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Gaucher's Disease - Pipeline Review, H1 2015

  • January 2015
  • -
  • Global Markets Direct
  • -
  • 86 pages

Gaucher's Disease - Pipeline Review, H1 2015

Summary

Global Markets Direct’s, ‘Gaucher's Disease - Pipeline Review, H1 2015’, 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

Gaucher's Disease - Pipeline Review, H1 2015
Table of Contents
Introduction 8
Global Markets Direct Report Coverage 8
Gaucher's Disease Overview 9
Therapeutics Development 10
Pipeline Products for Gaucher's Disease - Overview 10
Pipeline Products for Gaucher's Disease - Comparative Analysis 11
Gaucher's Disease - Therapeutics under Development by Companies 12
Gaucher's Disease - Therapeutics under Investigation by Universities/Institutes 15
Gaucher's Disease - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Gaucher's Disease - Products under Development by Companies 19
Gaucher's Disease - Products under Investigation by Universities/Institutes 21
Gaucher's Disease - Companies Involved in Therapeutics Development 22
Amicus Therapeutics, Inc. 22
Bioorganic Research and Services S.A. 23
Biosidus S.A. 24
Dong-A Socio Group 25
Genzyme Corporation 26
greenovation Biotech GmbH 27
ISU ABXIS Co.,Ltd. 28
JCR Pharmaceuticals Co., Ltd. 29
Lixte Biotechnology Holdings, Inc. 30
Neuraltus Pharmaceuticals, Inc. 31
Pharming Group N.V. 32
Protalix BioTherapeutics, Inc. 33
ReGenX Biosciences, LLC 34
The International Biotechnology Center (IBC) “Generium” 35
UAB Profarma 36
Gaucher's Disease - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Combination Products 38
Assessment by Target 39
Assessment by Mechanism of Action 41
Assessment by Route of Administration 43
Assessment by Molecule Type 45
Drug Profiles 47
afegostat tartrate + ERT - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
ambroxol - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
BNT-001 - Drug Profile 50
Product Description 50
Mechanism of Action 50
RandD Progress 50
eliglustat tartrate - Drug Profile 51
Product Description 51
Mechanism of Action 51
RandD Progress 51
Gene therapy to Activate Glucocerebrosidase for Gaucher's Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
RandD Progress 53
Gluco-Cerebosidase - Drug Profile 54
Product Description 54
Mechanism of Action 54
RandD Progress 54
imiglucerase biosimilar - Drug Profile 55
Product Description 55
Mechanism of Action 55
RandD Progress 55
imiglucerase biosimilar - Drug Profile 56
Product Description 56
Mechanism of Action 56
RandD Progress 56
imiglucerase biosimilar - Drug Profile 57
Product Description 57
Mechanism of Action 57
RandD Progress 57
imiglucerase biosimilar - Drug Profile 58
Product Description 58
Mechanism of Action 58
RandD Progress 58
imiglucerase biosimilar - Drug Profile 59
Product Description 59
Mechanism of Action 59
RandD Progress 59
JR-101 - Drug Profile 60
Product Description 60
Mechanism of Action 60
RandD Progress 60
LB-201 - Drug Profile 61
Product Description 61
Mechanism of Action 61
RandD Progress 61
LB-205 - Drug Profile 62
Product Description 62
Mechanism of Action 62
RandD Progress 62
NP-003 - Drug Profile 63
Product Description 63
Mechanism of Action 63
RandD Progress 63
PRX-112 - Drug Profile 64
Product Description 64
Mechanism of Action 64
RandD Progress 64
Recombinant Enzyme for Gaucher's Disease - Drug Profile 65
Product Description 65
Mechanism of Action 65
RandD Progress 65
Recombinant Enzyme to Replace Beta-Cerebrosidase for Gaucher's Disease - Drug Profile 66
Product Description 66
Mechanism of Action 66
RandD Progress 66
Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's disease - Drug Profile 67
Product Description 67
Mechanism of Action 67
RandD Progress 67
Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 68
Product Description 68
Mechanism of Action 68
RandD Progress 68
Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease - Drug Profile 69
Product Description 69
Mechanism of Action 69
RandD Progress 69
Small Molecules to Inhibit Alpha-glucosidase for Diabetes and Gaucher's Disease - Drug Profile 70
Product Description 70
Mechanism of Action 70
RandD Progress 70
Gaucher's Disease - Recent Pipeline Updates 71
Gaucher's Disease - Dormant Projects 75
Gaucher's Disease - Discontinued Products 76
Gaucher's Disease - Product Development Milestones 77
Featured News and Press Releases 77
Nov 21, 2014: Cerdelga recommended for approval in type 1 Gaucher disease 77
Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 77
Aug 19, 2014: FDA approves new drug to treat a form of Gaucher disease 78
Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 79
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 80
Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 80
Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 81
Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 82
Dec 11, 2013: FDA Grants Priority Review for Genzyme's Cerdelga, an Investigational Oral Therapy for Gaucher Disease 83
Dec 11, 2013: FDA Grants Priority Review for Genzyme's Cerdelga, an Investigational Oral Therapy for Gaucher Disease 83
Appendix 85
Methodology 85
Coverage 85
Secondary Research 85
Primary Research 85
Expert Panel Validation 85
Contact Us 86
Disclaimer 86

List of Tables

Number of Products under Development for Gaucher's Disease, H1 2015 10
Number of Products under Development for Gaucher's Disease - Comparative Analysis, H1 2015 11
Number of Products under Development by Companies, H1 2015 13
Number of Products under Development by Companies, H1 2015 (Contd..1) 14
Number of Products under Investigation by Universities/Institutes, H1 2015 15
Comparative Analysis by Late Stage Development, H1 2015 16
Comparative Analysis by Clinical Stage Development, H1 2015 17
Comparative Analysis by Early Stage Development, H1 2015 18
Products under Development by Companies, H1 2015 19
Products under Development by Companies, H1 2015 (Contd..1) 20
Products under Investigation by Universities/Institutes, H1 2015 21
Gaucher's Disease - Pipeline by Amicus Therapeutics, Inc., H1 2015 22
Gaucher's Disease - Pipeline by Bioorganic Research and Services S.A., H1 2015 23
Gaucher's Disease - Pipeline by Biosidus S.A., H1 2015 24
Gaucher's Disease - Pipeline by Dong-A Socio Group, H1 2015 25
Gaucher's Disease - Pipeline by Genzyme Corporation, H1 2015 26
Gaucher's Disease - Pipeline by greenovation Biotech GmbH, H1 2015 27
Gaucher's Disease - Pipeline by ISU ABXIS Co.,Ltd., H1 2015 28
Gaucher's Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H1 2015 29
Gaucher's Disease - Pipeline by Lixte Biotechnology Holdings, Inc., H1 2015 30
Gaucher's Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H1 2015 31
Gaucher's Disease - Pipeline by Pharming Group N.V., H1 2015 32
Gaucher's Disease - Pipeline by Protalix BioTherapeutics, Inc., H1 2015 33
Gaucher's Disease - Pipeline by ReGenX Biosciences, LLC, H1 2015 34
Gaucher's Disease - Pipeline by The International Biotechnology Center (IBC) “Generium”, H1 2015 35
Gaucher's Disease - Pipeline by UAB Profarma, H1 2015 36
Assessment by Monotherapy Products, H1 2015 37
Assessment by Combination Products, H1 2015 38
Number of Products by Stage and Target, H1 2015 40
Number of Products by Stage and Mechanism of Action, H1 2015 42
Number of Products by Stage and Route of Administration, H1 2015 44
Number of Products by Stage and Molecule Type, H1 2015 46
Gaucher's Disease Therapeutics - Recent Pipeline Updates, H1 2015 71
Gaucher's Disease - Dormant Projects, H1 2015 75
Gaucher's Disease - Discontinued Products, H1 2015 76

List of Figures

Number of Products under Development for Gaucher's Disease, H1 2015 10
Number of Products under Development for Gaucher's Disease - Comparative Analysis, H1 2015 11
Number of Products under Development by Companies, H1 2015 12
Comparative Analysis by Late Stage Development, H1 2015 16
Comparative Analysis by Clinical Stage Development, H1 2015 17
Comparative Analysis by Early Stage Products, H1 2015 18
Assessment by Monotherapy Products, H1 2015 37
Number of Products by Top 10 Targets, H1 2015 39
Number of Products by Stage and Top 10 Targets, H1 2015 40
Number of Products by Top 10 Mechanism of Actions, H1 2015 41
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 42
Number of Products by Top 10 Routes of Administration, H1 2015 43
Number of Products by Stage and Top 10 Routes of Administration, H1 2015 44
Number of Products by Top 10 Molecule Types, H1 2015 45
Number of Products by Stage and Top 10 Molecule Types, H1 2015 46

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