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PharmaPoint: Hemophilia A and B - US Drug Forecast and Market Analysis to 2022

LONDON, April 16, 2014 /PRNewswire/ -- has added a new market research report:

PharmaPoint: Hemophilia A and B - US Drug Forecast and Market Analysis to 2022

PharmaPoint: Hemophilia A and B - US Drug Forecast and Market Analysis to 2022


The competitive landscape of hemophilia A and B recombinant therapy in the US and 5EU is dominated by the use of recombinant FVIII and FIX replacement factors, and patients with severe forms of the disease receive frequent prophylactic infusions beginning from the first one or two years of life and often continuing through adulthood. However, the burden on patients and their families to maintain the prophylactic treatment schedule is high, and there is a significant unmet need for new therapies that can reduce the number of weekly prophylactic infusions and alleviate some of this treatment burden.

The US is the largest hemophilia market, due to the high rates of disease diagnosis combined with high rates of primary rFVIII and rFIX prophylaxis among diagnosed severe hemophilia A and B patients. GlobalData anticipates that companies such as Baxter, Bayer, Novo Nordisk, CSL Behring and Biogen Idec will be able to command slight premiums over currently marketed recombinant standard of care therapies, thus contributing to overall market growth.


- Overview of Hemophilia A and B including epidemiology, etiology, symptoms, diagnosis, pathology and treatment guidelines as well as an overview on the competitive landscape.
- Detailed information on the key drugs in the US including product description, safety and efficacy profiles as well as a SWOT analysis.
- Sales forecast for the top drugs in the US from 2012-2022.
- Analysis of the impact of key events as well the drivers and restraints affecting the US Hemophilia A and B market.

Reasons to buy

- Understand and capitalize by identifying products that are most likely to ensure a robust return
- Stay ahead of the competition by understanding the changing competitive landscape for Hemophilia A and B
- Effectively plan your M&A and partnership strategies by identifying drugs with the most promising sales potential
- Make more informed business decisions from insightful and in-depth analysis of drug performance
- Obtain sales forecast for drugs from 2012-2022 in the US
1 Table of Contents
1 Table of Contents 5
1.1 List of Tables 9
1.2 List of Figures 11
2 Introduction 12
2.1 Catalyst 12
2.2 Related Reports 13
2.3 Upcoming Related Reports 15
3 Disease Overview 16
3.1 Etiology and Pathophysiology 16
3.1.1 Etiology 16
3.1.2 Pathophysiology 17
3.1.3 Prognosis and Quality of Life 19
3.2 Symptoms 21
3.2.1 Hemophilia A and B 21
3.2.2 Inhibitors 22
4 Disease Management 23
4.1 Treatment Overview 23
4.1.1 Hemophilia A and B Diagnosis 24
4.1.2 Hemophilia A and B Treatment 24
4.1.3 Inhibitors Diagnosis 26
4.1.4 Inhibitors Treatment 26
4.2 US 28
4.2.1 Clinical Practice 28
5 Competitive Assessment 31
5.1 Overview 31
5.2 Strategic Competitive Assessment 32
5.3 Product Profiles - Major Brands, Hemophilia A 36
5.3.1 Advate (octocog alfa; Antihemophilic Factor [Recombinant] Plasma/Albumin-Free) 36
5.3.2 Kogenate FS/Helixate FS (Antihemophilic Factor (Recombinant)) 40
5.3.3 Xyntha/ReFacto AF (moroctocog alfa; Antihemophilic Factor [Recombinant], Plasma/Albumin-Free) 43
5.3.4 Recombinate (octocog alfa; Antihemophilic Factor [Recombinant] Plasma/Albumin-Free) 46
5.4 Product Profiles - Hemophilia B 48
5.4.1 BeneFIX (nonacog alfa; coagulation factor IX [recombinant]) 48
5.4.2 Rixubis (BAX-326) 51
5.5 Product Profiles - Hemophilia A and B with Inhibitors 54
5.5.1 NovoSeven RT (coagulation factor VIIa [recombinant]) 54
6 Opportunity and Unmet Need 57
6.1 Overview 57
6.2 Opportunities Analysis 58
6.2.1 Longer-Lasting Recombinant Factors to Reduce the Frequency of Prophylactic Injections 58
6.2.2 Lower-Cost Recombinant Replacement Therapies 59
6.2.3 Lower Risk of Inhibitor Formation in Previously Untreated Patients 60
6.2.4 More Convenient Drug Administration 60
6.2.5 Oral Formulation of Recombinant Replacement Therapies 61
6.3 Unmet Needs Gap Analysis 62
6.3.1 Long-Acting FVIII Replacement Therapies 62
6.3.2 Gene Therapy Treatments for Hemophilia A and B 62
7 Pipeline Assessment 63
7.1 Overview 63
7.2 Promising Drugs in Clinical Development - Hemophilia A 63
7.2.1 Eloctate (rFVIII-Fc) (recombinant factor VIII fusion protein) 66
7.2.2 BAY 94-9027 (octocog alfa) 72
7.2.3 BAX-855 75
7.2.4 Novoeight (turoctocog alfa; NN7008) 79
7.2.5 N8-GP (NN7088) 83
7.2.6 CSL-627 (rFVIII-SC) 86
7.3 Promising Drugs in Clinical Development - Hemophilia B 90
7.3.1 Alprolix (rFIXFc) 92
7.3.2 CSL-654 (rIX-FP) 96
7.3.3 N9-GP (NN-7999) 99
7.3.4 IB1001 (trenacog alfa) 102
7.4 Promising Drugs in Clinical Development - Hemophilia A and B Patients with Inhibitors 106
7.4.1 BAX-817 (rFVIIa BI) 107
8 Market Outlook 110
8.1 US 110
8.1.1 Forecast 110
8.1.2 Key Events 114
8.1.3 Drivers and Barriers 114
9 Appendix 117
9.1 Bibliography 117
9.2 Abbreviations 130
9.3 Methodology 133
9.4 Forecasting Methodology 133
9.4.1 Diagnosed Hemophilia Patients 134
9.4.2 Percent Drug-Treated Patients 134
9.4.3 Drugs Included in Each Therapeutic Class 134
9.4.4 Launch and Patent Expiry Dates 135
9.4.5 General Pricing Assumptions 135
9.4.6 Individual Drug Assumptions 136
9.4.7 Pricing of Pipeline Agents 139
9.5 Physicians and Specialists Included in this Study 140
9.6 About the Authors 142
9.6.1 Authors 142
9.6.2 Global Head of Healthcare 143
9.7 About GlobalData 144
9.8 Disclaimer 144

1.1 List of Tables

Table 1: Symptoms of Hemophilia A and B 22
Table 2: Most Prescribed Hemophilia A and B Drugs in the Global Markets, 2013 27
Table 3: Leading Recombinant Replacement Therapies for Hemophilia A and B and Patients with Inhibitors, 2013 34
Table 4: Leading Plasma-Based Replacement Therapies for Hemophilia A and B and Patients with Inhibitors, 2012 35
Table 5: Product Profile - Advate (octocog alfa; Antihemophilic Factor [Recombinant] Plasma/Albumin-Free) 37
Table 6: Advate SWOT Analysis, 2013 39
Table 7: Product Profile - Kogenate FS/Helixate FS (Antihemophilic Factor (Recombinant)) 41
Table 8: Kogenate FS/Helixate FS SWOT Analysis, 2013 43
Table 9: Product Profile - Xyntha/ReFacto AF (moroctocog alfa; Antihemophilic Factor [Recombinant], Plasma/Albumin-Free) 44
Table 10: Xyntha/ReFacto AF SWOT Analysis, 2013 45
Table 11: Product Profile - Recombinate (octocog alfa; Antihemophilic Factor [Recombinant] Plasma/Albumin-Free) 46
Table 12: Recombinate SWOT Analysis, 2013 47
Table 13: Product Profile - BeneFIX (nonacog alfa; coagulation factor IX [recombinant]) 49
Table 14: BeneFIX SWOT Analysis, 2013 51
Table 15: Product Profile - Rixubis (BAX-326) 52
Table 16: Rixubis SWOT Analysis, 2013 54
Table 17: Product Profile - NovoSeven RT (coagulation factor VIIa [recombinant]) 55
Table 18: NovoSeven RT SWOT Analysis, 2013 56
Table 19: Overall Unmet Needs - Current Level of Attainment 58
Table 20: Hemophilia A - Phase I/II Pipeline, 2013 63
Table 21: Hemophilia A - Phase III Pipeline, 2013 64
Table 22: Product Profile - Eloctate (rFVIII-Fc) 67
Table 23: Eloctate SWOT Analysis, 2013 71
Table 24: Product Profile - BAY 94-9027 72
Table 25: BAY 94-9027 SWOT Analysis, 2013 75
Table 26: Product Profile - BAX-855 76
Table 27: BAX-855 SWOT Analysis, 2013 78
Table 28: Product Profile - Novoeight (turoctocog alfa; NN7008) 79
Table 29: Novoeight SWOT Analysis, 2013 82
Table 30: Product Profile - N8-GP (NN7088) 83
Table 31: N8-GP SWOT Analysis, 2013 86
Table 32: Product Profile - CSL-627 (rFVIII-SC) 87
Table 33: CSL-627 SWOT Analysis, 2013 89
Table 34: Hemophilia B - Phase I/II Pipeline, 2013 90
Table 35: Hemophilia B - Phase III Pipeline, 2013 90
Table 36: Product Profile - Alprolix (rFIXFc) 92
Table 37: Alprolix SWOT Analysis, 2013 95
Table 38: Product Profile - CSL-654 96
Table 39: CSL-654 SWOT Analysis, 2013 99
Table 40: Product Profile - N9-GP 100
Table 41: N9-GP SWOT Analysis, 2013 102
Table 42: Product Profile - IB1001 103
Table 43: IB1001 SWOT Analysis, 2013 106
Table 44: Hemophilia A and B with Inhibitors - Phase I/II Pipeline, 2013 106
Table 45: Hemophilia A and B with Inhibitors - Phase III Pipeline, 2013 106
Table 46: Product Profile - BAX-817 (rFVIIa BI) 107
Table 47: BAX-817 SWOT Analysis, 2013 109
Table 48: Sales Forecasts ($m) for Hemophilia A and B in the US, 2012-2022 112
Table 49: Key Events Impacting Sales for Hemophilia A and B in the US, 2013 114
Table 50: US Hemophilia A and B Market - Drivers and Barriers, 2013 114
Table 51: Product Launch Dates 135
Table 52: Physicians Surveyed 140

1.2 List of Figures

Figure 1: Competitive Assessment of Late-Stage Pipeline Agents in Hemophilia A, 2012-2022 65
Figure 2: Competitive Assessment of Late-Stage Pipeline Agents in Hemophilia B, 2012-2022 91
Figure 3: Sales for Hemophilia A and B in the US by Drug Class, 2012-2022 113

Read the full report:
PharmaPoint: Hemophilia A and B - US Drug Forecast and Market Analysis to 2022

For more information:
Sarah Smith
Research Advisor at
Email: [email protected]
Tel: +44 208 816 85 48

SOURCE ReportBuyer

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