Gaucher's Disease Therapeutics - Pipeline Assessment and Market Forecasts to 2018

Gaucher’s Disease Therapeutics Market Assessment and Market Forecasts to 2018

Summary

GlobalData, the industry analysis specialist, has released its new report, “Gaucher’s Disease Therapeutics - Pipeline Assessment and Market Forecasts to 2018”. The report is an essential source of information and analysis on the global Gaucher's Disease Therapeutics market. The report identifies the key trends shaping and driving the global Gaucher's Disease Therapeutics market. The report also provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the market positioning of the current market leaders. Most importantly, the report provides valuable insights on the pipeline products within the global Gaucher's Disease Therapeutics sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GlobalData’s team of industry experts.

Scope

The report provides information on the key drivers and challenges of the Gaucher's Disease Therapeutics market. Its scope includes -

• Annualized seven key markets (US, France, Germany, Italy, Spain, UK and Japan) Gaucher's Disease Therapeutics market revenues data from 2005 to 2009, forecast for eight years to 2018.
• Pipeline analysis data providing a split across the different phases, mechanisms of action being developed and emerging trends by seven key markets. Pipeline candidates fall under major therapeutic classes.
• Analysis of the current and future competition in the seven key countries Gaucher's Disease Therapeutics market.
• Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide a qualitative analysis of its implications.
• Key topics covered include strategic competitor assessment, market characterization, unmet needs and the implications for the Gaucher's Disease Therapeutics market.
• Analysis of key recent licensing and partnership agreements in Gaucher's Disease Therapeutics market

Reasons to buy

The report will enhance your decision making capability. It will allow 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 Gaucher's Disease Therapeutics market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global Gaucher's Disease Therapeutics market in future.
• Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing 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.
• What’s the next big thing in the global Gaucher's Disease Therapeutics market landscape? - Identify, understand and capitalize.

Additional Information

Gaucher’s Disease Therapeutics Market Forecast to Display High Growth until 2018

GlobalData’s estimates that the global Gaucher’s disease therapeutics market was worth $851.2m in 2010, after increasing at a Compound Annual Growth Rate (CAGR) of 3.8% during 2005 - 2010. The market is expected to record a CAGR of 7.4% from 2010 - 2018, to value $1,512.2m by 2018. Enzyme Reduction Therapy (ERT) and Substrate Replacement Therapy (SRT) are the two currently approved classes for the treatment of Gaucher’s disease. ERT includes Cerezyme (imiglucerase) from Genzyme (a Sanofi company) and Vpriv (velaglucerase alfa) from Shire Pharmaceuticals. Zavesca (miglustat) is an orally administered SRT drug approved in Europe and the US in November 2002 and July 2003 respectively. Cerezyme gained orphan drug designation was approved in the US in May 1994 Vpriv has not been launched in Japan, meaning that there is only one drug available in the country for the treatment of Gaucher’s disease. Genzyme Corporation has been the market leader in the Gaucher’s disease therapeutics market since 1991 and Cerezyme has enjoyed a monopoly for the last two decades. Supply shortages in June 2009 due to viral contamination at the Allston plant in the US allowed Shire’s drug Vpriv to gain a share of the market share in Gaucher’s disease therapeutics market. The production of Cerezyme has not been fully resumed until now.

GlobalData’s analysis found that the significant growth recorded in Gaucher’s disease therapeutics market during 2005-2010 can be primarily attributed to increased sales of Cerezyme as well as the increased annual cost of therapy and increasing disease awareness among physicians and patients. The Gaucher’s disease therapeutics market is expected to grow at a high rate during the forecast period due to the expected launch of novel molecules Uplyso (taliglucerase alfa) eliglustat tartrate and ISU302. These late stage pipeline molecules have improved safety and efficacy profiles, and are more cost effective than current therapeutic options, meaning that accessibility and affordability will increase. These factors are expected to drive the future market growth.

Significant Unmet Need in Gaucher’s Disease Therapeutics Market

GlobalData’s analysis suggests that the Gaucher’s disease therapeutics market has significant unmet need, not only in terms of the limited number of therapeutic options available for type 1 Gaucher’s disease, but also a lack of effective treatment options for severe brain damage cases that can occur in type 2 and 3 Gaucher’s patients. Although ERT is effective in the treatment of type 1 Gaucher’s disease, there is insufficient evidence that ERT or SRT improve neurological symptoms in type 2 and 3 Gaucher’s patients. The lack of an early diagnosis may lead to further disease progression and neurological manifestations, ultimately worsening patient quality of life. The annual cost of therapy with Cerezyme exceeds $300,000 and so it is likely that many patients do not have access to medication. As well as early diagnosis, patient adherence to treatment has also been found to present a major unmet need. For example, a serious complication of Gaucher’s disease is bone disease. If left untreated, it can result in pain, permanent damage to the bone and disability. There is significant potential for the introduction of novel therapies with improved safety and efficacy profiles in the Gaucher’s disease therapeutics market. The expected launch of late stage pipeline molecules such as Uplyso, eliglustat tartrate and ISU302 with better safety and efficacy profiles than existing approved drugs will fulfill the unmet need.

Current Competition in the Gaucher’s Disease Therapeutics Market is Weak

GlobalData’s analysis suggests that the current competition in the Gaucher’s disease therapeutics market is weak. The products currently dominating the market are Cerezyme, Vpriv and Zavesca. Zavesca is the only orally administered approved treatment option available for Gaucher’s patients for whom ERT is unsuitable. The recently launched Vpriv has intensified competition in the market as it has been launched at a much lower price, as much as 15% lower, than the currently marketed products. The players in the Gaucher’s disease therapeutics market are Genzyme Corporation, Shire Pharmaceuticals and Actelion Pharmaceuticals.

Strong Pipeline for the Gaucher’s Disease Therapeutics Market

GlobalData’s analysis shows that the Gaucher’s disease therapeutics pipeline is strong with nine molecules in various Phases of clinical development. One molecule is currently filed with the US Food and Drug Administration (FDA) and European Medicines Agency (EMEA). Two molecules are in Phase III of clinical development, one molecule is in Phase II of clinical development and five molecules are in the preclinical stage. There are no molecules in Phase I. Recently, late stage pipeline molecule Uplyso has been granted Temporary Authorization (ATU) for use by the French Health Products and Safety Agency for the treatment of Gaucher’s patients. The ATU is a special regulatory mechanism which enables the use of non approved products in France when there is a genuine public health need. Eliglustat tartrate is in late Phase III of clinical development and will be the second SRT drug for the treatment of Gaucher’s disease if approved. Another molecule, ISU302, is also in Phase III and will be the first bio-similar in the Gaucher’s disease therapeutics market if approved.

Overall, GlobalData’s analysis suggests that the pipeline for the Gaucher’s disease therapeutics market is strong and that the two promising pipeline candidates have the potential to compete with the currently approved products in addressing unmet market need.

1 Table of Contents
    1.1 List of Tables
    1.2 List of Figures

2 Gaucher’s Disease Therapeutics - Introduction
    2.1 Disease Overview

    2.1.1 Types of Gaucher’s Disease

    2.2 Epidemiology
    2.3 Etiology
    2.4 Pathophysiology
    2.5 Signs and Symptoms

    2.5.1 Type 1 Gaucher’s Disease Clinical Manifestation
    2.5.2 Type 2 Gaucher’s Disease Clinical Manifestation
    2.5.3 Type 3 Gaucher’s Disease Clinical Manifestation

    2.6 Commonly Used Diagnostic Tests

    2.6.1 Commonly Diagnostic Tests

    2.7 Treatment and Management Options for Gaucher’s Disease

    2.7.1 Types of Therapeutics Options

    2.8 GlobalData Pipeline Report Guidance

3 Gaucher’s Disease Therapeutics - Market Characterization
    3.1 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - Global
    3.2 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - Global
    3.3 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - The US
    3.4 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - The US
    3.5 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - France
    3.6 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - France
    3.7 Gaucher’s Disease Therapeutics Market Size (2005-2010) - Germany
    3.8 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - Germany
    3.9 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - Italy
    3.10 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - Italy
    3.11 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - Spain
    3.12 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - Spain
    3.13 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - The UK
    3.14 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - The UK
    3.15 Gaucher’s Disease Therapeutics Market Size (2005 - 2010) - Japan
    3.16 Gaucher’s Disease Therapeutics Market Forecast (2010 - 2018) - Japan
    3.17 Drivers and Barriers for the Gaucher’s Disease Therapeutics Market

    3.17.1 Drivers for the Gaucher’s Disease Therapeutics Market
    3.17.2 Barriers for the Gaucher’s Disease Therapeutics Market

    3.18 Opportunity and Unmet Need Analysis
    3.19 Key Takeaway

4 Gaucher’s Disease Therapeutics - Competitive Assessment
    4.1 Overview
    4.2 Strategic Competitor Assessment
    4.3 Product Profiles for the Marketed Products in the Gaucher’s Disease Therapeutics Market

    4.3.1 Cerezyme (imiglucerase)
    4.3.2 Zavesca (miglustat)
    4.3.3 Vpriv (velaglucerase alfa)

    4.4 Key Takeaway

5 Gaucher's Disease Therapeutics - Pipeline Assessment
    5.1 Overview
    5.2 Strategic Pipeline Assessment
    5.3 Gaucher’s Disease Therapeutics Pipeline - Pipeline by Phases of Development

    5.3.1 Gaucher’s Disease Therapeutics - Regulatory Filed and Phase III Clinical Pipeline
    5.3.2 Gaucher’s Disease Therapeutics - Phase II Pipeline
    5.3.3 Gaucher’s Disease Therapeutics - Preclinical

    5.4 Technology Trends Analytic Framework
    5.5 Gaucher's Disease Therapeutics Market - Pipeline by Mechanism of Action
    5.6 Gaucher's Disease Therapeutics - Promising Drugs in Clinical Development
    5.7 Molecule Profile for Promising Drugs in Clinical Development

    5.7.1 Uplyso (taliglucerase alfa)
    5.7.2 Eliglustat tartrate(Genz-112638)
    5.7.3 ISU302

    5.8 Key Takeaway

6 Gaucher’s Disease Therapeutics - Clinical Trials Mapping
    6.1 Clinical Trials by Region and Country
    6.2 Clinical Trials by Phase
    6.3 Clinical Trials by Trial Status
    6.4 Clinical Trials Mapping by Sponsors
    6.5 Prominent Sponsors
    6.6 Top Companies Participating in Gaucher’s Disease Therapeutics Clinical Trials

7 Gaucher’s Disease Therapeutics - Strategic Assessment
    7.1 Key Events Impacting the Future Market
    7.2 Gaucher’s Disease Therapeutics - Implications for Future Market Competition

8 Gaucher’s Disease Therapeutics Market - Future Players
    8.1 Introduction
    8.2 Company Profiles

    8.2.1 Pfizer Inc.
    8.2.2 Protalix BioTherapeutics, Inc.
    8.2.3 ISU ABXIS Co., Limited
    8.2.4 Sanofi
    8.2.5 Actelion Limited
    8.2.6 Shire Plc

9 Gaucher’s Disease Therapeutics - Licensing and Partnership Deals

10 Gaucher’s Disease Therapeutics - Appendix
    10.1 Market Definitions
    10.2 Acronyms
    10.3 Research Methodology

    10.3.1 Coverage
    10.3.2 Secondary Research
    10.3.3 Forecasting
    10.3.4 Primary Research
    10.3.5 Expert Panel Validation

    10.4 Contact Us
    10.5 Disclaimer
    10.6 Bibliography

1.1 List of Tables
    Table 1: Clinical Subtypes and Manifestations of Gaucher’s Disease
    Table 2: Clinical Manifestations of Gaucher’s Disease
    Table 3: Gaucher’s Disease Therapeutics Market, Global, Revenue ($m), 2005 - 2010
    Table 4: Gaucher’s Disease Therapeutics Market, Global, Forecast ($m), 2010 - 2018
    Table 5: Gaucher’s Disease Therapeutics Market, The US, Revenue ($m), 2005 - 2010
    Table 6: Gaucher’s Disease Therapeutics Market, The US, Forecasts ($m), 2010 - 2018
    Table 7: Gaucher’s Disease Therapeutics Market, France, Revenue ($m), 2005 - 2010
    Table 8: Gaucher’s Disease Therapeutics Market, France, Forecasts ($m), 2010 - 2018
    Table 9: Gaucher’s Disease Therapeutics Market, Germany, Revenue ($m), 2005 - 2010
    Table 10: Gaucher’s Disease Therapeutics Market, Germany, Forecasts ($m), 2010 - 2018
    Table 11: Gaucher’s Disease Therapeutics Market, Italy, Revenue ($m), 2005 - 2010
    Table 12: Gaucher’s Disease Therapeutics Market, Italy, Forecasts ($m), 2010 - 2018
    Table 13: Gaucher’s Disease Therapeutics Market, Spain, Revenue ($m), 2005 - 2010
    Table 14: Gaucher’s Disease Therapeutics Market, Spain, Forecasts ($m), 2010 - 2018
    Table 15: Gaucher’s Disease Therapeutics Market, The UK, Revenue ($m), 2005 - 2010
    Table 16: Gaucher’s Disease Therapeutics Market, The UK, Forecasts ($m), 2010 - 2018
    Table 17: Gaucher’s Disease Therapeutics Market, Japan, Revenue ($m), 2005 - 2010
    Table 18: Gaucher’s Disease Therapeutics Market, Japan, Forecasts ($m), 2010 - 2018
    Table 19: Major Marketed Products in Gaucher’s Disease Therapeutics Market
    Table 20: Gaucher’s Disease Therapeutics - Regulatory Filed and Phase III Pipeline, 2011
    Table 21: Gaucher’s Disease Therapeutics - Phase II Pipeline, 2011
    Table 22: Gaucher’s Disease Therapeutics - Preclinical Phase Pipeline, 2011
    Table 23: Gaucher’s Disease Therapeutics - Most Promising Drug in Clinical Development, 2011
    Table 24: Gaucher’s Disease Therapeutics - Clinical Trials by Country, 2011
    Table 25: Gaucher’s Disease Therapeutics - Clinical Trials by Phase, 2011
    Table 26: Gaucher’s Disease Therapeutics - Clinical Trials by Status, 2011
    Table 27: Gaucher’s Disease Therapeutics - Overall Sponsors, 2011
    Table 28: Gaucher’s Disease Therapeutics - Prominent Sponsors, 2011
    Table 29: Gaucher’s Disease Therapeutics - Top Companies Participating in Therapeutics Clinical Trials, 2011
    Table 30: Pfizer Inc., Pipeline - 2011
    Table 31: Protalix BioTherapeutics, Inc., Pipeline - 2011
    Table 32: ISU ABXIS Co., Ltd., Pipeline - 2011
    Table 33: Gaucher’s Disease - Global, Deals, 2011

1.2 List of Figures
    Figure 1: Gaucher’s Disease Therapeutics Market, Global, Revenue ($m), 2005 - 2010
    Figure 2: Gaucher’s Disease Therapeutics Market, Global, Forecast ($m), 2010 - 2018
    Figure 3: Gaucher’s Disease Therapeutics Market, The US, Forecast ($m), 2005 - 2010
    Figure 4: Gaucher’s Disease Therapeutics Market, The US, Forecasts ($m), 2010 - 2018
    Figure 5: Gaucher’s Disease Therapeutics Market, France, Revenue ($m), 2005 - 2010
    Figure 6: Gaucher’s Disease Therapeutics Market, France, Forecast ($m), 2010 - 2018
    Figure 7: Gaucher’s Disease Therapeutics Market, Germany, Revenue ($m), 2005 - 2010
    Figure 8: Gaucher’s Disease Therapeutics Market, Germany, Forecast ($m), 2010 - 2018
    Figure 9: Gaucher’s Disease Therapeutics Market, Italy, Revenue ($m), 2005 - 2010
    Figure 10: Gaucher’s Disease Therapeutics Market, Italy, Forecast ($m), 2010 - 2018
    Figure 11: Gaucher’s Disease Therapeutics Market, Spain, Revenue ($m), 2005 - 2010
    Figure 12: Gaucher’s Disease Therapeutics Market, Spain, Forecast ($m), 2010 - 2018
    Figure 13: Gaucher’s Disease Therapeutics Market, The UK, Revenue ($m), 2005 - 2010
    Figure 14: Gaucher’s Disease Therapeutics Market, The UK, Forecast ($m), 2010 - 2018
    Figure 15: Gaucher’s Disease Therapeutics Market, Japan, Revenue ($m), 2005 - 2010
    Figure 16: Gaucher’s Disease Therapeutics Market, Japan, Forecasts ($m), 2010 - 2018
    Figure 17: Opportunity and Unmet Need in the Gaucher’s Disease Therapeutics Market
    Figure 18: Gaucher’s Disease Therapeutics - Strategic Competitor Assessment, 2011
    Figure 19: Gaucher’s Disease Therapeutics - Pipeline by Phase of Development, 2011
    Figure 20: Gaucher’s Disease Therapeutics - Technology Trends Analytics Framework, 2011
    Figure 21: Gaucher’s Disease Therapeutics - Technology Trends Analytics Framework - Description, 2011
    Figure 22: Gaucher's Disease Therapeutics - Pipeline by Mechanism of Action, 2011
    Figure 23: Gaucher’s Disease Therapeutics - Clinical Trials by Country, 2011
    Figure 24: Gaucher’s Disease Therapeutics - Clinical Trials by Phase, 2011
    Figure 25: Gaucher’s Disease Therapeutics - Clinical Trials by Status, 2011
    Figure 26: Gaucher’s Disease Therapeutics - Overall Sponsors, 2011
    Figure 27: Gaucher’s Disease Therapeutics - Prominent Sponsors, 2011
    Figure 28: Gaucher’s Disease Therapeutics - Top Companies Participating in Therapeutics Clinical Trials, 2011
    Figure 29: Gaucher’s Disease Therapeutics Market, Drivers and Barriers, 2011
    Figure 30: Implications for Future Market Competition in the Gaucher’s Disease Therapeutics Market, 2011
    Figure 31: Gaucher’s Disease Therapeutics Market - Clinical Pipeline by Company, 2011
    Figure 32: GlobalData Market Forecasting Model