Gauchers Disease - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 74 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Gaucher's Disease - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Gaucher's Disease - Pipeline Review, H2 2014', provides an overview of the Gaucher's Disease's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Gaucher's Disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Gaucher's Disease and special features on late-stage and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Gaucher's Disease
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in the therapeutics development for Gaucher's Disease and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- 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
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Gaucher's Disease pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Gaucher's Disease
- Plan mergers and acquisitions effectively by identifying key 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
Gaucher's Disease - Products under Development by Companies 17
Gaucher's Disease - Products under Investigation by Universities/Institutes 18
Gaucher's Disease - Companies Involved in Therapeutics Development 19
Amicus Therapeutics, Inc. 19
Biosidus S.A. 20
Dong-A Socio Group 21
ExSAR Corporation 22
Genzyme Corporation 23
greenovation Biotech GmbH 24
JCR Pharmaceuticals Co., Ltd. 25
Lixte Biotechnology Holdings, Inc. 26
Neuraltus Pharmaceuticals, Inc. 27
Pharming Group N.V. 28
Protalix BioTherapeutics, Inc. 29
UAB Profarma 30
Gaucher's Disease - Therapeutics Assessment 31
Assessment by Monotherapy Products 31
Assessment by Combination Products 32
Assessment by Target 33
Assessment by Mechanism of Action 35
Assessment by Route of Administration 37
Assessment by Molecule Type 39
Drug Profiles 41
afegostat tartrate + ERT - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
ambroxol - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
DOS-000011 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
eliglustat tartrate - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Gluco-Cerebosidase - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
imiglucerase biosimilar - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
imiglucerase biosimilar - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
imiglucerase biosimilar - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
JR-101 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
LB-201 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
LB-205 - 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
PRX-112 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Recombinant Enzyme for Gaucher's Disease - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Recombinant Enzyme to Replace Beta-Cerebrosidase for Gaucher's Disease - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Gaucher's Disease - Recent Pipeline Updates 60
Gaucher's Disease - Dormant Projects 64
Gaucher's Disease - Product Development Milestones 65
Featured News & Press Releases 65
Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 65
Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 65
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 66
Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 67
Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 68
Dec 11, 2013: FDA Grants Priority Review for Genzyme's Cerdelga, an Investigational Oral Therapy for Gaucher Disease 69
Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 69
Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 70
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
Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 71
Appendix 73
Methodology 73
Coverage 73
Secondary Research 73
Primary Research 73
Expert Panel Validation 73
Contact Us 74
Disclaimer 74

List of Tables
Number of Products under Development for Gaucher's Disease, H2 2014 9
Number of Products under Development for Gaucher's Disease - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 12
Number of Products under Investigation by Universities/Institutes, H2 2014 13
Comparative Analysis by Late Stage Development, H2 2014 14
Comparative Analysis by Clinical Stage Development, H2 2014 15
Comparative Analysis by Early Stage Development, H2 2014 16
Products under Development by Companies, H2 2014 17
Products under Investigation by Universities/Institutes, H2 2014 18
Gaucher's Disease - Pipeline by Amicus Therapeutics, Inc., H2 2014 19
Gaucher's Disease - Pipeline by Biosidus S.A., H2 2014 20
Gaucher's Disease - Pipeline by Dong-A Socio Group, H2 2014 21
Gaucher's Disease - Pipeline by ExSAR Corporation, H2 2014 22
Gaucher's Disease - Pipeline by Genzyme Corporation, H2 2014 23
Gaucher's Disease - Pipeline by greenovation Biotech GmbH, H2 2014 24
Gaucher's Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2014 25
Gaucher's Disease - Pipeline by Lixte Biotechnology Holdings, Inc., H2 2014 26
Gaucher's Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2014 27
Gaucher's Disease - Pipeline by Pharming Group N.V., H2 2014 28
Gaucher's Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2014 29
Gaucher's Disease - Pipeline by UAB Profarma, H2 2014 30
Assessment by Monotherapy Products, H2 2014 31
Assessment by Combination Products, H2 2014 32
Number of Products by Stage and Target, H2 2014 34
Number of Products by Stage and Mechanism of Action, H2 2014 36
Number of Products by Stage and Route of Administration, H2 2014 38
Number of Products by Stage and Molecule Type, H2 2014 40
Gaucher's Disease Therapeutics - Recent Pipeline Updates, H2 2014 60
Gaucher's Disease - Dormant Projects, H2 2014 64