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Phenylketonuria Therapeutics - Pipeline Assessment and Market Forecasts to 2019

Abstract

Phenylketonuria Therapeutics - Pipeline Assessment and Market Forecasts to 2019

Summary

GlobalData, the industry analysis specialist, has released its new report, "Phenylketonuria Therapeutics - Pipeline Assessment and Market Forecasts to 2019". The report is an essential source of information and analysis on the global Phenylketonuria Therapeutics market. The report identifies the key trends shaping and driving the global Phenylketonuria 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 Phenylketonuria 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 Phenylketonuria Therapeutics market. Its scope includes -

  • Annualized seven key markets (US, France, Germany, Italy, Spain, UK and Japan) Phenylketonuria Therapeutics market revenues data from 2006 to 2011, forecast for eight years to 2019.
  • 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 Phenylketonuria 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 Phenylketonuria Therapeutics market.
  • Analysis of key recent licensing and partnership agreements in Phenylketonuria 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 Phenylketonuria Therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global Phenylketonuria 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 Phenylketonuria Therapeutics market landscape? - Identify, understand and capitalize.

TOC

1 Table of Contents

1 Table of Contents 2

  • 1.1 List of Tables 4
  • 1.2 List of Figures 5

2 Phenylketonuria Therapeutics - Introduction 6

  • 2.1 Overview 6
  • 2.2 Epidemiology 8
  • 2.3 Etiology 10
  • 2.4 Signs and Symptoms 12
  • 2.5 Diagnosis 13
  • 2.6 Treatment 14
  • 2.7 Management 15
  • 2.8 Referral Pathway for Phenylketonuria 16
  • 2.9 GlobalData Pipeline Report Guidance 18

3 Phenylketonuria Therapeutics - Market Characterization 19

  • 3.1 Phenylketonuria Therapeutics Market Size (2007-2011) - Global 19
  • 3.2 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Global 21
  • 3.3 Phenylketonuria Therapeutics Market Size (2007-2011) - The US 23
  • 3.4 Phenylketonuria Therapeutics Market Forecast (2011-2019) - The US 24
  • 3.5 Phenylketonuria Therapeutics Market Size (2008-2011) - The UK 25
  • 3.6 Phenylketonuria Therapeutics Market Forecast (2011-2019) - The UK 26
  • 3.7 Phenylketonuria Therapeutics Market Size (2008-2011) - France 27
  • 3.8 Phenylketonuria Therapeutics Market Forecast (2011-2019) - France 28
  • 3.9 Phenylketonuria Therapeutics Market Size (2008-2011) - Germany 29
  • 3.10 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Germany 30
  • 3.11 Phenylketonuria Therapeutics Market Size (2008-2011) - Italy 31
  • 3.12 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Italy 32
  • 3.13 Phenylketonuria Therapeutics Market Size (2008-2011) - Spain 33
  • 3.14 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Spain 34
  • 3.15 Phenylketonuria Therapeutics Market Size (2008-2011) - Japan 35
  • 3.16 Phenylketonuria Therapeutics Market Forecast (2011-2019) - Japan 36
  • 3.17 Drivers and Barriers for the Phenylketonuria Therapeutics Market 37
    • 3.17.1 Drivers for the Phenylketonuria Therapeutics Market 37
    • 3.17.2 Barriers for the Phenylketonuria Therapeutics Market 38
  • 3.18 Key Events Impacting the Future Market 38
  • 3.19 Opportunity and Unmet Need 39
    • 3.19.1 Opportunity and Unmet Need for the Phenylketonuria Therapeutics Market 39
  • 3.20 Key Takeaway 40

4 Phenylketonuria Therapeutics Market - Competitive Assessment 41

  • 4.1 Overview 41
    • 4.1.1 Strategic Competitive Assessment 41
  • 4.2 Product Profiles of the Marketed Products in the Phenylketonuria Market 42
    • 4.2.1 Kuvan (sapropterin dihydrochloride) 42
  • 4.3 Key Takeaway 44

5 Phenylketonuria Therapeutics Market - Pipeline Assessment 45

  • 5.1 Overview 45
  • 5.2 Strategic Pipeline Assessment 45
    • 5.2.1 Phenylketonuria Therapeutics - Phase II Pipeline 45
  • 5.3 Phenylketonuria Pipeline by Mechanism of Action 45
  • 5.4 Phenylketonuria Technology Trends Analytical Framework 46
  • 5.5 Phenylketonuria Therapeutics - Drugs under Clinical Development 46
  • 5.6 Profile of Drugs under Clinical Development 47
    • 5.6.1 PEG-PAL 47
  • 5.7 Key Takeaway 48

6 Phenylketonuria Therapeutics - Clinical Trials Mapping 49

  • 6.1 Clinical Trials Mapping by Country (The US, EU5 and Japan) 49
  • 6.2 Clinical Trials Mapping by Phase 50
  • 6.3 Clinical Trials Mapping by Trial Status 51
  • 6.4 Clinical Trials Mapping by Overall Sponsors 52
  • 6.5 Clinical Trial Mapping by Prominent Sponsors 53
  • 6.6 Top Companies Participating in Phenylketonuria Therapeutics Clinical Trials 54

7 Phenylketonuria Therapeutics - Strategic Assessment 55

  • 7.1 Future Market Competition Scenario 55
  • 7.2 Key Takeaway 55

8 Global Phenylketonuria Therapeutics - Future Players in The Market 56

  • 8.1 Introduction 56
  • 8.2 Company Profile 56
    • 8.2.1 BioMarin Pharmaceuticals Inc. 56

9 Phenylketonuria Therapeutics - Licensing and Partnership Deals 58

10 Phenylketonuria Therapeutics - Appendix 59

  • 10.1 Acronyms 59
  • 10.2 Bibliography 60
  • 10.3 Research Methodology 61
    • 10.3.1 Coverage 61
    • 10.3.2 Secondary Research 61
    • 10.3.3 Primary Research 62
    • 10.3.4 Forecasting 62
    • 10.3.5 Expert Panel Validation 64
  • 10.4 Contact Us 64
  • 10.5 Disclaimer 65

List of Tables

1.1 List of Tables

  • Table 1: Biochemical Basis for Phenylketonuria 7
  • Table 2: Historical Prevalence for Phenylketonuria for Seven Major Markets, 2006-2011 8
  • Table 3: Forecasted Prevalence for Phenylketonuria for Seven Major Markets, 2011-2019 9
  • Table 4: Characteristics of Therapeutics for BH4 responsive phenylketonuria patients 14
  • Table 5: Phenylketonuria Therapeutics Market, Seven Major Markets, Revenue ($m), 2007-2011 19
  • Table 6: Phenylketonuria Therapeutics Market, Seven Major Markets, Market Forecast ($m), 2011-2019 22
  • Table 7: Phenylketonuria Therapeutics Market, The US, Revenue ($m), 2007-2011 23
  • Table 8: Phenylketonuria Therapeutics Market, The US, Market Forecast ($m), 2011-2019 24
  • Table 9: Phenylketonuria Therapeutics Market, The UK, Revenue ($m), 2008-2011 25
  • Table 10: Phenylketonuria Therapeutics Market, The UK, Market Forecast ($m), 2011-2019 26
  • Table 11: Phenylketonuria Therapeutics Market, France, Revenue ($m), 2008-2011 27
  • Table 12: Phenylketonuria Therapeutics Market, France, Market Forecast ($m), 2011-2019 28
  • Table 13: Phenylketonuria Therapeutics Market,Germany, Revenue ($m), 2008-2011 29
  • Table 14: Phenylketonuria Therapeutics Market, Germany, Market Forecast ($m), 2011-2019 30
  • Table 15: Phenylketonuria Therapeutics Market, Italy, Revenue ($m), 2008-2011 31
  • Table 16: Phenylketonuria Therapeutics Market, Italy, Market Forecast ($m), 2011-2019 32
  • Table 17: Phenylketonuria Therapeutics Market, Spain, Revenue ($m), 2008-2011 33
  • Table 18: Phenylketonuria Therapeutics Market, Spain, Market Forecast ($m), 2011-2019 34
  • Table 19: Phenylketonuria Therapeutics Market, Japan, Revenue ($m), 2008-2011 35
  • Table 20: Phenylketonuria Therapeutics Market, Japan, Market Forecast ($m), 2011-2019 36
  • Table 21: Phenylketonuria - Phase II Pipeline 45
  • Table 22: Phenylketonuria Therapeutics - Promising Drugs Under Clinical Development, 2012 46
  • Table 23: Phenylketonuria Therapeutics - Clinical Trials by Country, 2012 49
  • Table 24: Phenylketonuria Therapeutics - Clinical Trials by Phase, 2012 50
  • Table 25: Phenylketonuria Therapeutics - Clinical Trials by Status, 2012 51
  • Table 26: Phenylketonuria Therapeutics - Clinical Trials by Overall Sponsors, 2012 52
  • Table 27: Phenylketonuria Therapeutics - Clinical trials by Prominent Sponsors, 2012 53
  • Table 28: Phenylketonuria Therapeutics - Clinical Trials by Top Companies Participating in Therapeutics Clinical Trials, 2012 54
  • Table 29: Phenylketonuria Therapeutics, Clinical Pipeline by Company, 2012 56
  • Table 30: BioMarin Pharmaceuticals Inc. - Promising Molecules in the Pipeline for Phenylketonuria, 2012 57
  • Table 31: PhenylketonuriaTherapeutics, Global, Deals ($m), 2012 58

List of Figures

1.2 List of Figures

  • Figure 1: Factors Modulating Phenylketonuria 7
  • Figure 2: Patient Share of Phenylketonuria in the Seven Major Markets, 2011 9
  • Figure 3: Mechanism of Formation of Phenylketonuria 10
  • Figure 4: The Basic Structure of the Human PAH Gene 11
  • Figure 5: Allelic Mutations Associated with Phenylketonuria 12
  • Figure 6: Treatment Algorithm for Phenylketonuria 15
  • Figure 7: Referral Pathway for Phenylketonuria 17
  • Figure 8: Phenylketonuria Therapeutics Market, Seven Major Markets, Revenue ($m), 2007-2011 19
  • Figure 9: Phenylketonuria Therapeutics Market, Market Shares (%), 2011 20
  • Figure 10: Phenylketonuria Therapeutics Market, Seven Major Markets, Forecast ($m), 2011-2019 21
  • Figure 11: Phenylketonuria Therapeutics, Market Share (%), 2019 22
  • Figure 12: Phenylketonuria Therapeutics Market, The US, Revenue ($m), 2007-2011 23
  • Figure 13: Phenylketonuria Therapeutics Market, The US, Market Forecast ($m), 2011-2019 24
  • Figure 14: Phenylketonuria Therapeutics Market, The UK, Revenue ($m), 2008-2011 25
  • Figure 15: Phenylketonuria Therapeutics Market, The UK, Market Forecast ($m), 2011-2019 26
  • Figure 16: Phenylketonuria Therapeutics Market, France, Revenue ($m), 2008-2011 27
  • Figure 17: Phenylketonuria Therapeutics Market, France, Market Forecast ($m), 2011-2019 28
  • Figure 18: Phenylketonuria Therapeutics Market, Germany, Revenue ($m), 2008-2011 29
  • Figure 19: Phenylketonuria Therapeutics Market, Germany, Market Forecast ($m), 2011-2019 30
  • Figure 20: Phenylketonuria Therapeutics Market, Italy, Revenue ($m), 2008-2011 31
  • Figure 21: Phenylketonuria Therapeutics Market, Italy, Market Forecast ($m), 2011-2019 32
  • Figure 22: Phenylketonuria Therapeutics Market, Spain, Revenue ($m), 2008-2011 33
  • Figure 23: Phenylketonuria Therapeutics Market, Spain, Market Forecast ($m), 2011-2019 34
  • Figure 24: Phenylketonuria Therapeutics Market, Japan, Revenue ($m), 2008-2011 35
  • Figure 25: Phenylketonuria Therapeutics Market, Japan, Market Forecast ($m), 2011-2019 36
  • Figure 26: Phenylketonuria Therapeutics, Key Events Impacting Future Market, 2012 38
  • Figure 27: Opportunity and Unmet Need in the Phenylketonuria Therapeutics Market, 2012 40
  • Figure 28: Phenylketonuria Market - Strategic Competitor Assessment, 2012 41
  • Figure 29: Mechanism of Action of Kuvan 42
  • Figure 30: Technology Trends Analytical Framework of the Phenylketonuria Pipeline - Description, 2012 46
  • Figure 31: Phenylketonuria Therapeutics - Clinical Trials by Country, 2012 49
  • Figure 32: Phenylketonuria Therapeutics - Clinical Trials by Phase, 2012 50
  • Figure 33: Phenylketonuria Therapeutics - Clinical Trials by Status, 2012 51
  • Figure 34: Phenylketonuria Therapeutics - Clinical Trials by Overall Sponsors, 2012 52
  • Figure 35: Phenylketonuria Therapeutics - Clinical Trials by Prominent Sponsors, 2012 53
  • Figure 36: Phenylketonuria Therapeutics Clinical Trials - Company Sponsors by Phase, 2012 54
  • Figure 37: Phenylketonuria Therapeutics, Strategic Assessment, 2012 55
  • Figure 38: GlobalData market Forecasting Model, 2012 64
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