Hemophilia - Drug Pipeline Analysis and Market Forecasts to 2016

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Hemophilia - Drug Pipeline Analysis and Market Forecasts to 2016
Published on May 2010

Report Summary


Summary

GlobalData's pharmaceutical and healthcare report, 'Hemophilia ' Drug Pipeline Analysis and Market Forecasts to 2016'. The report is
an essential source of information and analysis on the global hemophilia market. The report identifies the key trends shaping and
driving the global hemophilia market. The report also provides insights on the prevalent competitive landscape and the emerging
players expected to significantly alter the market positioning of the existing market leaders. Most importantly, the report provides
valuable insights on the pipeline products within the global hemophilia 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.

GlobalData's analysis suggests that the global hemophilia market was valued at $6.5 billion in 2009. This slow growth is primarily
attributed to a low diagnosis rate and the high price of therapy. The safety and efficacy profiles of the existing therapies are moderate.
The major treatments for hemophilia A include recombinant factor VIII drugs such as NovoSeven, Kogenate, Advate, ReFacto,
Xyntha, Feiba and others for severe hemophilia cases. For mild to moderate hemophilia cases, DDAVP (desmopressin), which
stimulates the release of factor VIII and also increases the level of proteins in the blood, is used. For hemophilia B, the most widely
used branded drug is BeneFIX from Baxter Laboratories. BeneFIX is a clotting factor IX.

Scope

The Scope of the report includes:
- Annualized global hemophilia market revenues data from 2001 to 2009, forecast for seven years to 2016.
- Geographies covered in this report include the US (United States), the UK (United Kingdom), Italy, Spain, Germany, France and
Japan.
- Pipeline analysis data providing a split across different phases by the mechanism of action being developed and the emerging
trends. The key classes of mechanism of action include substitution of coagulation factor VIII, increasing clotting factor VIII,
plasma-derived FVIII.
- Analysis of the current and future market competition in the global hemophilia market. The key market players covered are PTC
Therapeutics, Recoly N.V., Novo Nordisk.
- 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 a strategic competitor assessment, market characterization, unmet needs and the implications for the
hemophilia market.

Reasons to buy

The report will enhance your decision making capability in a more rapid and time sensitive manner. It will allow you to:
- Develop and design your in-licensing and out-licensing strategies through review of pipeline products and technologies and by

Hemophilia - Drug Pipeline Analysis and Market Forecasts to 2016 Page 1/6


identifying companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global haemophilia market.
- Drive revenues by understanding key trends, innovative products and technologies, market segments and companies likely to
impact the global haemophilia 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 portfolio and create effective counter-strategies to gain 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 haemophilia market landscape' ' Identify, understand and capitalize.

Table of Content

1 Table of contents 2
1.1 List of Tables 4
1.2 List of Figures 4
2 Hemophilia Market: Market Characterization 5
2.1 Overview 5
2.2 Hemophilia Therapeutics Market Size 5
2.3 Hemophilia Therapeutics Market Forecast and CAGR 6
2.4 Drivers and Barriers for the Hemophilia Therapeutics Market 7
2.4.1 Drivers for the Hemophilia Therapeutics Market 7
2.4.2 Barriers for the Hemophilia Therapeutics Market 8
2.5 Opportunity and Unmet Need 8
2.6 Key Takeaway 9
3 Hemophilia Therapeutics Market: Competitive Assessment 10
3.1 Overview 10
3.2 Strategic Competitor Assessment 10
3.3 Product Profile for the Major Marketed Products in the Hemophilia Therapeutics Market 11
3.3.1 NovoSeven 11
3.3.2 Kogenate FS 11
3.3.3 Xyntha 12
3.3.4 Advate 13
3.4 Key Takeaway 14
4 Hemophilia Therapeutics Market: Pipeline Assessment 15
4.1 Overview 15
4.2 Strategic Pipeline Assessment 15
4.2.1 Technology Trends Analytic Framework 15
4.3 Hemophilia Therapeutics ' Promising Drugs under Clinical Development 16
4.4 Molecule Profile for Promising Drugs under Clinical Development 16
4.4.1 Ataluren (PTC124) 16
4.4.2 NecLip-rFVIIa (LongSeven) 17
4.4.3 rFVIII (Recombinant factor VIIa (N8)) 18
4.5 Hemophilia Therapeutics Market ' Clinical Pipeline by Mechanism of Action 18
4.6 Hemophilia Therapeutics Pipeline ' Pipeline by Clinical Phase of Development 19
4.6.1 Hemophilia Therapeutics ' Phase III Clinical Pipeline 19
4.6.2 Hemophilia Therapeutics ' Phase II Clinical Pipeline 19



4.6.3 Hemophilia Therapeutics ' Phase I Clinical Pipeline 20
4.6.4 Hemophilia Therapeutics ' Pre-clinical Pipeline 20
4.7 Key Takeaway 20
5 Hemophilia Therapeutics Market: Implications for Future Market Competition 21
6 Hemophilia Therapeutics Market: Future Players in the Hemophilia Therapeutics Market 22
6.1 Introduction 22
6.2 PTC Therapeutics 22
6.2.1 Overview 22
6.2.2 Hemophilia Portfolio 23
6.2.3 Hemophilia Pipeline Portfolio 23
6.2.4 Ataluren (PTC124) 23
6.3 Recoly N.V. 24
6.3.1 Overview 24
6.3.4 NecLip-rFVIIa (LongSeven) 24
6.4 Novo Nordisk 24
6.4.1 Overview 24
6.4.4 rFVIII (Recombinant factor VIIa (N8)) 25
7 Hemophilia Therapeutics Market: Appendix 26
7.1 Definitions 26
7.2 Acronyms 26
7.3 Research Methodology 26
7.3.1 Coverage 27
7.3.2 Secondary Research 28
7.3.3 Forecasting 28
7.3.4 Primary Research 31
7.3.5 Expert Panel Validation 31
7.4 Contact Us 31
7.5 Disclaimer 31
7.6 Sources 32

1.1 List of Tables
Table 1: Hemophilia Therapeutics Market, Global, Revenues ($bn), 2001-2009 6
Table 2: Hemophilia Therapeutics Market, Global, Revenue Forecasts ($bn), 2009-2016 7
Table 3: Major Marketed Products Comparison in the Hemophilia Therapeutics Market, 2010 14
Table 4: Hemophilia Therapeutics ' Most Promising Drugs Under Clinical Development, 2010 16
Table 5: Hemophilia Therapeutics ' Phase III Clinical Pipeline, 2010 19
Table 6: Hemophilia Therapeutics ' Phase II Clinical Pipeline, 2010 19
Table 7: Hemophilia Therapeutics ' Phase I Clinical Pipeline, 2010 20
Table 8: Hemophilia Therapeutics ' Pre-Clinical Pipeline, 2010 20
Table 9: PTC Therapeutics, Inc. ' Hemophilia Pipeline Products, 2010 23
Table 10: Recoly N.V. ' Hemophilia Pipeline Products, 2010 24
Table 11: Novo Nordisk ' Hemophilia Pipeline Products, 2010 25

1.2 List of Figures
Figure 1: Hemophilia Therapeutics Market, Global, Revenues ($bn), 2001-2009 6
Figure 2: Hemophilia Therapeutics Market, Global, Revenue Forecasts ($bn), 2009-2016 7



Figure 3: Opportunity and Unmet Need in the Hemophilia Therapeutics Market, 2010 8
Figure 4: Strategic Competitor Assessment of the Major Marketed Products in Hemophilia Therapeutics, 2010 10
Figure 5: Technology Trends Analytic Framework of the Hemophilia Pipeline, 2010 15
Figure 6: Technology Trends Analytic Framework of the Hemophilia Therapeutics Pipeline ' Description, 2010 16
Figure 7: Hemophilia Therapeutics Market, Global, Clinical Pipeline by Mechanism of Action (%), 2010 18
Figure 8: Hemophilia Therapeutics Pipeline by Phase of Clinical Development, 2010 19
Figure 9: Implications for Future Market Competition in the Hemophilia Therapeutics Market, 2010 21
Figure 10: Hemophilia Therapeutics Market ' Clinical Pipeline by Company (No. of Molecules), 2010 22
Figure 11: GlobalData Methodology, 2010 27
Figure 12: GlobalData Market Forecasting Model, 2010 30

Companies mentionedPTC Therapeutics
Recoly N.V
Novo Nordisk



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