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Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential - Product Image

Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential

  • Published: January 2013
  • 128 Pages
  • GBI Research

This report provides insights into the up-and-coming trends of a portion of the acquired orphan blood disorder pharmaceutical market by examining Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF), Paroxysmal Nocturnal Hemoglobinuria (PNH) and Polycythemia Vera (PV) in seven major markets: the US, the top five European countries (France, Germany, Italy, Spain and the UK) and Japan.

The report includes market forecasts to 2019, detailed pipeline analysis, in-depth profiling of major products and details of recent industry deals.

The report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by a team of industry experts.

The MF market will be the fastest growing of the five markets during the forecast period, at a Compound Annual Growth Rate (CAGR) of 34% and reaching a total market size of $1.1bn in 2019. MDS represents the largest market, expected to reach $2.2bn by 2019.

Scope

- Current and future treatment trends for three rare diseases

- Market forecasts for five rare diseases across seven major markets

- Analysis of READ MORE >

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

2 Acquired Orphan Blood Diseases - Introduction
2.1 Overview

3 Acquired Orphan Blood Diseases - Therapeutic Landscape
3.1 Paroxysmal Nocturnal Hemoglobinuria
3.1.1 Disease Overview
3.1.2 Revenues
3.1.3 Revenue Analysis by Country
3.1.4 Annual Cost of Therapy
3.1.5 Treatment Usage Patterns
3.1.6 Marketed Products
3.1.7 Market Drivers
3.1.8 Market Restraints
3.2 Paroxysmal Nocturnal Hemoglobinuria: Research and Development Pipeline
3.2.1 Overview
3.2.2 Trends in Paroxysmal Nocturnal Hemoglobinuria Pipeline
3.2.3 Promising Pipeline Molecules
3.2.4 Key Takeaway
3.3 Idiopathic Thrombocytopenic Purpura
3.3.1 Disease Overview
3.3.2 Revenues
3.3.3 Revenue Analysis by Country
3.3.4 Annual Cost of Therapy
3.3.5 Treatment Usage Patterns
3.3.6 Marketed Products
3.3.7 Market Drivers
3.3.8 Market Restraints
3.4 Idiopathic Thrombocytopenic Purpura: Research and Development Pipeline
3.4.1 Overview
3.4.2 Pipeline by Clinical Phase of Development
3.4.3 Trends in Idiopathic Thrombocytopenic Purpura Pipeline
3.4.4 Promising Pipeline Molecules
3.4.5 Key Takeaway
3.5 Myelodysplastic Syndrome
3.5.1 Disease Overview
3.5.2 Revenues
3.5.3 Revenue Analysis by Country
3.5.4 Annual Cost of Therapy
3.5.5 Treatment Usage Patterns
3.5.6 Marketed Products
3.5.7 Market Drivers
3.5.8 Market Restraints
3.6 Myelodysplastic Syndrome: Research and Development Pipeline
3.6.1 Overview
3.6.2 Pipeline by Clinical Phase of Development
3.6.3 Trends in Myelodysplastic Syndrome Pipeline
3.6.4 Promising Pipeline Molecules
3.6.5 Key Takeaway
3.7 Myelofibrosis
3.7.1 Disease Overview
3.7.2 Revenues
3.7.3 Revenue Analysis by Country
3.7.4 Annual Cost of Therapy
3.7.5 Treatment Usage Patterns
3.7.6 Marketed Products
3.7.7 Market Drivers
3.7.8 Market Restraints
3.8 Myelofibrosis: Research and Development Pipeline
3.8.1 Overview
3.8.2 Pipeline by Clinical Phase of Development
3.8.3 Trends in Myelofibrosis Pipeline
3.8.4 Promising Pipeline Molecules
3.8.5 Key Takeaway
3.9 Polycythemia Vera
3.9.1 Disease Overview
3.9.2 Revenues
3.9.3 Revenue Analysis by Country
3.9.4 Annual Cost of Therapy
3.9.5 Treatment Usage Patterns
3.9.6 Marketed Products
3.9.7 Market Drivers
3.9.8 Market Restraints
3.10 Polycythemia Vera: Research and Development Pipeline
3.10.1 Overview
3.10.2 Pipeline by Clinical Phase of Development
3.10.3 Trends in Polycythemia Vera Pipeline
3.10.4 Promising Pipeline Molecules
3.10.5 Key Takeaway

4 Acquired Orphan Blood Diseases - Strategic Consolidations
4.1 Overview
4.1.1 Segmentation by Year
4.1.2 Segmentation by Indication
4.1.3 Segmentation by Geography
4.1.4 Segmentation by Deal Value
4.1.5 Segmentation by Deal Type
4.2 Major Mergers and Acquisitions
4.3 Major Licensing Agreements

5 Acquired Orphan Blood Diseases - Appendix
5.1 Abbreviations
5.2 Sources
5.3 Research Methodology
5.3.1 Coverage
5.3.2 Secondary Research
5.3.3 Primary Research
5.3.4 Therapeutic Landscape
5.3.5 Geographical Landscape
5.3.6 Pipeline Analysis
5.3.7 Competitive Landscape
5.3.8 Expert Panel Validation
5.4 Contact Us
5.5 Disclaimer

1.1 List of Tables
Table 1: Approximate PNH Patient Population, by Country, 2011
Table 2: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019
Table 3: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Table 4: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019
Table 5: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Molecule Details, 2012
Table 6: Idiopathic Thrombocytopenic Purpura, Approximate Patient Population by Country (‘000), 2011
Table 7: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019
Table 8: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Table 9: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019
Table 10: Idiopathic Thrombocytopenic Purpura, Pipeline, Discovery, 2012
Table 11: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I, 2012
Table 12: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II, by Therapeutic Class, 2012
Table 13: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III, 2012
Table 14: Myelodysplastic Syndrome, Patient Population by Country (‘000), 2011
Table 15: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019
Table 16: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Table 17: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019
Table 18: Myelodysplastic Syndrome, Pipeline Overview, Phase I, 2012
Table 19: Myelodysplastic Syndrome, Pipeline Overview, Phase II
Table 20: Myelodysplastic Syndrome, Pipeline Overview, Phase III
Table 21: Myelodysplastic Syndrome, Pipeline Overview, Other
Table 22: Myelofibrosis, Patient Population by Country (‘000), 2011
Table 23: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019
Table 24: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Table 25: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019
Table 26: Myelofibrosis, Pipeline, Phase I, 2012
Table 27: Myelofibrosis, Pipeline, Phase II, 2012
Table 28: Myelofibrosis, Pipeline, Phase III, 2012
Table 29: Polycythemia Vera, Approximate Patient Population by Country (‘000), 2011
Table 30: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019
Table 31: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Table 32: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019
Table 33: Polycythemia Vera, Pipeline, Preclinical and Phase I, 2012
Table 34: Polycythemia Vera, Pipeline, Phase II, 2012
Table 35: Polycythemia Vera, Pipeline, Phase III, 2012

1.2 List of Figures
Figure 1: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019
Figure 2: Paroxysmal Nocturnal Hemoglobinuria, US, Revenue Forecast ($m), 2011-2019
Figure 3: Paroxysmal Nocturnal Hemoglobinuria, UK, Revenue Forecast ($m), 2011-2019
Figure 4: Paroxysmal Nocturnal Hemoglobinuria, France, Revenue Forecast ($m), 2011-2019
Figure 5: Paroxysmal Nocturnal Hemoglobinuria, Germany, Revenue Forecast ($m), 2011-2019
Figure 6: Paroxysmal Nocturnal Hemoglobinuria, Italy, Revenue Forecast ($m), 2011-2019
Figure 7: Paroxysmal Nocturnal Hemoglobinuria, Spain, Revenue Forecast ($m), 2011-2019
Figure 8: Paroxysmal Nocturnal Hemoglobinuria, Japan, Revenue Forecast ($m), 2011-2019
Figure 9: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Figure 10: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019
Figure 11: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Overview
Figure 12: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Therapeutic Class
Figure 13: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Molecule Type
Figure 14: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Mechanism of Action
Figure 15: Idiopathic Thrombocytopenic Purpura, Underlying Mechanism
Figure 16: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019
Figure 17: Idiopathic Thrombocytopenic Purpura, US, Revenue Forecast ($m), 2011-2019
Figure 18: Idiopathic Thrombocytopenic Purpura, UK, Revenue Forecast ($m), 2011-2019
Figure 19: Idiopathic Thrombocytopenic Purpura, France, Revenue Forecast ($m), 2011-2019
Figure 20: Idiopathic Thrombocytopenic Purpura, Germany, Revenue Forecast ($m), 2011-2019
Figure 21: Idiopathic Thrombocytopenic Purpura, Italy, Revenue Forecast ($m), 2011-2019
Figure 22: Idiopathic Thrombocytopenic Purpura, Spain, Revenue Forecast ($m), 2011-2019
Figure 23: Idiopathic Thrombocytopenic Purpura, Japan, Revenue Forecast ($m), 2011-2019
Figure 24: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Figure 25: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019
Figure 26: Idiopathic Thrombocytopenic Purpura, Pipeline Overview
Figure 27: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I by Therapeutic Class
Figure 28: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II Molecule Comparison
Figure 29: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III Molecule Comparison
Figure 30: Idiopathic Thrombocytopenic Purpura, Pipeline by Therapeutic Class
Figure 31: Idiopathic Thrombocytopenic Purpura, Pipeline by Molecule Type
Figure 32: Idiopathic Thrombocytopenic Purpura, Pipeline by Mechanism of Action
Figure 33: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019
Figure 34: Myelodysplastic Syndrome, US, Revenue Forecast ($m), 2011-2019
Figure 35: Myelodysplastic Syndrome, UK, Revenue Forecast ($m), 2011-2019
Figure 36: Myelodysplastic Syndrome, France, Revenue Forecast ($m), 2011-2019
Figure 37: Myelodysplastic Syndrome, Germany, Revenue Forecast ($m), 2011-2019
Figure 38: Myelodysplastic Syndrome, Italy, Revenue Forecast ($m), 2011-2019
Figure 39: Myelodysplastic Syndrome, Spain, Revenue Forecast ($m), 2011-2019
Figure 40: Myelodysplastic Syndrome, Japan, Revenue Forecast ($m), 2011-2019
Figure 41: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Figure 42: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019
Figure 43: Myelodysplastic Syndrome, Pipeline Overview
Figure 44: Myelodysplastic Syndrome, Pipeline, Phase I Molecule Comparison
Figure 45: Myelodysplastic Syndrome, Pipeline, Phase II Molecule Comparison
Figure 46: Myelodysplastic Syndrome, Pipeline, Phase III Molecule Comparison
Figure 47: Myelodysplastic Syndrome, Pipeline by Therapeutic Class
Figure 48: Myelodysplastic Syndrome, Pipeline by Molecule Type
Figure 49: Myelodysplastic Syndrome, Pipeline by Mechanism of Action
Figure 50: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019
Figure 51: Myelofibrosis, US, Revenue Forecast ($m), 2011-2019
Figure 52: Myelofibrosis, UK, Revenue Forecast ($m), 2011-2019
Figure 53: Myelofibrosis, France, Revenue Forecast ($m), 2011-2019
Figure 54: Myelofibrosis, Germany, Revenue Forecast ($m), 2011-2019
Figure 55: Myelofibrosis, Italy, Revenue Forecast ($m), 2011-2019
Figure 56: Myelofibrosis, Spain, Revenue Forecast ($m), 2011-2019
Figure 57: Myelofibrosis, Japan, Revenue Forecast ($m), 2011-2019
Figure 58: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Figure 59: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019
Figure 60: Myelofibrosis, Pipeline Overview
Figure 61: Myelofibrosis, Pipeline, Phase I Molecule Comparison
Figure 62: Myelofibrosis, Pipeline, Phase II Molecule Comparison
Figure 63: Myelofibrosis, Pipeline, Phase III Molecule Comparison
Figure 64: Myelofibrosis, Pipeline by Therapeutic Class
Figure 65: Myelofibrosis, Pipeline by Molecule Type
Figure 66: Myelofibrosis, Pipeline by Mechanism of Action
Figure 67: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019
Figure 68: Polycythemia Vera, US, Revenue Forecast ($m), 2011-2019
Figure 69: Polycythemia Vera, UK, Revenue Forecast ($m), 2011-2019
Figure 70: Polycythemia Vera, France, Revenue Forecast ($m), 2011-2019
Figure 71: Polycythemia Vera, Germany, Revenue Forecast ($m), 2011-2019
Figure 72: Polycythemia Vera, Italy, Revenue Forecast ($m), 2011-2019
Figure 73: Polycythemia Vera, Spain, Revenue Forecast ($m), 2011-2019
Figure 74: Polycythemia Vera, Japan, Revenue Forecast ($m), 2011-2019
Figure 75: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019
Figure 76: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019
Figure 77: Polycythemia Vera, Pipeline Overview
Figure 78: Polycythemia Vera, Pipeline, Phase I Molecule Comparison
Figure 79: Polycythemia Vera, Pipeline, Phase II Molecule Comparison
Figure 80: Polycythemia Vera, Pipeline by Therapeutic Class
Figure 81: Polycythemia Vera, Pipeline by Molecule Type
Figure 82: Polycythemia Vera, Pipeline by Mechanism of Action
Figure 83: Deal Segmentation by Year, 2004-2012
Figure 84: Deal Segmentation by Indication
Figure 85: Deal Segmentation by Geography, Total Market
Figure 86: Deal Segmentation by Value
Figure 87: Deal Segmentation by Deal Type
Figure 88: GBI Research Market Forecasting Model

"Pioneering Pharma Companies Benefit from Untapped Market of Acquired Orphan Blood Disease Drugs

Physicians treating rare blood disorders are gaining more drug treatments to choose from, which allow patients to avoid demanding surgeries and chemotherapy, and fill the pockets of pharmaceutical companies who can charge top dollar in these untapped markets, states a new report by healthcare experts GBI Research.

This new report* looks at the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF) and Polycythemia Vera (PV), acquired orphan blood diseases which result in a sufferer’s body not producing blood correctly. These diseases have serious risks, and treatments are often limited to symptom management, and improvement of quality of life, with risky surgical procedures offering the only alternative. This bleak outlook makes the orphan disease market a great opportunity for drug discovery and development, as patient populations are small but untapped.

Soliris (eculizumab) is a perfect example of a successful orphan blood disease drug. PNH is a potentially life-threatening blood disease, characterised by severe anemia caused by the destruction of red blood cells in the bloodstream. Bone marrow transplants can provide a cure, but the high risk procedure is suitable for only a few patients. Treatment methods include blood transfusions, iron therapy, and growth factors, but monoclonal antibody Soliris dominates the PNH treatment market, and holds the title of the most expensive drug in the world. The drug reduces the activity of the immune system, reducing hemolysis and risk of thrombosis associated with PNH market. Due to the market dominance of Soliris, the PNH pipeline is very weak, so Alexion Pharmaceuticals look set to continue to benefit from this high pricing.

The PNH treatment market in the US, the top five EU countries (the UK, France, Germany, Italy and Spain), and Japan is expected to grow at a Compound Annual Growth Rate (CAGR) of 4% over the 2011–2019 period to $1,164m by 2019.

MF is another acquired orphan blood disease, and success in the drug pipeline has rewarded Incyte Corporation handsomely. MFis a myeloproliferative disease which sees patient’s bone marrow get replaced by collagen fibrosis, impairing the individual’s ability to produce new blood cells. Stem cell transplantation is the only curative treatment available for MF sufferers, but this procedure is highly risky, requires a young patient and suitable donor, and often only leads to remission rather than a cure. Treatments to alleviate disease symptoms include blood transfusions, androgen therapy, surgery, chemotherapy and radiation therapy, but these gruelling therapies have debilitating side effects.

Jakafi (ruxolitinib) is a JAK inhibitor which works to target the genes believed to cause MF, approved by the FDA in November 2011 for the treatment of intermediate or high-risk MF, and perhaps set to be a future blockbuster drug. Kimberley Carter, Associate Analyst at GBI Research states: “The exceptional performance of Soliris has shown that there is a great opportunity for pharma companies in the development of novel, curative drugs for orphan blood disorders. Many patients with these diseases rely on generic drugs that often come with debilitating side effects and may require a high level of commitment from the patient, such as regular hospital treatments. Therefore, the unmet needs of these patients are very high and the right therapy would perform very well.”

Other drugs with the same mechanism of action are currently in the development pipeline, as other pharmaceutical companies fight to compete for Jakafi’s market share, and as a result of this healthy competition, the market for pharmaceutical treatment for MF is expected to grow at a CAGR of 34% throughout 2011–2019 to reach a value of $1,127m by 2019.

*Acquired Orphan Blood Diseases Therapeutics Market to 2019 - Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential

This report provides insights into the up-and-coming trends of the acquired orphan blood disorder pharmaceutical market by examining Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF), Paroxysmal Nocturnal Hemoglobinuria (PNH) and Polycythemia Vera (PV) in seven major markets: the US, the top five European countries (France, Germany, Italy, Spain and the UK) and Japan. The report includes market forecasts to 2019, detailed pipeline analysis, in-depth profiling of major products and details of recent industry deals.

The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by a team of industry experts.

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