• 1-800-526-8630U.S. (TOLL FREE)
  • 1-917-300-0470EAST COAST U.S.
  • +353-1-416-8900REST OF WORLD
Urea Cycle Disorders - Pipeline Review, H2 2012 Product Image

Urea Cycle Disorders - Pipeline Review, H2 2012

  • ID: 2244973
  • September 2012
  • 43 pages
  • Global Markets Direct

Urea Cycle Disorders – Pipeline Review, H2 2012

Summary

Global Markets Direct’s, 'Urea Cycle Disorders - Pipeline Review, H2 2012', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Urea Cycle 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 Urea Cycle Disorders. Urea Cycle 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 Urea Cycle Disorders.
- A review of the Urea Cycle Disorders products under development by companies and universities/research READ MORE >

Note: Product cover images may vary from those shown

2
List of Tables 4
List of Figures 4
Introduction 5
REPORT COVERAGE 5
Urea Cycle Disorders Overview 6
Therapeutics Development 7
An Overview of Pipeline Products for Urea Cycle Disorders 7
Urea Cycle Disorders Therapeutics under Development by Companies 9
Urea Cycle Disorders Therapeutics under Investigation by Universities/Institutes 10
Late Stage Products 11
Comparative Analysis 11
Mid Clinical Stage Products 12
Comparative Analysis 12
Early Clinical Stage Products 13
Comparative Analysis 13
Urea Cycle Disorders Therapeutics – Products under Development by Companies 14
Urea Cycle Disorders Therapeutics – Products under Investigation by Universities/Institutes 15
Companies Involved in Urea Cycle Disorders Therapeutics Development 16
Aspen Pharmacare Holdings Ltd 16
Ocera Therapeutics, Inc. 17
Hyperion Therapeutic, Inc. 18
Promethera Biosciences 19
Urea Cycle Disorders – Therapeutics Assessment 20
Assessment by Monotherapy Products 20
Assessment by Route of Administration 21
Assessment by Molecule Type 23
Drug Profiles 25
glycerol phenylbutyrate - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
ornithine phenylacetate - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
carglumic acid - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Human Heterologous Liver Cells - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
carglumic acid - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Promethera HepaStem - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Urea Cycle Disorders Therapeutics – Drug Profile Updates 34
Urea Cycle Disorders – Product Development Milestones 37
Featured News & Press Releases 37
May 14, 2012: Promethera Biosciences Treats First Patients With Promethera HepaStem 37
Oct 19, 2011: Cytonet's Liver Cell Therapy Trial For Children With UCD Expands To Canada 37
May 17, 2011: Cytonet's Liver Cell Therapy Receives Orphan Drug Designation From FDA 38
Apr 13, 2011: Cytonet Launches Phase II Trial For Hereditary Urea Cycle Disorders (UCD) In Children 38
Nov 02, 2010: Hyperion' Glycerol Phenylbutyrate Meets Primary Endpoint In Phase III Pivotal Study In Urea Cycle Disorders 39
Oct 18, 2010: Hyperion Receives FDA Fast Track Designation For Glycerol Phenylbutyrate For Treatment Of Urea Cycle Disorders 40
Nov 02, 2009: UCL (University College London) Liver Failure Group Present Two Studies On A Novel Treatment Option (OCR-002) For Complications Of Hyperammonemia In Liver Failure At The 60th Annual AASLD Meeting 40
Feb 18, 2005: Medicis Announces FDA Approval of AMMONUL for Urea Cycle Disorder 41
Appendix 42
Methodology 42
Coverage 42
Secondary Research 42
Primary Research 42
Expert Panel Validation 42
Contact Us 43
Disclaimer 43

List of Tables
Number of Products Under Development for Urea Cycle Disorders, H2 2012 7
Products under Development for Urea Cycle Disorders – Comparative Analysis, H2 2012 8
Number of Products under Development by Companies, H2 2012 9
Number of Products under Investigation by Universities/Institutes, H2 2012 10
Comparative Analysis by Late Stage Development, H2 2012 11
Comparative Analysis by Mid Clinical Stage Development, H2 2012 12
Comparative Analysis by Early Clinical Stage Development, H2 2012 13
Products under Development by Companies, H2 2012 14
Products under Investigation by Universities/Institutes, H2 2012 15
Aspen Pharmacare Holdings Ltd, H2 2012 16
Ocera Therapeutics, Inc., H2 2012 17
Hyperion Therapeutic, Inc., H2 2012 18
Promethera Biosciences, H2 2012 19
Assessment by Monotherapy Products, H2 2012 20
Assessment by Stage and Route of Administration, H2 2012 22
Assessment by Stage and Molecule Type, H2 2012 24
Urea Cycle Disorders Therapeutics – Drug Profile Updates 34

List of Figures
Number of Products under Development for Urea Cycle Disorders, H2 2012 7
Products under Development for Urea Cycle Disorders – Comparative Analysis, H2 2012 8
Products under Development by Companies, H2 2012 9
Products under Investigation by Universities/Institutes, H2 2012 10
Late Stage Products, H2 2012 11
Mid Clinical Stage Products, H2 2012 12
Early Clinical Stage Products, H2 2012 13
Assessment by Monotherapy Products, H2 2012 20
Assessment by Route of Administration, H2 2012 21
Assessment by Stage and Route of Administration, H2 2012 22
Assessment by Molecule Type, H2 2012 23
Assessment by Stage and Molecule Type, H2 2012 24

Note: Product cover images may vary from those shown
Note: Product cover images may vary from those shown

RELATED PRODUCTS

Our Clients

Our clients' logos