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市場調査レポート

希少疾病:医薬品情勢・機会分野

Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas

発行 ROOTS ANALYSIS 商品コード 324982
出版日 ページ情報 英文 481 Pages
納期: 即日から翌営業日
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希少疾病:医薬品情勢・機会分野 Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas
出版日: 2015年03月23日 ページ情報: 英文 481 Pages
概要

当レポートでは、希少疾病治療薬の開発市場について包括的に調査分析し、パイプライン分析、疾病別の市場潜在性、および大手製薬企業50社の企業プロファイルをまとめ、お届けいたします。

第1章 序論

第2章 イントロダクション

第3章 希少疾病:パイプライン分析

  • 本章の概要
  • 希少疾病の治療薬
  • 規制委員会:希少疾病治療薬市場の促進

第4章 希少疾病:市場潜在性

  • 本章の概要
  • 市場潜在性
  • 希少疾病:市場潜在性
    • 卵巣癌
    • 慢性リンパ性白血病
    • 多発性骨髄腫
    • 胃癌
    • 急性骨髄性白血病
    • 転移性乳癌
      • 概要
      • 統計データ
      • 市販薬の例
      • パイプライン薬
      • アナリストの見通し

第5章 希少疾病:企業プロファイル

  • 本章の概要
  • 主な貢献企業:潜在性分析
  • 主な貢献企業:R&D支出
  • 主な貢献企業:希少疾病の研究費
  • 主な貢献企業:治療分野別の配分
  • 企業プロファイル (50社)
    • 企業概要
    • 財務実績
    • 希少疾病治療薬のポートフォリオ
    • 提携
    • 近年の開発
    • 企業イニシアチブ

第6章 結論

第7章 付録:企業リスト

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目次
Product Code: RA10034

The service oriented and dynamic nature of the healthcare industry has led to the discovery of a plethora of treatment options for a wide range of ailments.However, there are certain diseases, termed as rare, that continue to remain untreated and neglected. Rare / orphan diseases, as the name suggests, are those diseases which are relatively infrequent in occurrence. Many orphan diseases are life-threatening and progressive in nature. Absence of potential treatment regimes catering to these diseases is a big unmet need in the market.

The prevalence and adversity of these ailments has gained significant attention in the global healthcare industry. Stakeholders have realized the necessity and importance of urgently addressing the widening concerns and the need for discovering treatment options for several rare diseases. There is a tremendous scope offered by the innovation driven research being executed by various organizations in an effort to bridge gaps in the current market landscape.

The enactment of the Orphan Drugs Act in the US and EU emerged as ground breaking and provided the necessary support to guide research focused on rare diseases worldwide.The orphan designated drugs are granted important incentives which include market exclusivity and fees reductions. This regulatory assistance has provided a strong impetus to the pharmaceutical companies and led to their active participation in the market. Collaborative agreements and partnerships, amongst the pharmaceutical firms and other active organizations (such as EURORDIS, NORD and Global Genes), are also playing a key role in addressing the needs of this market.

The wide gap between the demand and supply of drugs is likely to provide a major boost to the market in the coming decade. Several drugs for various rare diseases are currently under development and, once approved, are likely to be readily accepted by the untreated patient population.

Synopsis

The 'Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas ' report is a comprehensive study of the market of drugs being developed by the top pharmaceutical companies for rare diseases. The focus of this study is primarily to uncover several initiatives currently underway to harness the potential presented in different therapeutic areas. One of the basic building blocks for analysing the market was to prepare an extensive pipeline of drugs for rare diseases. This effort encompassed reviewing several databases, company websites and other information available in the public domain.

We confined the focus of our study to 50 large pharmaceutical companies which invest a proportion of their R&D spend on drugs for rare diseases. These pharmaceutical companies were profiled to get an in-depth view of the work being done by them in this domain. The profiles include a general overview of the firms, financial performance over the last five to six years, and the current marketed and clinical pipeline products specific to rare diseases.

With a robust pipeline in place, we then analysed where the industry's current vision lies and highlighted a number of efforts which have been taken by pharmaceutical companies, healthcare organizations and other industry stakeholders. Specifically, our market potential analysis outlines key patient demographics and underscores potential areas anticipated to emerge as major revenue generators for the pharmaceutical companies. In addition to the above, we have also provided our own independent perspective on the key drivers behind the growth of this market, and the associated challenges.

Key Highlights

  • 1.With over 7000 rare diseases reported in the world, lack of awareness, dispersed target population and inefficient reporting of these diseases limits the pace of development in this industry. However, the industry-academia coalition has helped in filling some of these gaps. An active participation by pharma firms through various initiatives, such as the Rare Disease Day, has helped to address the existing lacunae in the demand supply gap for these drugs.
  • 2.The market is characterized by the presence of several pipeline drugs under development for the treatment of rare diseases. Of these, 30% drugs are in phase III of clinical development whereas 38% of drugs are under development in phase II clinical trials.Phase I drugs account for 20% to the total market.
  • 3.Companies such as Roche, BMS, Sanofi, GSK, Boehringer Ingelheim, Celgene, Novartis and Pfizer are some of the key players in the market. However, the market remains opportunistic for newer players to step in and benefit from the vast potential in different therapeutic areas.
  • 4.With a share of more than 50%, oncology remains the key focus area of pharmaceutical firms. Inflammatory disorders and neural disorders are some other well-known areas being targeted.
  • 5.On an average, 13-14 drugs are being developed by each pharmaceutical company covered in our analysis. However, there is a stark variation amongst the companies. Some of the bigger pharma companies, such as Roche and BMS, have a much wider portfolio.

Research Methodology

Most of the data presented in this report has been gathered via secondary and primary research. For most of our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include

  • Annual reports
  • Investor presentations
  • SEC filings
  • Industry databases
  • News releases from company websites
  • Government policy documents
  • Industry analysts' views

Chapter Outlines

Chapter 2 provides a general introduction to rare diseases. We have highlighted the indispensable role played by several regulatory organizations in accelerating this market. The chapter also elaborates upon the various drivers responsible for the growth in this market.

Chapter 3 provides a comprehensive market landscape of drugs in development for rare diseases. This chapter includes an elaborate analysis of pipeline drugs which are currently in different stages of development. The detailed analysis includes information on the targeted indications, types of molecules and the current phases of development. The chapter also provides the historic ladder for the orphan drug designations conferred to several drugs in the US and the European markets.

Chapter 4 offers an insightful discussion of six most popular indications targeted by the drugs under development. This analysis revolves around current landscape, prevailing competition, anticipated shift in the market trend and the key focus for the future.

Chapter 5 provides detailed company profiles of the top pharmaceutical companies focusing in the field of rare diseases. Each company profile includes information such as financial performance, geographical presence, marketed / pipeline drugs, recent collaborations specific to rare diseases and the initiatives taken by the firm towards the treatment of rare diseases.

Chapter 6 summarises the overall report. In this chapter, we have provided a recap of the key takeaways and our overall opinion on where the market is headed in the mid-long term.

Chapter 7 provides the list of companies covered in this report.

Table of Contents

1. PREFACE

  • 1.1. Overview
  • 1.2. Research Methodology
  • 1.3. Chapter Outlines

2. INTRODUCTION

  • 2.1. Overview
  • 2.2. Scope of the Study
  • 2.3. Methodology
  • 2.4. Key Highlights

3. RARE DISEASE: PIPLEINE ANALYSIS

  • 3.1. Chapter Overview
  • 3.2. Drugs for Rare Diseases
    • 3.2.1. Drugs for Rare Diseases: Distribution by Highest Phase of Development
    • 3.2.2. Drugs for Rare Diseases: Distribution by Therapeutic Area
    • 3.2.3. Drugs for Oncological Rare Diseases: Distribution by Highest Phase of Development
    • 3.2.4. Drugs for Rare Diseases: Distribution by Nature of Drug
  • 3.3. Regulatory Committees: Impetus to the Rare Disease Drug Market
    • 3.3.1. US Orphan Drug Designations: Timeline
    • 3.3.2. EU Orphan Drug Designations: Timeline

4. RARE DISEASES: MARKET POTENTIAL

  • 4.1. Chapter Overview
  • 4.2. Market Potential
  • 4.3. Rare Indications: Market Potential
    • 4.3.1. Ovarian Cancer
      • 4.3.1.1. Overview
      • 4.3.1.2. Facts
      • 4.3.1.3. Example Marketed Drugs
      • 4.3.1.4. Pipeline Drugs
      • 4.3.1.5. Roots Analysis' Perspective
    • 4.3.2. Chronic Lymphocytic Leukemia
      • 4.3.2.1. Overview
      • 4.3.2.2. Facts
      • 4.3.2.3. Example Marketed Drugs
      • 4.3.2.4. Pipeline Drugs
      • 4.3.2.5. Roots Analysis' Perspective
    • 4.3.3. Multiple Myeloma
      • 4.3.3.1. Overview
      • 4.3.3.2. Facts
      • 4.3.3.3. Example Marketed Drugs
      • 4.3.3.4. Pipeline Drugs
      • 4.3.3.5. Roots Analysis' Perspective
    • 4.3.4. Gastric Cancer
      • 4.3.4.1. Overview
      • 4.3.4.2. Facts
      • 4.3.4.3. Example Marketed Drugs
      • 4.3.4.4. Pipeline Drugs
      • 4.3.4.5. Roots Analysis' Perspective
    • 4.3.5. Acute Myeloid Leukemia
      • 4.3.5.1. Overview
      • 4.3.5.2. Facts
      • 4.3.5.3. Example Marketed Drugs
      • 4.3.5.4. Pipeline Drugs
      • 4.3.5.5. Roots Analysis' Perspective
    • 4.3.6. Metastatic Breast Cancer
      • 4.3.6.1. Overview
      • 4.3.6.2. Facts
      • 4.3.6.3. Example Marketed Drugs
      • 4.3.6.4. Pipeline Drugs
      • 4.3.6.5. Roots Analysis' Perspective

5. RARE DISEASES: COMPANY PROFILES

  • 5.1. Chapter Overview
  • 5.2. Key Contributors: Potential Analysis
  • 5.3. Key Contributors: R&D Spend
  • 5.4. Key Contributors: Research Spend on Rare Diseases
  • 5.5. Key Contributors: Distribution by Therapeutic Area
  • 5.6. Company Profiles
    • 5.6.1. Roche Laboratories
      • 5.6.1.1. Company Overview
      • 5.6.1.2. Financial Performance
      • 5.6.1.3. Rare Disease Drug Portfolio
      • 5.6.1.4. Partnerships
      • 5.6.1.5. Recent Developments
      • 5.6.1.6. Company Initiatives
    • 5.6.2. Bristol Myers Squibb
      • 5.6.2.1. Overview
      • 5.6.2.2. Financial Performance
      • 5.6.2.3. Rare Disease Drug Portfolio
      • 5.6.2.4. Partnerships
      • 5.6.2.5. Recent Developments
      • 5.6.2.6. Company Initiatives
    • 5.6.3. Celgene
      • 5.6.3.1. Company Overview
      • 5.6.3.2. Financial Performance
      • 5.6.3.3. Rare Disease Drug Portfolio
      • 5.6.3.4. Partnerships
      • 5.6.3.5. Recent Developments
      • 5.6.3.6. Company Initiatives
    • 5.6.4. AstraZeneca
      • 5.6.4.1. Company Overview
      • 5.6.4.2. Financial Performance
      • 5.6.4.3. Rare Disease Drug Portfolio
      • 5.6.4.4. Partnerships
      • 5.6.4.5. Recent Developments
      • 5.6.4.6. Company Initiatives
    • 5.6.5. Eli Lilly
      • 5.6.5.1. Company Overview
      • 5.6.5.2. Financial Performance
      • 5.6.5.3. Rare Disease Drug Portfolio
      • 5.6.5.4. Partnerships
      • 5.6.5.5. Recent Developments
      • 5.6.5.6. Company Initiatives
    • 5.6.6. Pfizer
      • 5.6.6.1. Company Overview
      • 5.6.6.2. Financial Performance
      • 5.6.6.3. Rare Disease Product Portfolio
      • 5.6.6.4. Partnerships
      • 5.6.6.5. Recent Developments
      • 5.6.6.6. Company Initiatives
    • 5.6.7. GSK
      • 5.6.7.1. Company Overview
      • 5.6.7.2. Financial Performance
      • 5.6.7.3. Rare Disease Drug Portfolio
      • 5.6.7.4. Partnerships
      • 5.6.7.5. Recent Developments
      • 5.6.7.6. Company Initiatives
    • 5.6.8. Boehringer Ingelheim
      • 5.6.8.1. Company Overview
      • 5.6.8.2. Financial Performance
      • 5.6.8.3. Rare Disease Drug Portfolio
      • 5.6.8.4. Partnerships
      • 5.6.8.5. Recent Developments
      • 5.6.8.6. Company Initiatives
    • 5.6.9. Amgen
      • 5.6.9.1. Overview
      • 5.6.9.2. Financial Performance
      • 5.6.9.3. Rare Disease Drug Portfolio
      • 5.6.9.4. Partnerships
      • 5.6.9.5. Recent Developments
      • 5.6.9.6. Company Initiatives
    • 5.6.10. AbbVie
      • 5.6.10.1. Overview
      • 5.6.10.2. Financial Performance
      • 5.6.10.3. Rare Disease Drug Portfolio
      • 5.6.10.4. Partnerships
      • 5.6.10.5. Recent Developments
      • 5.6.10.6. Company Initiatives
    • 5.6.11. Novartis
      • 5.6.11.1. Company Overview
      • 5.6.11.2. Financial Performance
      • 5.6.11.3. Rare Disease Drug Portfolio
      • 5.6.11.4. Partnerships
      • 5.6.11.5. Recent Developments
      • 5.6.11.6. Company Initiatives
    • 5.6.12. Sanofi
      • 5.6.12.1. Company Overview
      • 5.6.12.2. Financial Performance
      • 5.6.12.3. Rare Disease Drug Portfolio
      • 5.6.12.4. Partnerships
      • 5.6.12.5. Recent Developments
      • 5.6.12.6. Company Initiatives
    • 5.6.13. Takeda
      • 5.6.13.1. Company Overview
      • 5.6.13.2. Financial Performance
      • 5.6.13.3. Rare Disease Drug Portfolio
      • 5.6.13.4. Partnerships
      • 5.6.13.5. Recent Developments
      • 5.6.13.6. Company Initiatives
    • 5.6.14. Ono Pharmaceuticals
      • 5.6.14.1. Company Overview
      • 5.6.14.2. Financial Performance
      • 5.6.14.3. Rare Disease Drug Portfolio
      • 5.6.14.4. Partnerships
      • 5.6.14.5. Recent Developments
    • 5.6.15. Otsuka
      • 5.6.15.1. Company Overview
      • 5.6.15.2. Financial Performance
      • 5.6.15.3. Rare Disease Drug Portfolio
      • 5.6.15.4. Partnerships
      • 5.6.15.5. Recent Developments
    • 5.6.16. Eisai
      • 5.6.16.1. Company Overview
      • 5.6.16.2. Financial Performance
      • 5.6.16.3. Rare Disease Drug Portfolio
      • 5.6.16.4. Partnerships
      • 5.6.16.5. Recent Developments
      • 5.6.16.6. Company Initiatives
    • 5.6.17. Daiichi Sankyo
      • 5.6.17.1. Company Overview
      • 5.6.17.2. Financial Performance
      • 5.6.17.3. Rare Disease Drug Portfolio
      • 5.6.17.4. Partnerships
      • 5.6.17.5. Recent Developments
      • 5.6.17.6. Company Initiatives
    • 5.6.18. Shire
      • 5.6.18.1. Company Overview
      • 5.6.18.2. Financial Performance
      • 5.6.18.3. Rare Disease Drug Portfolio
      • 5.6.18.4. Partnerships
      • 5.6.18.5. Recent Developments
      • 5.6.18.6. Company Initiatives
    • 5.6.19. Johnson & Johnson
      • 5.6.19.1. Company Overview
      • 5.6.19.2. Financial Performance
      • 5.6.19.3. Rare Diseases Drug Portfolio
      • 5.6.19.4. Partnerships
      • 5.6.19.5. Recent Developments
      • 5.6.19.6. Company Initiatives
    • 5.6.20. Kyowa Hakko Kirin
      • 5.6.20.1. Company Overview
      • 5.6.20.2. Financial Performance
      • 5.6.20.3. Rare Disease Drug Portfolio
      • 5.6.20.4. Partnerships
      • 5.6.20.5. Recent Developments
    • 5.6.21. Astellas
      • 5.6.21.1. Company Overview
      • 5.6.21.2. Financial Performance
      • 5.6.21.3. Rare Disease Drug Portfolio
      • 5.6.21.4. Partnerships
      • 5.6.21.5. Recent Developments
      • 5.6.21.6. Company Initiatives
    • 5.6.22. Biogen Idec
      • 5.6.22.1. Company Overview
      • 5.6.22.2. Financial Performance
      • 5.6.22.3. Rare Disease Drug Portfolio
      • 5.6.22.4. Partnerships
      • 5.6.22.5. Recent Developments
      • 5.6.22.6. Company Initiatives
    • 5.6.23. Chugai Pharmaceuticals
      • 5.6.23.1. Company Overview
      • 5.6.23.2. Financial Performance
      • 5.6.23.3. Rare Disease Drug Portfolio
      • 5.6.23.4. Partnerships
      • 5.6.23.5. Recent Developments
      • 5.6.23.6. Company Initiatives
    • 5.6.24. Gilead Sciences
      • 5.6.24.1. Company Overview
      • 5.6.24.2. Financial Performance
      • 5.6.24.3. Rare Disease Drug Portfolio
      • 5.6.24.4. Partnerships
      • 5.6.24.5. Recent Developments
      • 5.6.24.6. Company Initiatives
    • 5.6.25. Mitsubishi Tanabe Pharma
      • 5.6.25.1. Company Overview
      • 5.6.25.2. Financial Performance
      • 5.6.25.3. Rare Disease Drug Portfolio
      • 5.6.25.4. Partnerships
      • 5.6.25.5. Recent Developments
    • 5.6.26. Sumitomo Dainippon
      • 5.6.26.1. Company Overview
      • 5.6.26.2. Financial Performance
      • 5.6.26.3. Rare Disease Drug Portfolio
      • 5.6.26.4. Partnerships
      • 5.6.26.5. Recent Developments
    • 5.6.27. ChiesiFarmaceutici
      • 5.6.27.1. Company Overview
      • 5.6.27.2. Financial Performance
      • 5.6.27.3. Rare Disease Drugs Portfolio
      • 5.6.27.4. Partnerships
      • 5.6.27.5. Recent Developments
      • 5.6.27.6. Company Initiatives
    • 5.6.28. Teva
      • 5.6.28.1. Company Overview
      • 5.6.28.2. Financial Performance
      • 5.6.28.3. Rare Disease Drug Portfolio
      • 5.6.28.4. Partnerships
      • 5.6.28.5. Recent Developments
      • 5.6.28.6. Company Initiatives
    • 5.6.29. Baxter
      • 5.6.29.1. Overview
      • 5.6.29.2. Financial Performance
      • 5.6.29.3. Rare disease Drug Portfolio
      • 5.6.29.4. Partnerships
      • 5.6.29.5. Recent Developments
      • 5.6.29.6. Company Initiatives
    • 5.6.30. Grifols
      • 5.6.30.1. Company Overview
      • 5.6.30.2. Financial Performance
      • 5.6.30.3. Rare Disease Drug Portfolio
      • 5.6.30.4. Partnerships
      • 5.6.30.5. Recent Developments
      • 5.6.30.6. Company Initiatives
    • 5.6.31. Bayer
      • 5.6.31.1. Company Overview
      • 5.6.31.2. Financial Performance
      • 5.6.31.3. Rare Diseases Drug Portfolio
      • 5.6.31.4. Partnerships
      • 5.6.31.5. Recent Developments
      • 5.6.31.6. Company Initiatives
    • 5.6.32. Actelion
      • 5.6.32.1. Company Overview
      • 5.6.32.2. Financial Performance
      • 5.6.32.3. Rare Disease Drug Portfolio
      • 5.6.32.4. Partnerships
      • 5.6.32.5. Recent Developments
    • 5.6.33. Ipsen
      • 5.6.33.1. Company Overview
      • 5.6.33.2. Financial Performance
      • 5.6.33.3. Rare Disease Drug Portfolio
      • 5.6.33.4. Partnerships
      • 5.6.33.5. Recent Developments
    • 5.6.34. Nippon Shinyaku Pharmaceuticals
      • 5.6.34.1. Company Overview
      • 5.6.34.2. Financial Performance
      • 5.6.34.3. Rare Disease Drug Portfolio
      • 5.6.34.4. Partnerships
      • 5.6.34.5. Recent Developments
    • 5.6.35. Merck KGaA
      • 5.6.35.1. Company Overview
      • 5.6.35.2. Financial Performance
      • 5.6.35.3. Rare Disease Drug Portfolio
      • 5.6.35.4. Partnerships
      • 5.6.35.5. Recent Developments
      • 5.6.35.6. Company Initiatives
    • 5.6.36. UCB
      • 5.6.36.1. Company Overview
      • 5.6.36.2. Financial Performance
      • 5.6.36.3. Rare Disease Drug Portfolio
      • 5.6.36.4. Company Initiatives
    • 5.6.37. Commonwealth Serum Laboratories (CSL)
      • 5.6.37.1. Company Overview
      • 5.6.37.2. Financial Performance
      • 5.6.37.3. Rare Disease Drug Portfolio
      • 5.6.37.4. Partnerships
      • 5.6.37.5. Recent Development
      • 5.6.37.6. Company Initiatives
    • 5.6.38. Merck & Co.
      • 5.6.38.1. Company Overview
      • 5.6.38.2. Financial Performance
      • 5.6.38.3. Rare Disease Drug Portfolio
      • 5.6.38.4. Partnerships
      • 5.6.38.5. Recent Developments
      • 5.6.38.6. Company Initiatives
    • 5.6.39. Sigma-Tau Pharmaceuticals
      • 5.6.39.1. Company Overview
      • 5.6.39.2. Rare Disease Drug Portfolio
      • 5.6.39.3. Pipeline Drugs
      • 5.6.39.4. Partnerships
      • 5.6.39.5. Recent Developments
      • 5.6.39.6. Company Initiatives
    • 5.6.40. Alexion
      • 5.6.40.1. Company Overview
      • 5.6.40.2. Financial Performance
      • 5.6.40.3. Rare Disease Drug Portfolio
      • 5.6.40.4. Partnerships
      • 5.6.40.5. Recent Developments
      • 5.6.40.6. Company Initiatives
    • 5.6.41. Recordati
      • 5.6.41.1. Company Overview
      • 5.6.41.2. Financial Performance
      • 5.6.41.3. Rare Disease Drug Portfolio
      • 5.6.41.4. Partnerships
      • 5.6.41.5. Recent Developments
      • 5.6.41.6. Company Initiatives
    • 5.6.42. Novo Nordisk
      • 5.6.42.1. Company Overview
      • 5.6.42.2. Financial Performance
      • 5.6.42.3. Rare Disease Drug Portfolio
      • 5.6.42.4. Partnerships
      • 5.6.42.5. Recent Developments
      • 5.6.42.6. Company Initiatives
    • 5.6.43. Shionogi Inc
      • 5.6.43.1. Company Overview
      • 5.6.43.2. Financial Performance
      • 5.6.43.3. Rare Disease Drug Portfolio
      • 5.6.43.4. Partnerships
      • 5.6.43.5. Recent Developments
      • 5.6.43.6. Company Initiatives
    • 5.6.44. Vertex Pharmaceuticals
      • 5.6.44.1. Company Overview
      • 5.6.44.2. Financial Performance
      • 5.6.44.3. Rare Disease Drugs Portfolio
      • 5.6.44.4. Partnerships
      • 5.6.44.5. Recent Developments
    • 5.6.45. Meiji Seika Pharma Co, Ltd.
      • 5.6.45.1. Company Overview
      • 5.6.45.2. Financial Performance
      • 5.6.45.3. Rare Disease Drug Portfolio
      • 5.6.45.4. Partnerships
    • 5.6.46. Ferring Pharmaceuticals
      • 5.6.46.1. Company Overview
      • 5.6.46.2. Rare Disease Drug Portfolio
      • 5.6.46.3. Recent Developments
      • 5.6.46.4. Company Initiatives
    • 5.6.47. Orion Pharma
      • 5.6.47.1. Company Overview
      • 5.6.47.2. Financial Performance
      • 5.6.47.3. Rare Disease Drug Portfolio
      • 5.6.47.4. Partnerships
      • 5.6.47.5. Recent Developments
    • 5.6.48. Santen
      • 5.6.48.1. Company Overview
      • 5.6.48.2. Financial Performance
      • 5.6.48.3. Rare Disease Drug Portfolio
      • 5.6.48.4. Recent Developments
    • 5.6.49. Teijin Pharmaceuticals
      • 5.6.49.1. Company Overview
      • 5.6.49.2. Financial Performance
      • 5.6.49.3. Rare Disease Drug Portfolio
      • 5.6.49.4. Partnerships
      • 5.6.49.5. Recent Developments
    • 5.6.50. Lundbeck
      • 5.6.50.1. Company Overview
      • 5.6.50.2. Financial Performance
      • 5.6.50.3. Rare Disease Drug Portfolio
      • 5.6.50.4. Partnerships
      • 5.6.50.5. Recent Developments
      • 5.6.50.6. Company Initiatives

6. CONCLUSION

  • 6.1. The Emerging Focus in a Widely Untapped Market
  • 6.2. Several Stakeholders have Collaborated to Lead the Initiatives
  • 6.3. Big Pharma are the Major Contributors
  • 6.4. Industry- Academia Coalition: Expunge the MarketConstraints
  • 6.5. Rare Disease Market: Overall Opportunity is Big

7. APPENDIX: LIST OF COMPANIES

List of Figures

  • Figure 2.1 Rare Disease Drugs Market: Key Takeaways
  • Figure 3.1 Rare Disease Drugs: Distribution by Highest Phase of Development
  • Figure 3.2 Rare Disease Drugs: Distribution by Target Indication
  • Figure 3.3 Rare Disease Drugs for Oncology: Distribution by Highest Phase of Development
  • Figure 3.4 Rare Disease Drugs: Distribution by Nature of Drug
  • Figure 3.5 Rare Disease Drugs: US Orphan Drug Designation Timeline
  • Figure 3.6 Rare Disease Drugs: EU Orphan Drug Designation Timeline
  • Figure 4.1 Rare Diseases: Market Potential
  • Figure 4.2 Ovarian Cancer Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.3 Ovarian Cancer Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.4 Ovarian Cancer: Market Potential
  • Figure 4.5 CLL Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.6 CLL Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.7 CLL: Market Potential
  • Figure 4.8 Multiple Myeloma Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.9 Multiple Myeloma Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.10 Multiple Myeloma: Market Potential
  • Figure 4.11 Gastric Cancer Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.12 Gastric Cancer Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.13 Gastric Cancer: Market Potential
  • Figure 4.14 AML Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.15 AML Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.16 AML: Market Potential
  • Figure 4.17 Metastatic Breast Cancer Pipeline Drugs: Distribution by Nature of Drug
  • Figure 4.18 Metastatic Breast Cancer Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 4.19 Metastatic Breast Cancer: Market Potential
  • Figure 5.1 Key Contributors: Pipeline Strength
  • Figure 5.2 Key Contributors: R&D Spend
  • Figure 5.3 Key Contributors: Proxy R&D Spending (USD Billion)
  • Figure 5.4 Key Contributors: Distribution by Therapeutic Area
  • Figure 5.5 Roche Revenues, 2009-2014 (CHF Billion)
  • Figure 5.6 Roche Revenues: Distribution by Business Segments, 2011 - 2014 (CHF Billion)
  • Figure 5.7 Roche Pharmaceutical Revenues: Distribution by Therapeutic Areas, 2014 (CHF Billion)
  • Figure 5.8 Roche Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.9 Roche Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.10 Roche Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.11 BMS Revenues, 2009-2014 (USD Billion)
  • Figure 5.12 BMS Revenues: Distribution by Therapeutic Areas, 2014 (USD Billion)
  • Figure 5.13 BMS Revenues: Distribution by Reported Segments, 2012-2014 (USD Billion)
  • Figure 5.14 Celgene Annual Revenues, 2009-2014 (USD Million)
  • Figure 5.15 Celgene Revenues, 2012-2013: Distribution by Business Segment (USD Million)
  • Figure 5.16 Celgene Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.17 Celgene Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.18 Celgene Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.19 AstraZeneca Revenues, 2009-Q3 2014 (USD Million)
  • Figure 5.20 AstraZeneca Revenues: Distribution by Therapeutic Areas, 2012 - 2013 (USD Billion)
  • Figure 5.21 AstraZeneca Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.22 AstraZeneca Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.23 AstraZeneca Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.24 Eli Lilly Revenues, 2009- 2014 (USD Million, %)
  • Figure 5.25 Eli Lilly Revenues: Distribution by Business Segments, 2012-2013 (USD Million, %)
  • Figure 5.26 Eli Lilly Human Pharmaceutical Segment Revenues: Distribution by Operating Segments, 2012- 2013 (USD Million, %)
  • Figure 5.27 Eli Lilly Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.28 Eli Lilly Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.29 Eli Lilly Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.30 Pfizer Annual Revenues, 2011-2014 (USD Million)
  • Figure 5.31 Pfizer Revenues: Distribution by Business Segments, 2014 (USD Million)
  • Figure 5.32 Pfizer Revenues: Distribution by Therapeutic Areas, 2012-2013 (USD Million)
  • Figure 5.33 Pfizer Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.34 Pfizer Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.35 GSK Annual Revenues, 2009-2014 (GBP Billion, %)
  • Figure 5.36 GSK Annual Revenues: Distribution by Business Domains, 2014-2012 (GBP Billion, %)
  • Figure 5.37 GSK Pharmaceutical Revenues: Distribution by Business Domains 2014, (GBP Billion)
  • Figure 5.38 GSK Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.39 GSK Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.40 GSK Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.41 Boehringer Ingelheim Net Revenues, 2009-2013 (EUR Million, %)
  • Figure 5.42 Boehringer Ingelheim: Distribution by Business Segments, 2012-2013 (EUR Million)
  • Figure 5.43 Boehringer Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.44 Boehringer Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.45 Boehringer Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.46 Amgen: Annual Revenues, 2009 -2014 (USD Billion)
  • Figure 5.47 Amgen Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.48 Amgen Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.49 Amgen Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.50 AbbVie Annual Revenues, 2012 -2014 (USD Billion)
  • Figure 5.51 AbbVie Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.52 AbbVie Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.53 AbbVie Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.54 Novartis Revenues, 2009-2013 (USD Billion, %)
  • Figure 5.55 Novartis Revenues: Distribution by Business Segments, 2013 (USD Billion)
  • Figure 5.56 Novartis Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.57 Novartis Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.58 Sanofi Net Revenues, 2009- 2014 (EUR Million, %)
  • Figure 5.59 Sanofi Revenues: Distribution by Business Segment, 2012-2014 (EUR Million)
  • Figure 5.60 Sanofi Revenues: Distribution by Therapeutic Areas H1, 2014 (EUR Million)
  • Figure 5.61 Genzyme Revenues, H1 2014 (EUR Million)
  • Figure 5.62 Sanofi: Revenues from Rare Disease Drugs, H1 2014 (EUR Million)
  • Figure 5.63 Sanofi Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.64 Sanofi Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.65 Sanofi Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.66 Takeda Revenues, 2010 -Q3 2015 (JPY million, %)
  • Figure 5.67 Takeda Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.68 Takeda Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.69 Takeda Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.70 Ono Pharmaceuticals Revenues, 2009-2013 (JPY Million, %)
  • Figure 5.71 Ono Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.72 Ono Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.73 Ono Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.74 Otsuka Net Revenues, 2009-Q2 2014 (JPY Million, %)
  • Figure 5.75 Otsuka Revenues: Distribution by Business Segment, 2012-Q2 2014 (JPY Million)
  • Figure 5.76 Otsuka Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.77 Otsuka Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.78 Eisai Annual Revenues, 2009-Q3 2014 (JPY Billion)
  • Figure 5.79 Eisai Revenues: Distribution by Reporting Segments, FY 2012-Q3 2015 (JPY Billion)
  • Figure 5.80 Eisai Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.81 Eisai Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.82 Eisai Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.83 Daiichi Sankyo Net Revenues, 2009 - Q3 2014 (JPY Million)
  • Figure 5.84 Daiichi Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.85 Daiichi Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.86 Daiichi Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.87 Shire Annual Revenues, 2009-Q3 2014 (USD Million)
  • Figure 5.88 Shire Revenues: Distribution by Reporting Segments, 2012-2014 (USD Million)
  • Figure 5.89 Johnson &Johnson Annual Revenues, 2009-2014 (USD Billion)
  • Figure 5.90 Johnson &Johnson Revenues: Distribution by Business Segments, 2014 (USD Billion)
  • Figure 5.91 Kyowa Hakko Kirin Annual Revenues, 2009-2014 (JPY Million, %)
  • Figure 5.92 Kyowa Kirin Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.93 Kyowa Kirin Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.94 Kyowa Kirin Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.95 Astellas Revenues, 2009- Q3 2014 (JPY Billion)
  • Figure 5.96 Astellas Revenues: Distribution by Business Segment, 2013-2014 (JPY Billion)
  • Figure 5.97 Biogen Idec Annual Revenues, 2009-2014 (USD Million)
  • Figure 5.98 Biogen Idec Revenues: Distribution by Segment, 2013-2014 (USD Million)
  • Figure 5.99 Biogen Idec Revenues: Distribution by Therapeutic Areas, 2014 (USD Million)
  • Figure 5.100 Chugai Annual Revenues, 2009-2014 (JPY Billion)
  • Figure 5.101 Chugai Revenues: Distribution by Product Domains, 2012-2014 (JPY Billion)
  • Figure 5.102 Chugai Pipeline Drugs: Distribution by Nature of Drug
  • Figure 5.103 Chugai Pipeline Drugs: Distribution by Therapeutic Areas
  • Figure 5.104 Chugai Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.105 Gilead Sciences Net Revenues, 2009 -2014 (USD Million)
  • Figure 5.106 Mitsubishi Tanabe Annual Revenues, 2009- Q3 2014 (JPY Billion)
  • Figure 5.107 Mitsubishi Tanabe Pharmaceuticals Revenues: Distribution by Segments, Q1-Q3 2014 (JPY Billion)
  • Figure 5.108 Sumitomo Dainippon Annual Revenues, FY2009-Q32014 (JPY Billion)
  • Figure 5.109 Sumitomo Dainippon Revenues: Distribution by Business Segments, FY 2012-FY 2013 (JPY Billion)
  • Figure 5.110 Sumitomo Dainippon Revenues: Distribution by Geographical Segments, FY 2012-FY 2013 (JPY Billion)
  • Figure 5.111 Chiesi Annual Revenues, 2009-2013 (EUR Million)
  • Figure 5.112 Teva Net Revenues, 2009- 2014 (USD Billion, %)
  • Figure 5.113 Teva Revenues: Distribution by Business Segments, 2012- 2014 (USD billion)
  • Figure 5.114 Teva Specialty Revenues: Distribution by Therapeutic Areas (USD Billion, 2014)
  • Figure 5.115 Teva Pipeline Drugs: Distribution by Therapeutic Area
  • Figure 5.116 Teva Pipeline Drugs: Distribution by Highest Phase of Development
  • Figure 5.117 Baxter Annual Revenues, 2009-2014 (USD Billion)
  • Figure 5.118 Baxter Revenues: Distribution by Business Segment, 2012-2014 (USD Billion)
  • Figure 5.119 Baxter Revenues: Distribution by Sub-Segments, 2014 (USD Billion)
  • Figure 5.120 Grifols Annual Revenues: 2009-Q3 2014 (EUR Million, %)
  • Figure 5.121 Grifols Revenues-Distribution by Business Segments, Q1-Q3 2014 (EUR Million, %)
  • Figure 5.122 Bayer Revenues, 2009-Q3 2014 (EUR Billion, %)
  • Figure 5.123 Bayer Revenues: Distribution by Business Segment, 2012-Q3 2014 (EUR Billion)
  • Figure 5.124 Bayer Revenues: Distribution by Healthcare Segments Q1-Q3 2014 (EUR Million)
  • Figure 5.125 Actelion Annual Revenues, 2009-2013 (CHF Million)
  • Figure 5.126 Actelion Revenues: Distribution by Business Segment, 2012-2013 (CHF Million)
  • Figure 5.127 Ipsen Revenues, 2009- Q3 2014 (EUR Million, %)
  • Figure 5.128 Ipsen Revenues: Distribution by Business Segments, 2014 (EUR Million)
  • Figure 5.129 Ipsen Specialty Revenues: Distribution by Therapeutic Area, 2013-2014 (EUR Million)
  • Figure 5.130 Ipsen Primary Care Revenues: Distribution by Therapeutic Area, 2013-2014 (EUR Million)
  • Figure 5.131 Nippon Revenues, 2009- Q3 2014 (JPY Million, %)
  • Figure 5.132 Nippon Revenues: Distribution by Business Segment, 2012-2014 (JPY Million)
  • Figure 5.133 Nippon Pharmaceutical segment Revenues: Distribution by Therapeutic Areas, 2012-2014 (JPY Million)
  • Figure 5.134 Merck Group Revenues, 2009- Q3 2014 (EUR Million, %)
  • Figure 5.135 Merck Revenues: Distribution by Business Divisions, 2013-Q3 2014(EUR Million)
  • Figure 5.136 UCB Annual Revenues, 2009-Q3 2014 (EUR Million)
  • Figure 5.137 CSL Net Revenues, 2009-2014 (USD Million, %)
  • Figure 5.138 CSL Revenues: Distribution by Business Segment, 2013-2014 (USD Million)
  • Figure 5.139 Merck Revenues, 2009-2014 (USD Billion)
  • Figure 5.140 Merck Revenues: Distribution by Business Segment, 2014 (USD Billion)
  • Figure 5.141 Alexion Revenues, 2009- 2014 (USD Million)
  • Figure 5.142 Recordati Annual Revenues, 2009-Q3 2014 (EUR Million)
  • Figure 5.143 Novo Nordisk Annual Revenues, 2009 -2014 (DKK Million)
  • Figure 5.144 Novo Nordisk Annual Revenues: Distribution by Business Segment, 2013- 2014 (DKK million)
  • Figure 5.145 Shionogi Annual Revenues, 2009-Q3 2014 (JPY Billion, %)
  • Figure 5.146 Vertex Revenues, 2009-2014 (USD Million, %)
  • Figure 5.147 Meiji Seika Revenues, 2009-Q3 2014 (JPY Billion, %)
  • Figure 5.148 Meiji Seika Revenues: Distribution by Business Segment, 2012-2014 (JPY Billion)
  • Figure 5.149 Meiji Seika Pharmaceutical Revenues: Distribution by Sub segments, Q1-Q3 2014, (JPY Billion)
  • Figure 5.150 Orion Revenues, 2009- 2014 (EUR Million, %)
  • Figure 5.151 Orion Revenues: Distribution by Business Segment, 2012-2014: (EUR Million)
  • Figure 5.152 Orion Pharmaceutical Revenues: Distribution by sub-divisions, 2012- 2014 (EUR Million)
  • Figure 5.153 Santen Annual Revenues, 2009-Q3 2014 (JPY Million)
  • Figure 5.154 Santen Revenues: Distribution by Business Segment, 2012- Q3 2014 (JPY Million)
  • Figure 5.155 Teijin Net Revenues, 2009- Q2 2015 (JPY Billion, %)
  • Figure 5.156 Teijin Revenues: Distribution by Business Segment, 2012- Q3 2015 (JPY Billion)
  • Figure 5.157 Lundbeck Annual Revenues, 2010- Q3 2014 (DKK Million, %)

List of Tables

  • Table 3.1 Rare Disease Drugs: Clinical Pipeline
  • Table 4.1 Ovarian Cancer: Key Facts
  • Table 4.2 Ovarian Cancer: Example Marketed Rare Disease Drugs
  • Table 4.3 Ovarian Cancer: Pipeline Rare Disease Drugs
  • Table 4.4 CLL: Key Facts
  • Table 4.5 CLL: Example Marketed Rare Disease Drugs
  • Table 4.6 CLL: Pipeline Rare Disease Drugs
  • Table 4.7 Multiple Myeloma: Factual Peep
  • Table 4.8 Multiple Myeloma: Example Marketed Rare Disease Drugs
  • Table 4.9 Multiple Myeloma: Pipeline Rare Disease Drugs
  • Table 4.10 Gastric Cancer: Key Facts
  • Table 4.11 Gastric Cancer: Example Marketed Rare Disease Drugs
  • Table 4.12 Gastric Cancer: Pipeline Rare Disease Drugs
  • Table 4.13 Acute Myeloid Leukaemia: Key Facts
  • Table 4.14 AML: Example Marketed Rare Disease Drugs
  • Table 4.15 AML: Pipeline Rare Disease Drugs
  • Table 4.16 Breast Cancer: Key Facts
  • Table 4.17 Metastatic Breast Cancer: Marketed Rare Disease Drugs
  • Table 4.18 Metastatic Breast Cancer: Pipeline Rare Disease Drugs
  • Table 5.1 Roche: Rare Disease Marketed Drugs
  • Table 5.2: Roche: Rare Disease Pipeline Drugs
  • Table 5.3 BMS: Rare Disease Marketed Drugs
  • Table 5.4 BMS: Rare Diseases Pipeline Drugs
  • Table 5.5 Celgene: Rare Disease Marketed Drugs
  • Table 5.6 Celgene: Rare Disease Pipeline Drugs
  • Table 5.7 AstraZeneca/ MedImmune: Rare Disease Marketed Drugs
  • Table 5.8 AstraZeneca/ MedImmune- Rare Disease Pipeline Drugs
  • Table 5.9 Eli Lilly: Rare Disease Marketed Drugs
  • Table 5.10 Eli Lilly: Rare Disease Pipeline Drugs
  • Table 5.11 Pfizer: Rare Disease Marketed Drugs
  • Table 5.12 Pfizer-Rare Disease Pipeline Drugs
  • Table 5.13 GSK: Rare Disease Marketed Drugs
  • Table 5.14 GSK: Rare Disease Pipeline Drugs
  • Table 5.15 Boehringer Ingelheim: Rare Disease Pipeline Drugs
  • Table 5.16 Amgen: Rare Disease Marketed Drugs
  • Table 5.17 Amgen: Rare Disease Pipeline Drugs
  • Table 5.18 AbbVie: Rare Disease Marketed Drugs
  • Table 5.19 AbbVie: Rare Disease Pipeline Drugs
  • Table 5.20 Novartis: Rare Disease Marketed Drugs
  • Table 5.21 Novartis: Rare Disease Pipeline Drugs
  • Table 5.22 Sanofi/ Genzyme: Rare Disease Marketed Drugs
  • Table 5.23 Sanofi/ Genzyme: Rare Disease Pipeline Drugs
  • Table 5.24 Takeda/ Millennium: Rare Disease Pipeline Drugs
  • Table 5.25 Ono Pharmaceuticals: Rare Disease Pipeline Drugs
  • Table 5.26 Otsuka: Rare Disease Pipeline Drugs
  • Table 5.27 Eisai: Rare Disease Marketed Drugs
  • Table 5.28 Eisai: Rare Disease Pipeline Drugs
  • Table 5.29 Daiichi Sankyo: Rare Disease Pipeline Drugs
  • Table 5.30 Shire: Rare Disease Marketed Drugs
  • Table 5.31 Shire: Rare Disease Pipeline Drugs
  • Table 5.32 Johnson & Johnson: Rare Disease Marketed Drugs
  • Table 5.33 Johnson & Johnson: Rare Disease Pipeline Drugs
  • Table 5.34 Kyowa Hakko Kirin: Rare Disease Pipeline Drugs
  • Table 5.35 Astellas: Rare Disease Marketed Drugs
  • Table 5.36 Astellas: Rare Disease Pipeline Drugs
  • Table 5.37 Biogen Idec: Rare Disease Marketed Drugs
  • Table 5.38 Biogen Idec: Rare Disease Pipeline Drugs
  • Table 5.39 Chugai: Rare Disease Pipeline Drugs
  • Table 5.40 Gilead Sciences: Rare Disease Marketed Drugs
  • Table 5.41 Gilead Sciences: Rare Disease Pipeline Drugs
  • Table 5.42 Mitsubishi Tanabe: Rare Disease Pipeline Drugs
  • Table 5.43 Sumitomo Dainippon: Rare Disease Pipeline Drugs
  • Table 5.44 Chiesi: Rare Disease Pipeline Drugs
  • Table 5.45 Teva: Rare Disease Marketed Drugs
  • Table 5.46 Teva: Rare Disease Pipeline Drugs
  • Table 5.47 Baxter: Rare Diseases Marketed Drugs
  • Table 5.48 Baxter: Rare Diseases Pipeline Drugs
  • Table 5.49 Grifols: Rare Disease Marketed Drugs
  • Table 5.50 Grifols: Rare Disease Pipeline Drugs
  • Table 5.51 Bayer: Rare Disease Marketed Drugs
  • Table 5.52 Bayer: Rare Disease Pipeline Drugs
  • Table 5.53 Actelion: Rare Disease Marketed Drugs
  • Table 5.54 Actelion: Rare Disease Pipeline Drugs
  • Table 5.55 Ipsen: Rare Disease Pipeline Drugs
  • Table 5.56 Nippon Shinyaku: Rare Disease Pipeline Drugs
  • Table 5.57 Merck KGaA: Rare Disease Marketed Drugs
  • Table 5.58 Merck KGaA: Rare Disease Pipeline Drugs
  • Table 5.59 UCB: Rare Disease Pipeline Drugs
  • Table 5.60 CSL: Rare Disease Marketed Drugs
  • Table 5.61 CSL: Rare Disease Pipeline Drugs
  • Table 5.62 Merck: Rare Disease Pipeline Drugs
  • Table 5.63 Merck Partnerships
  • Table 5.64 Sigma-Tau: Rare Disease Marketed Drugs
  • Table 5.65 Sigma-Tau: Rare Disease Pipeline Drugs
  • Table 5.66 Alexion: Rare Disease Marketed Drugs
  • Table 5.67 Alexion: Rare Disease Pipeline Drugs
  • Table 5.68 Recordati: Rare Disease Marketed Drugs
  • Table 5.69 Recordati: Rare Diseases Pipeline Drugs
  • Table 5.70 Novo Nordisk: Rare Disease Marketed Drugs
  • Table 5.71 Novo Nordisk: Rare Disease Pipeline Drugs
  • Table 5.72 Shionogi: Rare Disease Pipeline Drugs
  • Table 5.73 Vertex: Rare Disease Pipeline Drugs
  • Table 5.74 Meiji Seika: Rare Disease Pipeline Drugs
  • Table 5.75 Ferring: Rare Disease Pipeline Drugs
  • Table 5.76 Teijin: Rare Diseases Pipeline Drugs
  • Table 5.77 Santen: Rare Disease Pipeline Drugs
  • Table 5.78 Teijin: Rare Disease Pipeline Drugs
  • Table 5.79 Lundbeck: Rare Disease Marketed Drugs

Listed Companies

The following companiesand organizations have been mentioned in the report.

  • 1. Abbott
  • 2. AbbVie
  • 3. Abraxis Bioscience Ltd
  • 4. AC Immune
  • 5. Accleron
  • 6. Actelion
  • 7. Active Biotech
  • 8. AesRx LLC
  • 9. Agensys
  • 10. Agios Pharmaceuticals
  • 11. Alexion
  • 12. Alnara Pharmaceutical
  • 13. Alnylam
  • 14. Ambit Biosciences
  • 15. American BioScience Inc
  • 16. Amgen
  • 17. Amicus Therapeutics
  • 18. Amor Chem
  • 19. Amylin Pharmaceuticals
  • 20. Araclon Biotech
  • 21. Archimex
  • 22. ArmaGen
  • 23. ArQule Inc
  • 24. Array Biopharma
  • 25. Arresto Biosciences
  • 26. Aslan Pharmaceuticals
  • 27. Astellas Pharma Inc.
  • 28. Astex Pharmaceuticals
  • 29. AstraZeneca
  • 30. Avanir
  • 31. Aventis
  • 32. Aveo Pharmaceuticals
  • 33. Avid Radiopharmaceuticals
  • 34. Aviesan
  • 35. Babraham Institute
  • 36. Banner Alzheimer's Institute
  • 37. Barcelona Alzheimer's Treatment and Research Center
  • 38. Basilia Pharmaceuticals
  • 39. Baxter
  • 40. Bayer
  • 41. Bial Pharmaceuticals
  • 42. Bill & Melinda Gates Foundation
  • 43. BIO Ventures
  • 44. Bioarctic Neuroscience AB
  • 45. Biogen Idec
  • 46. BioMarin
  • 47. Biotie Therapies
  • 48. Biovail Laboratories International
  • 49. BioXcellence
  • 50. Blackswan Foundation
  • 51. BMS
  • 52. Boehringer Ingelheim
  • 53. Boston's Children Hospital
  • 54. Brazilian Association for Leukemia and Lymphoma
  • 55. Brazilian Association for Thalassemia
  • 56. Britannia Pharmaceuticals
  • 57. Broad Institute
  • 58. Calistoga Pharmaceuticals
  • 59. Camurus
  • 60. Cancer Research Technology
  • 61. Cambridge Institute
  • 62. Cangene Corporation
  • 63. Caritas Argentina
  • 64. Carter
  • 65. Catalyst Biosciences
  • 66. Cavis Pharma
  • 67. Cedars Sinai Medical Center
  • 68. Celgene
  • 69. Cell Therapeutics
  • 70. Celldex Therapeutics
  • 71. Center for iPS Cell Research and Application
  • 72. Centocor Inc
  • 73. Cephalon
  • 74. Ceptaris Therapeutics.
  • 75. Chatham Therapeutics
  • 76. Chelsea Therapeutics
  • 77. Chemocentryx Inc.
  • 78. Chiasma
  • 79. Chiesi
  • 80. Children's Hospital and Research Centre
  • 81. Chiron Corporation
  • 82. Chugai
  • 83. Cincinnati Children Hospital
  • 84. CIRAD
  • 85. Cleveland Clinic
  • 86. Coherus Biosciences
  • 87. Committee for Medicinal Products for Human Use
  • 88. Community Chest
  • 89. Concordia Pharmaceuticals Inc
  • 90. Cordis Corporation
  • 91. CSL
  • 92. Curie Institute
  • 93. Cystic Fibrosis Research Inc.
  • 94. Cystinosis Research Foundation
  • 95. Cytos Biotechnology
  • 96. Daiichi Sankyo
  • 97. Dainippon Pharmaceuticals
  • 98. DaniscoBioActives
  • 99. Debiopharm
  • 100. Diagnostic Division of the Shionogi &Co. Ltd
  • 101. Dimension Therapeutics
  • 102. Dong A Pharmaceuticals
  • 103. Drais Pharmaceuticals
  • 104. DS Pharma Animal Health Co
  • 105. Duke Clinical Research Institute
  • 106. Duke University
  • 107. Edison Pharmaceuticals
  • 108. Eisai
  • 109. Eli Lilly
  • 110. Enobia Pharma Corp
  • 111. EnVivo Pharmaceuticals
  • 112. Envoy Therapeutics
  • 113. Enzon Pharmaceuticals
  • 114. Epizyme Inc.
  • 115. EspeRare Foundation
  • 116. Eunefron
  • 117. European Crohn's and Colitis Organization
  • 118. European Organisation for Rare Diseases
  • 119. European Parkinson's Disease Association
  • 120. European Porphyria Network (EPNET)
  • 121. European Respiratory Society Congress
  • 122. European Society for Paediatric Research Congress
  • 123. EURORDIS
  • 124. EuroWilson
  • 125. Evotec
  • 126. Exelixis
  • 127. Ezose Sciences Inc
  • 128. Fast Forward LLC
  • 129. Ferring Pharmaceuticals
  • 130. Fondazione Telethon
  • 131. Foundation Medicine
  • 132. F-star Alpha Ltd.
  • 133. Fujisawa Pharmaceuticals
  • 134. Fumapharm AG.
  • 135. Fundacio ACE
  • 136. Galapagos
  • 137. Galderma
  • 138. Galecto Biotech AB
  • 139. GebertRuf Foundation
  • 140. Gedeon Richter Plc.
  • 141. Genentech
  • 142. Glenmark Pharmaceuticals
  • 143. Gilead Sciences
  • 144. Gladstone Institute
  • 145. GlaxoSmithKline
  • 146. Global Innovation Pharmaceuticals
  • 147. Gloucester Pharmaceuticals
  • 148. GlycoMetrics
  • 149. Grifols
  • 150. GTx Inc
  • 151. GW Pharmaceuticals Plc
  • 152. Halozyme
  • 153. Harvard and Massachusetts Institute of Technology
  • 154. Helsinn Group
  • 155. Hemobras
  • 156. Hologic, Inc
  • 157. Hospira
  • 158. HudsonAlpha Institute
  • 159. Human Combinatorial Antibody Laboratory
  • 160. Hutchison China MediTech Ltd.
  • 161. Idec Pharmaceuticals
  • 162. ILDONG Pharmaceuticals
  • 163. Immunex Corp.
  • 164. Impax Pharmaceuticals
  • 165. Inception Sciences
  • 166. Incyte Corporation
  • 167. Infinity
  • 168. Innovive Pharmaceuticals
  • 169. INSERM
  • 170. Insmed
  • 171. Inspiration Biotech
  • 172. Institute of Experimental Neurology
  • 173. Intellect Neurosciences
  • 174. Intercept Pharmaceuticals
  • 175. InterMune
  • 176. International Organization of Multiple Sclerosis Nurses
  • 177. International Pancreatic Cancer Patient Advocacy Committee
  • 178. International Union against Tuberculosis and Lung Disease
  • 179. Invida Group
  • 180. Ipsen
  • 181. Ipsos
  • 182. Iris Pharmaceuticals
  • 183. Isis Pharmaceuticals
  • 184. Italian Centre for Cystic fibrosis
  • 185. Janssen Pharmaceuticals
  • 186. Japan Research Foundation for Clinical Pharmacology
  • 187. Japan Tobacco Inc.
  • 188. Jerini AG
  • 189. Johnson & Johnson
  • 190. Jude's Children Research Institute
  • 191. Kamada Ltd.
  • 192. KEK (The High Energy Accelerator Research Organization)
  • 193. Kirin Amgen
  • 194. Kirin Brewery Company
  • 195. Kirin Co. Ltd
  • 196. Kirin-Amgen
  • 197. Kissei Pharmaceuticals Co. Ltd
  • 198. Knopp Neurosciences
  • 199. Kotobuki Pharmaceuticals Ltd
  • 200. Kyoto University
  • 201. Kyowa Hakko Kirin
  • 202. LabCorp Beijing
  • 203. Leukemic Children's Association
  • 204. Ligand Pharmaceuticals
  • 205. Liverpool School of Tropical Medicine
  • 206. Lotus Tissue Repair, Inc.
  • 207. Lumena Pharmaceuticals
  • 208. Luminex Corporation
  • 209. Lundbeck
  • 210. Lymphoma Academic Research Organization
  • 211. Macusight
  • 212. Manchester Collaborative Centre
  • 213. Marnac Inc.
  • 214. Massachusetts General Hospital
  • 215. Mayo Clinic
  • 216. Medarex
  • 217. MediKidz Limited
  • 218. MedImmune
  • 219. Medivation
  • 220. Medtronic
  • 221. Meiji Seika
  • 222. Merck
  • 223. Merck Serono
  • 224. Merrimack Pharmaceuticals
  • 225. MGI Pharma
  • 226. Michael J Fox Foundation
  • 227. Micromet
  • 228. Millennium
  • 229. Minophagen Pharmaceuticals
  • 230. Mitsubishi Tanabe Pharma
  • 231. Moderna Therapeutics
  • 232. MorphoSys AG
  • 233. Mount Sinai Medical Center
  • 234. Mylan Pharmaceuticals
  • 235. Myogen
  • 236. Nagasaki University
  • 237. National Cancer Institute
  • 238. National Eye Institute
  • 239. National Institute for Health and Care Excellence (NICE)
  • 240. National Institute of Advanced Industrial Science and Technology
  • 241. National Institute of Health
  • 242. National Multiple Sclerosis Society
  • 243. National Organization for Rare Disorders
  • 244. National University Corporation
  • 245. Natrogen
  • 246. Neurimmune Holdings AG
  • 247. NeuroSearch A/S
  • 248. Newron Pharmaceutical
  • 249. Nippon Shinyaku
  • 250. Nogra Pharma Ltd
  • 251. Novartis
  • 252. Novo Nordisk
  • 253. NPS Pharma
  • 254. Oncodesign
  • 255. Oncogenex Pharmaceuticals
  • 256. Onconova Therapeutics
  • 257. Online Education Research Center
  • 258. Ono Pharmaceuticals
  • 259. Onyx Pharmaceutical
  • 260. Opexa Therapeutics
  • 261. Optimer Pharmaceuticals
  • 262. Orion Pharma
  • 263. Orphan Disease Treatment Institute Co Ltd.
  • 264. Orphan Europe
  • 265. Orphatec Pharmaceuticals
  • 266. Ortho-Clinical Diagnostic Inc
  • 267. Oswald Cruz Foundation
  • 268. Otsuka Pharmaceuticals
  • 269. Ovation Pharmaceuticals
  • 270. Oxford Biomedica
  • 271. Oxford GlycoSciences
  • 272. Oryzon
  • 273. Pancreatic Cancer Action Network
  • 274. PATH Organisation
  • 275. Patient Advocate Foundation
  • 276. PDL BioPharma
  • 277. Peptimmune Inc.
  • 278. Pfizer
  • 279. Pharmacia
  • 280. Pharmacyclics
  • 281. PharmaLink AB
  • 282. PharmaSwiss S.A
  • 283. Pink Ribbon Red Ribbon Foundation
  • 284. Plexxikon
  • 285. Premacure AB
  • 286. ProStrakan Group plc
  • 287. Protalix
  • 288. PTC Therapeutics
  • 289. Pulmonary Fibrosis Foundation
  • 290. Qiagen
  • 291. Ranbaxy
  • 292. Recordati
  • 293. Repligen Corp
  • 294. Roche Laboratories
  • 295. Roche's Translational Clinical Research Center
  • 296. Royalty Pharma
  • 297. Saegis
  • 298. Sailing Sclerosis Foundation
  • 299. Saladax Biomedical
  • 300. Samsung Medical Center
  • 301. San Raffaele University and Research Hospital
  • 302. Sangamo Biosciences
  • 303. Sanofi- Aventis
  • 304. Sanofi- Synthelabo
  • 305. Sanquin Blood Supply Foundation
  • 306. Santen
  • 307. Santhera Pharmaceuticals
  • 308. Schering
  • 309. Seaside Therapeutics
  • 310. Seattle Genetics
  • 311. Sekisui Medical Ltd.
  • 312. Sepracor Inc
  • 313. Seragon Pharmaceuticals
  • 314. Server &Xoma
  • 315. Shionogi &Co. Ltd
  • 316. Shire
  • 317. Siemens Medical Solution
  • 318. Sigma-Tau Pharmaceuticals
  • 319. Sobi
  • 320. Solasia Pharma Ltd
  • 321. Spanish National Cancer Research Centre
  • 322. Sumitomo Dainippon
  • 323. Sunvonion Pharmaceuticals
  • 324. Swiss Tropical and Public Health Institute
  • 325. SWOG cancer institute
  • 326. Synageva Biopharma
  • 327. Syntonix Pharmaceuticals Inc
  • 328. Taiho Pharmaceuticals
  • 329. Takeda
  • 330. Talecris
  • 331. TargeGen
  • 332. Teijin Pharma
  • 333. Teikoku Pharma
  • 334. Telethon's Institute of Genetics and Medicine
  • 335. Teva
  • 336. The Eunice Kennedy Shriver National Institute Of Child Health and Human Development
  • 337. The European Cystic Fibrosis Society
  • 338. The KarolinskaInstitute
  • 339. The National Human Genome Research Institute
  • 340. The Spanish Federation of Parkinson's Disease
  • 341. Threshold Pharmaceuticals
  • 342. Tillots Pharma AG
  • 343. Tokyo Institute of Technology
  • 344. U3-Pharma
  • 345. UCB
  • 346. Ultragenyx Pharmaceuticals
  • 347. UniQure
  • 348. University College London
  • 349. University of California, San Francisco
  • 350. University of Cambridge
  • 351. University of Cape Town
  • 352. University of Chicago Medical Center
  • 353. University of Illinois
  • 354. University of Liverpool
  • 355. University of Manchester
  • 356. University of Texas MD Anderson Cancer Centre
  • 357. University of Tokyo
  • 358. University of Tsukuba
  • 359. Upjohn
  • 360. Valeant Pharmaceuticals
  • 361. Valld'Hebron Research Institute
  • 362. Vanderbilt University
  • 363. Veloxis Pharmaceuticals
  • 364. Veridex LLC
  • 365. Vernalis Inc.
  • 366. Versant
  • 367. Vertex Pharmaceuticals
  • 368. Ventana Medical Systems
  • 369. ViroPharma
  • 370. Warner Lambert
  • 371. Weill Cornell Medical College
  • 372. World Health Organization
  • 373. World Bank
  • 374. World Federation of Hemophilia
  • 375. World Intellectual Property Organization
  • 376. XeneticBioSciences
  • 377. Xenon
  • 378. Yamanounchi Pharmaceuticals
  • 379. YM Biosciences
  • 380. Zeltia
  • 381. ZenyakuKogyu Co. Ltd
  • 382. Zeria Pharmaceuticals
  • 383. Zinfandel Pharmaceuticals
  • 384. Zymenex
  • 385. Zymo Genetic
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