Europe Orphan Drug Pipeline Analysis 2014

Publish Date:- Jan-2014       No Of Pages (1430)

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Rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. The term “orphan” was associated with these drugs in order to reflect the lack of interest by pharma companies. In the European Union, orphan drugs are known as orphan medicinal products. Many treatments which are commercially available in today’s world lack the ability to address most of the rare disease indications. This throws open significant potential and opportunities in the European Union and other regions of the world which are currently untapped.

The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening and impact lesser than 5 people in every 10,000 people. However, as a group, these diseases together could possibly affect close to 30 million people directly or indirectly in Europe. This shows the significant of the need to develop an increasing number of orphan medicinal products in the European region.

Europe Orphan Drug Pipeline Analysis 2014” by PNS Pharma gives comprehensive insight on the various orphan designated drugs being developed in Europe. Research report covers all orphan designated drugs being developed in various clinical phases. This report enables pharmaceutical companies, collaborators and other associated stake holders to identify and analyze the available investment opportunity in the Europe orphan designated drug market based upon development process.

Following parameters for each drug profile in development phase are covered in “Europe Orphan Drug Pipeline Analysis 2014” research report:

• Drug Profile Overview

• Active Indication

• Phase of Development

• Country for Clinical Trial

• Owner / Originator/ Licensee/Collaborator

• Administrative Route

• Drug Class

• Patent Information

• Molecular Formula

• Brand Names

• Development Agreements

• ATC Codes



Number of Orphan Designated Drugs in Pipeline by Clinical Phase:

• Preclinical: 35

• Clinical: 2

• Phase-I: 27

• Phase-I/II: 49

• Phase-II: 92

• Phase-II/III: 16

• Phase-III: 86

• Preregistration: 24

• Registered: 21

• Marketed: 75

• Unknown: 1

• No Development Reported: 5

• Suspended: 6

• Discontinued: 80

• Application Withdrawn: 4


Rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. The term “orphan” was associated with these drugs in order to reflect the lack of interest by pharma companies. In the European Union, orphan drugs are known as orphan medicinal products. Many treatments which are commercially available in today’s world lack the ability to address most of the rare disease indications. This throws open significant potential and opportunities in the European Union and other regions of the world which are currently untapped.

The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening and impact lesser than 5 people in every 10,000 people. However, as a group, these diseases together could possibly affect close to 30 million people directly or indirectly in Europe. This shows the significant of the need to develop an increasing number of orphan medicinal products in the European region.

Europe Orphan Drug Pipeline Analysis 2014” by PNS Pharma gives comprehensive insight on the various orphan designated drugs being developed in Europe. Research report covers all orphan designated drugs being developed in various clinical phases. This report enables pharmaceutical companies, collaborators and other associated stake holders to identify and analyze the available investment opportunity in the Europe orphan designated drug market based upon development process.

Following parameters for each drug profile in development phase are covered in “Europe Orphan Drug Pipeline Analysis 2014” research report:

• Drug Profile Overview

• Active Indication

• Phase of Development

• Country for Clinical Trial

• Owner / Originator/ Licensee/Collaborator

• Administrative Route

• Drug Class

• Patent Information

• Molecular Formula

• Brand Names

• Development Agreements

• ATC Codes



Number of Orphan Designated Drugs in Pipeline by Clinical Phase:

• Preclinical: 35

• Clinical: 2

• Phase-I: 27

• Phase-I/II: 49

• Phase-II: 92

• Phase-II/III: 16

• Phase-III: 86

• Preregistration: 24

• Registered: 21

• Marketed: 75

• Unknown: 1

• No Development Reported: 5

• Suspended: 6

• Discontinued: 80

• Application Withdrawn: 4

1. Europe Orphan Drug Market Overview

  1.1 Orphan Drug Market Intoduction

  1.2 Factors Driving Popularity of Orphan Drugs in Europe

  1.3 Orphan Drug Market Challenges

  1.4 Orphan Drug Market Regulation



2. Orphan Drug Clinical Development Phase: Unknown

  2.1 Overview

  2.2 Orphan Drug Profile in Clinical Phase



3. Orphan Drug Clinical Development Phase: Preclinical

  3.1 Overview

  3.2 Orphan Drug Profile in Clinical Phase



4. Orphan Drug Clinical Development Phase: Clinical

  4.1 Overview

  4.2 Orphan Drug Profile in Clinical Phase



5. Orphan Drug Clinical Development Phase: Phase-I

  5.1 Overview

  5.2 Orphan Drug Profile in Clinical Phase



6. Orphan Drug Clinical Development Phase: Phase-I/II

  6.1 Overview

  6.2 Orphan Drug Profile in Clinical Phase



7. Orphan Drug Clinical Development Phase: Phase-II

  7.1 Overview

  7.2 Orphan Drug Profile in Clinical Phase



8. Orphan Drug Clinical Development Phase: Phase-II/III

  8.1 Overview

  8.2 Orphan Drug Profile in Clinical Phase



9. Orphan Drug Clinical Development Phase: Phase-III

  9.1 Overview

  9.2 Orphan Drug Profile in Clinical Phase



10. Orphan Drug Clinical Development Phase: Preregistration

  10.1 Overview

  10.2 Orphan Drug Profile in Clinical Phase



11. Orphan Drug Clinical Development Phase: Registered

  11.1 Overview

  11.2 Orphan Drug Profile in Clinical Phase



12. Marketed

  12.1 Overview

  12.2 Marketed Orphan Drug Profile in Market



13. Suspended & Discontinued Orphan Drug Profiles

  13.1 No Development Reported

  13.2 Discontinued Orphan Drug Profiles

  13.3 Suspended Orphan Drug Profile

  13.4 Applications Withdrawl


Each Drug Profile has Tables Representing Following Information:

• Alternate Names

• Originator & Owner

• Collaborator

• Technology Provider

• Licensee

• Highest Development Phase

• Indications

• Class

• Mechanism of Action

• ATC Code

• Designated Brand Name