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Inherited Metabolic Disorders - Pipeline Review, H2 2013

  • July 2013
  • -
  • Global Markets Direct
  • -
  • 34 pages

Inherited Metabolic Disorders - Pipeline Review, H2 2013

Summary

Global Markets Direct’s, 'Inherited Metabolic Disorders - Pipeline Review, H2 2013', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Inherited Metabolic Disorders, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Inherited Metabolic Disorders. Inherited Metabolic Disorders - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Inherited Metabolic Disorders.
- A review of the Inherited Metabolic Disorders products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Inherited Metabolic Disorders pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Inherited Metabolic Disorders.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Inherited Metabolic Disorders pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential scope.
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.

Table Of Contents

Inherited Metabolic Disorders - Pipeline Review, H2 2013
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Inherited Metabolic Disorders Overview 6
Therapeutics Development 7
An Overview of Pipeline Products for Inherited Metabolic Disorders 7
Inherited Metabolic Disorders Therapeutics under Development by Companies 9
Late Stage Products 10
Comparative Analysis 10
Mid Clinical Stage Products 11
Comparative Analysis 11
Discovery and Pre-Clinical Stage Products 12
Comparative Analysis 12
Inherited Metabolic Disorders Therapeutics - Products under Development by Companies 13
Companies Involved in Inherited Metabolic Disorders Therapeutics Development 14
Menarini Group 14
Raptor Pharmaceuticals Corp. 15
Agios Pharmaceuticals 16
Asklepion Pharmaceuticals, LLC. 17
Inherited Metabolic Disorders - Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Route of Administration 19
Assessment by Molecule Type 21
Drug Profiles 23
fomepizole - Drug Profile 23
Product Description 23
Mechanism of Action 23
RandD Progress 23
cholic acid - Drug Profile 25
Product Description 25
Mechanism of Action 25
RandD Progress 25
MEN-1128 - Drug Profile 26
Product Description 26
Mechanism of Action 26
RandD Progress 26
IEM-1 - Drug Profile 27
Product Description 27
Mechanism of Action 27
RandD Progress 27
Drug For Inborn Errors Of Metabolism - Drug Profile 28
Product Description 28
Mechanism of Action 28
RandD Progress 28
LUM-001 - Drug Profile 29
Product Description 29
Mechanism of Action 29
RandD Progress 29
Inherited Metabolic Disorders Therapeutics - Drug Profile Updates 30
Inherited Metabolic Disorders Therapeutics - Discontinued Products 31
Inherited Metabolic Disorders Therapeutics - Dormant Products 32
Appendix 33
Methodology 33
Coverage 33
Secondary Research 33
Primary Research 33
Expert Panel Validation 33
Contact Us 34
Disclaimer 34

List of Tables

Number of Products Under Development for Inherited Metabolic Disorders, H2 2013 7
Products under Development for Inherited Metabolic Disorders - Comparative Analysis, H2 2013 8
Number of Products under Development by Companies, H2 2013 9
Comparative Analysis by Late Stage Development, H2 2013 10
Comparative Analysis by Mid Clinical Stage Development, H2 2013 11
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 12
Products under Development by Companies, H2 2013 13
Menarini Group, H2 2013 14
Raptor Pharmaceuticals Corp., H2 2013 15
Agios Pharmaceuticals, H2 2013 16
Asklepion Pharmaceuticals, LLC., H2 2013 17
Assessment by Monotherapy Products, H2 2013 18
Assessment by Stage and Route of Administration, H2 2013 20
Assessment by Stage and Molecule Type, H2 2013 22
Inherited Metabolic Disorders Therapeutics - Drug Profile Updates 30
Inherited Metabolic Disorders Therapeutics - Discontinued Products 31
Inherited Metabolic Disorders Therapeutics - Dormant Products 32

List of Figures

Number of Products under Development for Inherited Metabolic Disorders, H2 2013 7
Products under Development for Inherited Metabolic Disorders - Comparative Analysis, H2 2013 8
Products under Development by Companies, H2 2013 9
Late Stage Products, H2 2013 10
Mid Clinical Stage Products, H2 2013 11
Discovery and Pre-Clinical Stage Products, H2 2013 12
Assessment by Monotherapy Products, H2 2013 18
Assessment by Route of Administration, H2 2013 19
Assessment by Stage and Route of Administration, H2 2013 20
Assessment by Molecule Type, H2 2013 21
Assessment by Stage and Molecule Type, H2 2013 22

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