: 7MM, Sources Used to Forecast the Total Prevalent Cases of Gaucher Disease 37
Table 4: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, Both Sexes, N, 2014-2024 41
Table 5: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, by Age, Both Sexes, N (Row %), 2014 43
Table 6: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, N (Row %), by Sex, 2014 45
Table 7: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, Age ?4 Years, Both Sexes, N, 2014-2024 48
Table 8: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age Table 9: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, by Age, Both Sexes, N (Row %), 2014 53
Table 10: Leading Branded Products for the Treatment of Gaucher Disease 60
Table 11: Gaucher Disease: Risk Assessment 66
Table 12: Recommendations and Guidelines for the Management of Gaucher Disease 71
Table 13: Product Profile - Cerezyme 80
Table 14: Mean Changes in Clinical Parameters from Baseline to Month 9 in Patients with Type 1 Gaucher Disease in Response to Cerezyme Versus Ceredase 81
Table 15: Cerezyme SWOT Analysis, 2015 85
Table 16: Product Profile - VPRIV 88
Table 17: Mean Changes in Clinical Parameters from Baseline to 12 Months in Patients with Type 1 Gaucher Disease in Response to VPRIV in the First Phase III Study 89
Table 18: Mean Changes in Clinical Parameters from Baseline to Month 9 in Patients with Type 1 Gaucher Disease in Response to VPRIV or Cerezyme 90
Table 19: VPRIV SWOT Analysis, 2015 94
Table 20: Product Profile - Elelyso 96
Table 21: Mean Changes in Clinical Parameters from Baseline to Month Nine in Patients with Type 1 GD in Response to Elelyso in the Phase III Study 97
Table 22: Mean Increases in Height and Weight from Baseline to Month 12 in Pediatric Patients with Type 1 GD in Response to Elelyso in a Phase III Study 98
Table 23: Mean Changes in Clinical Parameters at Baseline and at Month Nine in Patients with Type 1 GD in Response to Elelyso in a Phase III Study 99
Table 24: Number of Patients with Drug-Related Adverse Events in the Phase III Study of Elelyso by Dose Group 100
Table 25: Elelyso SWOT Analysis, 2015 102
Table 26: Product Profile - Zavesca 107
Table 27: Changes in the Clinical Parameters in Response to Zavesca 100mg Three Times Daily in Study 1 and Its Extension Study 108
Table 28: Changes in the Clinical Parameters in Response to Zavesca 50mg Three Times Daily in Study 2 and Its Extension Study 109
Table 29: Changes in the Clinical Parameters in Response to Treatment with Zavesca 100mg Three Times Daily in the Two-Year Maintenance Study 111
Table 30: Patients Experiencing Adverse Events During the Randomized and Extension Phases of Study 3 of Zavesca 115
Table 31: Most Frequently Reported Adverse Events in Patients Receiving Zavesca in Combination with Cerezyme in the Two-Year Maintenance Study 116
Table 32: Zavesca SWOT Analysis, 2015 117
Table 33: Recommended Cerdelga Dosage by CYP2D6 Metabolizer Status 118
Table 34: Product Profile - Cerdelga 121
Table 35: Changes in the Primary and Secondary Endpoints After Nine Months of Treatment with Cerdelga Versus Placebo in the ENGAGE Trial 122
Table 36: CYP2D6 Metabolizer Status of Patients Receiving Cerdelga or Cerezyme in the ENCORE Trial 123
Table 37: Mean Changes in the Clinical Parameters from Baseline to Month 12 in Patients with Type 1 GD Who Were Switched to Cerdelga or Received Cerezyme in the ENCORE Trial 124
Table 38: Adverse Events that Occurred in ?10% of Patients Treated with Cerdelga and More Frequently Than with Placebo in the ENGAGE Trial 127
Table 39: Adverse Events Occurring in ?10% of Patients and More Frequently Than with Placebo in the ENGAGE Trial 128
Table 40: Cerdelga SWOT Analysis, 2015 129
Table 41: Unmet Need and Opportunity in Gaucher Disease 131
Table 42: Early-Stage Pipeline Products for Gaucher Disease - US, 5EU, Israel, 2014-2024 174
Table 43: Clinical Benchmarking of the Key Gaucher Disease Drugs - ERTs and SRTs 185
Table 44: Commercial Benchmarking of the Key Drugs in Gaucher Disease - ERTs and SRTs 187
Table 45: Top-Line Sales Forecasts ($m) for GD, 2014-2024 192
Table 46: Key Events Impacting Sales for the Gaucher Disease Market, 2014-2024 193
Table 47: Gaucher Disease Market - Drivers and Barriers, 2014-2024 198
Table 48: Key Product Launch Dates in the Gaucher Disease Market, 2014-2024 232
Table 49: Key Patent Expiry Dates in the Gaucher Disease Market, 2014-2024 232

1.2 List of Figures
Figure 1: Classification of Gaucher Disease 21
Figure 2: Simultaneous Accumulation of Unfolded Proteins and GC in Lysosomal Macrophages in Gaucher Disease - Current Therapeutic Interventions 25
Figure 3: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, Both Sexes, N, 2014-2024 42
Figure 4: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, by Age, Both Sexes, N 44
Figure 5: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, N, by Sex, 2014 46
Figure 6: 7MM, Age-Standardized Total Prevalence of Type 1 Gaucher Disease (%), All Ages, by Sex, 2014 47
Figure 7: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, Age ?4 Years, Both Sexes, N, 2014-2024 49
Figure 8: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, All Ages, by Sex, N, 2014 50
Figure 9: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age Figure 10: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, by Age, Both Sexes, N, 2014 54
Figure 11: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age Figure 12: 7MM, Age-Standardized Total Prevalence of Type 3 Gaucher Disease (%), Age Figure 13: Competitive Assessment of Gaucher Disease Therapies in the 7MM, 2014-2024 189
Figure 14: Sales for the Gaucher Disease Market in the 7MM, 2014-2024 192
Figure 15: Sales of Gaucher Disease Drugs by Country, 2014 and 2024 197

View This Report »

Get Industry Insights. Simply.

  • Latest reports & slideshows with insights from top research analysts
  • 24 Million searchable statistics with tables, figures & datasets
  • More than 10,000 trusted sources
24/7 Customer Support

Talk to Veronica

+1 718 514 2762

Purchase Reports From Reputable Market Research Publishers
PharmaPoint: Atrial Fibrillation - Global Drug Forecast and Market Analysis to 2025

PharmaPoint: Atrial Fibrillation - Global Drug Forecast and Market Analysis to 2025

  • $ 10995
  • Industry report
  • August 2016
  • by Global Data

PharmaPoint: Atrial Fibrillation - Global Drug Forecast and Market Analysis to 2025 Summary Atrial fibrillation (AF) is the most common cardiac arrhythmia and is a heart condition that causes an irregula ...

PharmaPoint: Venous Thromboembolism - Global Drug Forecast and Market Analysis to 2025

PharmaPoint: Venous Thromboembolism - Global Drug Forecast and Market Analysis to 2025

  • $ 10995
  • Industry report
  • August 2016
  • by Global Data

PharmaPoint: Venous Thromboembolism - Global Drug Forecast and Market Analysis to 2025 Summary Venous thromboembolism (VTE) is a disease that includes a myriad of life-threatening disorders ranging from ...

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2025

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2025

  • $ 10995
  • Industry report
  • August 2016
  • by Global Data

PharmaPoint: Acute Coronary Syndrome - Global Drug Forecast and Market Analysis to 2025 Summary Cardiovascular diseases (CVD), including ischemic heart disease and stroke, are the leading causes of mortality ...

Ulcerative Colitis: Kol Insight

September 2016 $ 8085

Hepatitis C: Kol Insight

September 2016 $ 8085

Download Unlimited Documents from Trusted Public Sources

Therapy Market in Europe

  • November 2016
    19 pages
  • Therapy  

  • Europe  

View report >

Therapy Market and Hypertension Statistics in Asia and the US

  • November 2016
    26 pages
  • Therapy  

    Hypertension  

    Cardiovascular ...  

  • Asia  

    United States  

    Europe  

View report >

Therapy Market in the US - Forecast

  • November 2016
    6 pages
  • Therapy  

    Opioid  

  • United States  

View report >

Related Market Segments :

Therapy
Enzyme

ref:plp2015

Reportlinker.com © Copyright 2016. All rights reserved.

ReportLinker simplifies how Analysts and Decision Makers get industry data for their business.