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Gauchers Disease - Pipeline Review, H2 2013

Published By :

Global Markets Direct

Published Date : Dec 2013

Category :

Metabolic Disorders

No. of Pages : 78 Pages


Global Markets Directs, \'Gaucher\'s Disease Pipeline Review, H2 2013\', provides an overview of the indications therapeutic pipeline. This report provides information on the therapeutic development for Gaucher\'s Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Gaucher\'s Disease. Gaucher\'s Disease Pipeline Review, Half Year is built using data and information sourced from Global Markets Directs 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 Directs 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 Gaucher\'s Disease.
  • A review of the Gaucher\'s Disease 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 Gaucher\'s Disease 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 Gaucher\'s Disease.
  • 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 Gaucher\'s Disease 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.
Table of Contents

Table of Contents 2

List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Gaucher\'s Disease Overview 8
Therapeutics Development 9
Pipeline Products for Gaucher\'s Disease - Overview 9
Pipeline Products for Gaucher\'s Disease - Comparative Analysis 10
Gaucher\'s Disease - Therapeutics under Development by Companies 11
Gaucher\'s Disease - Therapeutics under Investigation by Universities/Institutes 13
Gaucher\'s Disease - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Unknown Stage Products 17
Gaucher\'s Disease - Products under Development by Companies 18
Gaucher\'s Disease - Products under Investigation by Universities/Institutes 19
Gaucher\'s Disease - Companies Involved in Therapeutics Development 20
Genzyme Corporation 20
Amicus Therapeutics, Inc. 21
Lixte Biotechnology Holdings, Inc. 22
Protalix BioTherapeutics, Inc. 23
JCR Pharmaceuticals Co., Ltd. 24
greenovation Biotech GmbH 25
Neuraltus Pharmaceuticals, Inc. 26
Biosidus S.A. 27
ExSAR Corporation 28
Bioorganic Research and Services S.A. 29
Dong-A Socio Holdings Co Ltd 30
Gaucher\'s Disease - Therapeutics Assessment 31
Assessment by Monotherapy Products 31
Assessment by Target 32
Assessment by Mechanism of Action 35
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Assessment by Therapeutic Class 42
Drug Profiles 45
eliglustat tartrate - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
acetylcysteine - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
ambroxol - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
PRX-112 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
AT-3375 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
JR-101 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
NP-003 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
imiglucerase Biosimilar - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
LB-201 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
LB-205 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Gluco-Cerebosidase - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
BNT-001 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Glucocerebrosidase Activators - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
DOS-000011 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
imiglucerase Biosimilar - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Gaucher Disease Program - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Gaucher\'s Disease - Recent Pipeline Updates 64
Gaucher\'s Disease - Dormant Projects 68
Gaucher\'s Disease - Discontinued Products 69
Gaucher\'s Disease - Product Development Milestones 70
Featured News & Press Releases 70
Dec 11, 2013: FDA Grants Priority Review for Genzymes Cerdelga, an Investigational Oral Therapy for Gaucher Disease 70
Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 70
Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 71
Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease 71
Feb 07, 2013: Amicus Therapeutics To Present Data On Its Pharmacological Chaperones At Lysosomal Disease Network WORLD Symposium 72
Aug 15, 2012: Orphazyme Receives European Patent Covering Use Of HSP70 For Treatment Of Lysosomal Storage Diseases 73
Feb 09, 2012: Amicus Therapeutics Presents Preclinical Studies Of Chaperone AT3375 For Gaucher Disease 73
Dec 19, 2011: Lixte\'s LB-205 Shows Therapeutic Potential For Gaucher Disease 74
Feb 18, 2011: Genzyme Reports Promising Three-Year Follow-Up Data From Phase II Gaucher Disease Trial 74
Nov 09, 2010: Actelion Receives Approval For Zavesca For Reimbursement Under Ontario Public Drug Program 76

Appendix 77
Methodology 77
Coverage 77
Secondary Research 77
Primary Research 77
Expert Panel Validation 77
Contact Us 78
Disclaimer 78

List of Table


Number of Products under Development for Gaucher\'s Disease, H2 2013 9
Number of Products under Development for Gaucher\'s Disease - Comparative Analysis, H2 2013 10
Number of Products under Development by Companies, H2 2013 12
Number of Products under Investigation by Universities/Institutes, H2 2013 13
Comparative Analysis by Late Stage Development, H2 2013 14
Comparative Analysis by Clinical Stage Development, H2 2013 15
Comparative Analysis by Early Stage Development, H2 2013 16
Comparative Analysis by Unknown Stage Development, H2 2013 17
Products under Development by Companies, H2 2013 18
Products under Investigation by Universities/Institutes, H2 2013 19
Gaucher\'s Disease - Pipeline by Genzyme Corporation, H2 2013 20
Gaucher\'s Disease - Pipeline by Amicus Therapeutics, Inc., H2 2013 21
Gaucher\'s Disease - Pipeline by Lixte Biotechnology Holdings, Inc., H2 2013 22
Gaucher\'s Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2013 23
Gaucher\'s Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2013 24
Gaucher\'s Disease - Pipeline by greenovation Biotech GmbH, H2 2013 25
Gaucher\'s Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2013 26
Gaucher\'s Disease - Pipeline by Biosidus S.A., H2 2013 27
Gaucher\'s Disease - Pipeline by ExSAR Corporation, H2 2013 28
Gaucher\'s Disease - Pipeline by Bioorganic Research and Services S.A., H2 2013 29
Gaucher\'s Disease - Pipeline by Dong-A Socio Holdings Co Ltd, H2 2013 30
Assessment by Monotherapy Products, H2 2013 31
Number of Products by Stage and Target, H2 2013 34
Number of Products by Stage and Mechanism of Action, H2 2013 37
Number of Products by Stage and Route of Administration, H2 2013 39
Number of Products by Stage and Molecule Type, H2 2013 41
Number of Products by Stage and Therapeutic Class, H2 2013 44
Gaucher\'s Disease Therapeutics - Recent Pipeline Updates, H2 2013 64
Gaucher\'s Disease - Dormant Projects, H2 2013 68
Gaucher\'s Disease - Discontinued Products, H2 2013 69

List of Chart


Number of Products under Development for Gaucher\'s Disease, H2 2013 9
Number of Products under Development for Gaucher\'s Disease - Comparative Analysis, H2 2013 10
Number of Products under Development by Companies, H2 2013 11
Number of Products under Investigation by Universities/Institutes, H2 2013 13
Comparative Analysis by Clinical Stage Development, H2 2013 15
Comparative Analysis by Early Stage Products, H2 2013 16
Assessment by Monotherapy Products, H2 2013 31
Number of Products by Top 10 Target, H2 2013 32
Number of Products by Stage and Top 10 Target, H2 2013 33
Number of Products by Top 10 Mechanism of Action, H2 2013 35
Number of Products by Stage and Top 10 Mechanism of Action, H2 2013 36
Number of Products by Top 10 Route of Administration, H2 2013 38
Number of Products by Stage and Top 10 Route of Administration, H2 2013 39
Number of Products by Top 10 Molecule Type, H2 2013 40
Number of Products by Stage and Top 10 Molecule Type, H2 2013 41
Number of Products by Top 10 Therapeutic Class, H2 2013 42
Number of Products by Stage and Top 10 Therapeutic Class, H2 2013 43

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