Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2013

Market: Pharmaceuticals and Healthcare

Global, 35 pages report, published by Global Markets Direct

Keywords : Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutic Products under Development, Key Players in Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics, Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline Overview, Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline, Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline Assessment

Report ThumbnailSeptember-2013
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2013

Summary

Global Markets Direct’s, 'Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2013', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C. Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C.
- A review of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C.
- 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 and scope.
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.
  • List of Figures
  • Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2013 7
  • Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H2 2013 8
  • Products under Development by Companies, H2 2013 9
  • Products under Investigation by Universities/Institutes, H2 2013 10
  • Early Clinical Stage Products, H2 2013 11
  • Discovery and Pre-Clinical Stage Products, H2 2013 12
  • Assessment by Monotherapy Products, H2 2013 17
  • Assessment by Route of Administration, H2 2013 18
  • Assessment by Stage and Route of Administration, H2 2013 19
  • Assessment by Molecule Type, H2 2013 20
  • Assessment by Stage and Molecule Type, H2 2013 21
  • List of Tables
  • Number of Products Under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2013 7
  • Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H2 2013 8
  • Number of Products under Development by Companies, H2 2013 9
  • Number of Products under Investigation by Universities/Institutes, H2 2013 10
  • Comparative Analysis by Early 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
  • Products under Investigation by Universities/Institutes, H2 2013 14
  • BioMarin Pharmaceutical Inc., H2 2013 15
  • La Jolla Pharmaceutical Company, H2 2013 16
  • Assessment by Monotherapy Products, H2 2013 17
  • Assessment by Stage and Route of Administration, H2 2013 19
  • Assessment by Stage and Molecule Type, H2 2013 21
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Drug Profile Updates 28
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Dormant Products 29
  • Table of Contents
  • Table of Contents 2
  • List of Tables 4
  • List of Figures 4
  • Introduction 5
  • Global Markets Direct Report Coverage 5
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview 6
  • Therapeutics Development 7
  • An Overview of Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 7
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Development by Companies 9
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Investigation by Universities/Institutes 10
  • Early Clinical Stage Products 11
  • Comparative Analysis 11
  • Discovery and Pre-Clinical Stage Products 12
  • Comparative Analysis 12
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Products under Development by Companies 13
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Products under Investigation by Universities/Institutes 14
  • Companies Involved in Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics Development 15
  • BioMarin Pharmaceutical Inc. 15
  • La Jolla Pharmaceutical Company 16
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment 17
  • Assessment by Monotherapy Products 17
  • Assessment by Route of Administration 18
  • Assessment by Molecule Type 20
  • Drug Profiles 22
  • Ganglioside Inhibitor Program - Drug Profile 22
  • Product Description 22
  • Mechanism of Action 22
  • R&D Progress 22
  • Drug For Niemann-Pick C Disease - Drug Profile 23
  • Product Description 23
  • Mechanism of Action 23
  • R&D Progress 23
  • Hydroxypropyl Beta Cyclodextrin - Drug Profile 24
  • Product Description 24
  • Mechanism of Action 24
  • R&D Progress 24
  • ML-SA1 - Drug Profile 25
  • Product Description 25
  • Mechanism of Action 25
  • R&D Progress 25
  • OR-0005 - Drug Profile 26
  • Product Description 26
  • Mechanism of Action 26
  • R&D Progress 26
  • LJPC-0712 - Drug Profile 27
  • Product Description 27
  • Mechanism of Action 27
  • R&D Progress 27
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Drug Profile Updates 28
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Dormant Products 29
  • Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 30
  • Featured News & Press Releases 30
  • Jul 17, 2013: La Jolla Pharmaceutical Company Receives Orphan Designation From FDA 30
  • Apr 03, 2013: Old Drug Offers New Treatment For Niemann-Pick Type C, Study Finds 31
  • Feb 21, 2013: Orphazyme Receives Orphan Drug Designation For OR0005 In EU 32
  • Jan 23, 2013: NIH Clinical Trial Begins For Treatment Of Niemann-Pick Disease Type C1 32
  • Appendix 34
  • Methodology 34
  • Coverage 34
  • Secondary Research 34
  • Primary Research 34
  • Expert Panel Validation 34
  • Contact Us 35
  • Disclaimer 35

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