Aarkstore - Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics

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AArkstore enterprise 17th Novenber 2014

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics - Pipeline Assessment and Market Forecasts to 2019 Browse Full Report @ http://www.aarkstore.com/pharmaceuticals-healthcare/60331/paroxysmal-nocturnalhemoglobinuria-pnh-therapeutics-pipeline-assessment-and-market-forecasts Published: Feb 2012 | No. Of Pages: 62 Pages PDF: $ 3995 | Site Licence : $ 7990 | Enterprise Wide Licence : $ 11985

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics - Pipeline Assessment and Market Forecasts to 2019

Summary the industry analysis specialist, has released its new report, Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics - Pipeline Assessment and Market Forecasts to 2019. The report is an essential source of information and analysis on the global Paroxysmal Nocturnal Hemoglobinuria Therapeutics market. The report identifies the key trends shaping and driving the global Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria Therapeutics sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GlobalDatas team of industry experts.

Scope - Annualized seven key markets (US, France, Germany, Italy, Spain, UK and Japan) Paroxysmal Nocturnal Hemoglobinuria Therapeutics market revenues data from 2006 to 2011, forecast for eight years to 2019.

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AArkstore enterprise - 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 Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria Therapeutics market. - Analysis of key recent licensing and partnership agreements in Paroxysmal Nocturnal Hemoglobinuria Therapeutics market

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. - Develop business strategies by understanding the trends shaping and driving the global Paroxysmal Nocturnal Hemoglobinuria Therapeutics market. - Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global Paroxysmal Nocturnal Hemoglobinuria 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. - Whats the next big thing in the global Paroxysmal Nocturnal Hemoglobinuria Therapeutics market landscape? Identify, understand and capitalize.

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Tables Of Contents 2 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Introduction 6 2.1 Overview 6 2.2 Classification of PNH 7 2.2.1 Classic PNH 7 2.2.2 PNH in the setting of another specified bone marrow disorder 7 2.2.3 Subclinical PNH (PNH-sc) 7 2.3 Epidemiology 8 2.4 Etiology and Pathophysiology 8 2.5 Risk Factors 10 2.5.1 Chemicals 10 2.5.2 Other Risk Factors 10 2.6 Symptoms 10 2.7 Diagnosis of PNH 11 2.7.1 Complete Blood Count (CBC) 12 2.7.2 Reticulocyte Count 12 2.7.3 Lactate Dehydrogenase Test (LDH) 12 2.7.4 Bilirubin Test 12 2.7.5 Ferritin Test 12 2.7.6 Haptoglobin Test 12 2.7.7 Urinalysis 12

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AArkstore enterprise 2.7.8 Urine Test for Hemosiderin 12 2.7.9 Bone Marrow Examination 13 2.7.10 Sucrose Hemolysis Test 13 2.7.11 Hams Test 13 2.7.12 Flow Cytometry 13 2.7.13 Mutational analysis 14 2.8 Treatment 15 2.8.1 Treatment of PNH Patients Associated with Hemolysis 15 2.8.2 Treatment of PNH patients associated with Thrombosis 17 2.8.3 Treatment for PNH Patients Associated with Non- Hemolytic Anemia 17 2.8.4 Allogeneic Stem Cell Transplant (SCT)/Bone Marrow Transplant (BMT) 18 2.8.5 Treatment of PNH patients in Pregnancy 18 2.8.6 Treatment of Pediatric PNH patients 18 2.9 GlobalData Pipeline Report Guidance 19 3 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Market Characterization 20 3.1 PNH Therapeutics Market Size (2006-2011) - Global 20 3.2 PNH Therapeutics Market Forecast (2011-2019) - Global 22 3.3 PNH Therapeutics Market Size (2006-2011) - The US 24 3.4 PNH Therapeutics Market Forecast (2011-2019) - The US 25 3.5 PNH Therapeutics Market Size (2006-2011) - The UK 26 3.6 PNH Therapeutics Market Forecast (2011-2019) - The UK 27 3.7 PNH Therapeutics Market Size (2006-2011) - France 28 3.8 PNH Therapeutics Market Forecast (2011-2019) - France 29

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AArkstore enterprise 3.9 PNH Therapeutics Market Size (2006-2011) - Germany 30 3.10 PNH Therapeutics Market Forecast (2011-2019) - Germany 31 3.11 PNH Therapeutics Market Size (2006-2011) - Italy 32 3.12 PNH Therapeutics Market Forecast (2011-2019) - Italy 33 3.13 PNH Therapeutics Market Size (2006-2011) - Spain 34 3.14 PNH Therapeutics Market Forecast (2011-2019) - Spain 35 3.15 PNH Market Size (2006-2011) - Japan 36 3.16 PNH Therapeutics Market Forecast (2011-2019) - Japan 37 3.17 Drivers and Barriers for the PNH Market 38 3.17.1 Drivers for the PNH Market 38 3.17.2 Barriers for the PNH Market 38 3.18 Opportunity and Unmet Need 39 3.19 Key Takeaway 40 4 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Competitive Assessment 41 4.1 Overview 41 4.1.1 Strategic Competitor Assessment 41 4.2 Marketed Product Profile 42 4.2.1 Soliris (eculizumab) 42 4.3 Key Takeaway 43 5 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Pipeline Assessment 44 5.1 Overview 44 5.2 Strategic Pipeline Assessment 44 5.3 PNH Therapeutics Pipeline Analysis - Pipeline by Clinical Phases of Development 44

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AArkstore enterprise 5.3.1 PNH Therapeutics - Phase II Clinical Pipeline 45 5.3.2 PNH Therapeutics - Phase I Clinical Pipeline 45 5.3.3 PNH Therapeutics - Preclinical Phase 45 5.4 PNH Therapeutics Pipeline Analysis: Pipeline by Mechanism of Action 46 5.5 PNH Technology Trends Analytical Framework 47 5.6 Profile of Late Stage Molecule under Clinical Development 48 5.6.1 Alemtuzumab 48 5.7 Key Takeaway 48 6 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Clinical Trials Mapping 49 6.1 Clinical Trial Mapping by Country (US, EU5 and Japan) 49 6.2 Clinical Trial Mapping by Phase 50 6.3 Clinical Trial Mapping by Trial Status 51 6.4 Clinical Trial Mapping Overall Sponsors 52 6.5 Clinical Trial Mapping by Prominent Sponsors 53 7 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Strategic Assessment 54 7.1 PNH Therapeutics - Implications for Future Market Competition 54 8 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Future Players in the Market 55 8.1 Introduction 55 8.2 Company Profiles 55 8.2.1 Alexion Pharmaceuticals, Inc. 55 9 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Appendix 56 9.1 Definitions 56 9.2 Acronyms 56

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AArkstore enterprise 9.3 Research Methodology 57 9.3.1 Coverage 57 9.3.2 Secondary Research 58 9.3.3 Forecasting 58 9.3.4 Primary Research 60 9.3.5 Expert Panel Validation 61 9.4 Contact Us 61 9.5 Disclaimer 61 9.6 Bibliography 61

List Of Tables Table 1: Classification of PNH 7 Table 2: PNH Therapeutics Market, Global, Revenue ($m), 2006-2011 20 Table 3: PNH Therapeutics Market, Global Forecast ($m), 2011-2019 22 Table 4: PNH Therapeutics Market, The US, Revenue ($m), 2006-2011 24 Table 5: PNH Therapeutics Market, The US, Forecast ($m), 2011-2019 25 Table 6: PNH Therapeutics Market, The UK, Revenue ($m), 2006-2011 26 Table 7: PNH Therapeutics Market, The UK, Forecast ($m), 2011-2019 27 Table 8: PNH Therapeutics Market, France, Revenue ($m), 2006-2011 28 Table 9: PNH Therapeutics Market, France, Forecast ($m), 2011-2019 29 Table 10: PNH Therapeutics Market, Germany, Revenue ($m), 2006-2011 30 Table 11: PNH Therapeutics Market, Germany, Forecast ($m), 2011-2019 31 Table 12: PNH Therapeutics Market, Italy, Revenue ($m), 2006-2011 32

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AArkstore enterprise Table 13: PNH Therapeutics Market, Italy, Forecast ($m), 2011-2019 33 Table 14: PNH Therapeutics Market, Spain, Revenue ($m), 2006-2011 34 Table 15: PNH Therapeutics Market, Spain, Forecast ($m), 2011-2019 35 Table 16: PNH Therapeutics Market, Japan, Revenue ($m), 2006-2011 36 Table 17: PNH Therapeutics Market, Japan, Forecast ($m), 2011-2019 37 Table 18: PNH Therapeutics - Phase II Pipeline 45 Table 19: PNH Therapeutics - Phase I Pipeline 45 Table 20: PNH Therapeutics - Preclinical Phase 45 Table 21: PNH Therapeutics - Clinical Trials by Country, 2011 49 Table 22: PNH Therapeutics - Clinical Trials by Phase, 2011 50 Table 23: PNH Therapeutics - Clinical Trials by Status, 2011 51 Table 24: PNH Therapeutics, Overall Sponsors, 2011 52 Table 25: PNH Therapeutics - Prominent Sponsors, 2011 53 Table 26: Alexion Pharmaceuticals, Inc. - PNH Pipeline 55

List Of Figures Figure 1: Development of PNH 6 Figure 2: Pathophysiology of PNH 9 Figure 3: Consequences of Nitric Oxide Depletion by Intravascular Hemolysis 9 Figure 4: Diagnostic Pathway for PNH 11 Figure 5: PNH Diagnosis by Flow Cytometry - RBC Analysis 13 Figure 6: PNH Diagnosis by Flow Cytometry - Leukocyte Analysis 14 Figure 7: Treatment and Disease Management of PNH patients 16

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AArkstore enterprise Figure 8: PNH Therapeutics Market, Global, Revenue ($m), 2006-2011 20 Figure 9: PNH Therapeutics, Global Market Share ($m), 2011 21 Figure 10: PNH Therapeutics Market, Global, Forecast ($m), 2011-2019 22 Figure 11: PNH Therapeutics, Global Market Share ($m), 2019 23 Figure 12: PNH Therapeutics Market, The US, Revenue ($m), 2006-2011 24 Figure 13: PNH Therapeutics Market, The US, Forecast ($m), 2011-2019 25 Figure 14: PNH Therapeutics Market, The UK, Revenue ($m), 2006-2011 26 Figure 15: PNH Therapeutics Market, The UK, Forecast ($m), 2011-2019 27 Figure 16: PNH Therapeutics Market, France, Revenue ($m), 2006-2011 28 Figure 17: PNH Therapeutics Market, France, Forecast ($m), 2011-2019 29 Figure 18: PNH Therapeutics Market, Germany, Revenue ($m), 2006-2011 30 Figure 19: PNH Therapeutics Market, Germany, Forecast ($m), 2011-2019 31 Figure 20: PNH Therapeutics Market, Italy, Revenue ($m), 2006-2011 32 Figure 21: PNH Therapeutics Market, Italy, Forecast ($m), 2011-2019 33 Figure 22: PNH Therapeutics Market, Spain, Revenue ($m), 2006-2011 34 Figure 23: PNH Therapeutics Market, Spain, Forecast ($m), 2011-2019 35 Figure 24: PNH Therapeutics Market, Japan, Revenue ($m), 2006-2011 36 Figure 25: PNH Therapeutics Market, Japan, Forecast ($m), 2011-2019 37 Figure 26: Drivers and Barriers for PNH Therapeutics, 2011 39 Figure 27: Opportunity and Unmet Need in the Hemolytic PNH Market, 2011 40 Figure 28: PNH Therapeutics Market - Strategic Competitor Assessment, 2011 41 Figure 29: PNH Therapeutics - Pipeline by Phase of Development, 2011 44 Figure 30: PNH Market - Clinical Pipeline by Mechanism of Action, 2011 46

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AArkstore enterprise Figure 31: Technology Trends Analytical Framework of the PNH Pipeline, 2011 47 Figure 32: Technology Trends Analytical Framework of the PNH Pipeline, 2011 47 Figure 33: PNH Therapeutics - Clinical Trials by Country, 2011 49 Figure 34: PNH Therapeutics - Clinical Trials by Phase, 2011 50 Figure 35: PNH Therapeutics - Clinical Trials by Status, 2011 51 Figure 36: PNH Therapeutics - Overall Sponsors, 2011 52 Figure 37: PNH Therapeutics - Prominent Sponsors, 2011 53 Figure 38: Implications for Future Market Competition in the PNH Therapeutics Market, 2011 54 Figure 39: GlobalData Market Forecasting Model 60

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AArkstore enterprise About Aarkstore Enterprise Aarkstore Enterprise is a leading provider of business and financial information and solutions worldwide. We specialize in providing online market business information on market research reports, books, magazines, conference at competitive prices, and strive to provide excellent and innovative service to our customers.

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