Fabry Disease - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 72 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Fabry Disease - Pipeline Review, H2 2014

Summary

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

This report provides comprehensive information on the therapeutic development for Fabry 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 Fabry 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 Fabry 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 Fabry Disease and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Fabry 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 Fabry 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 Fabry Disease
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Fabry 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 5
Introduction 6
Global Markets Direct Report Coverage 6
Fabry Disease Overview 7
Therapeutics Development 8
Pipeline Products for Fabry Disease - Overview 8
Pipeline Products for Fabry Disease - Comparative Analysis 9
Fabry Disease - Therapeutics under Development by Companies 10
Fabry Disease - Pipeline Products Glance 11
Late Stage Products 11
Clinical Stage Products 12
Early Stage Products 13
Fabry Disease - Products under Development by Companies 14
Fabry Disease - Companies Involved in Therapeutics Development 15
Amicus Therapeutics, Inc. 15
Biosidus S.A. 16
Genzyme Corporation 17
greenovation Biotech GmbH 18
iBio, Inc. 19
ISU ABXIS Co.,Ltd. 20
JCR Pharmaceuticals Co., Ltd. 21
Neuraltus Pharmaceuticals, Inc. 22
Pharming Group N.V. 23
Protalix BioTherapeutics, Inc. 24
Fabry Disease - Therapeutics Assessment 25
Assessment by Monotherapy Products 25
Assessment by Combination Products 26
Assessment by Target 27
Assessment by Mechanism of Action 29
Assessment by Route of Administration 31
Assessment by Molecule Type 33
Drug Profiles 35
(migalastat hydrochloride + agalsidase alfa) - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
agalsidase alfa - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
agalsidase beta biosimilar - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
agalsidase beta biosimilar - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Alpha-Galactosidase - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Genz-682452 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
GZ-402671 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
JR-051 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
migalastat hydrochloride - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
migalastat hydrochloride + Enzyme Replacement Therapy - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
NP-003 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
PRX-102 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Recombinant Enzyme to Replace Alpha-Galactosidase A for Fabry's Disease - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Fabry Disease - Recent Pipeline Updates 51
Fabry Disease - Dormant Projects 57
Fabry Disease - Product Development Milestones 58
Featured News & Press Releases 58
Oct 19, 2014: Amicus Therapeutics Announces Additional Positive Phase 3 Data From Fabry Monotherapy Study 011 58
Sep 29, 2014: First GMP-C ompliant Batch of a Biologic Drug Substance from a Moss Expression System Produced for Clinical Use 58
Aug 20, 2014: Amicus Therapeutics Announces Positive Phase 3 Data From Fabry Monotherapy Study 012 59
Jun 30, 2014: Amicus Therapeutics Provides Updates and Final Analysis Plan for Phase 3 Fabry Monotherapy Study 012 61
Apr 29, 2014: Amicus Therapeutics Announces Positive 12- and 24-Month Data From Phase 3 Fabry Monotherapy Study 011 62
Feb 12, 2014: Amicus Therapeutics Highlights Data Featured at Lysosomal Disease Network WORLD Symposium 2014 64
Jun 17, 2013: Amicus Therapeutics Delays US Regulatory Filing For Migalastat HCl For Treatment Of Fabry Disease 66
Feb 15, 2013: Amicus Therapeutics Presents Additional Results From Phase III Fabry Monotherapy Study At LDN World Symposium 66
Feb 13, 2013: Amicus Therapeutics Presents Additional Results From Phase II Chaperone-enzyme Replacement Therapy Study For Fabry Disease At LDN World Symposium 68
Feb 07, 2013: Amicus Therapeutics To Present Data On Its Pharmacological Chaperones At Lysosomal Disease Network WORLD Symposium 69
Appendix 71
Methodology 71
Coverage 71
Secondary Research 71
Primary Research 71
Expert Panel Validation 71
Contact Us 72
Disclaimer 72

List of Tables
Number of Products under Development for Fabry Disease, H2 2014 8
Number of Products under Development for Fabry Disease - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Comparative Analysis by Late Stage Development, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Fabry Disease - Pipeline by Amicus Therapeutics, Inc., H2 2014 15
Fabry Disease - Pipeline by Biosidus S.A., H2 2014 16
Fabry Disease - Pipeline by Genzyme Corporation, H2 2014 17
Fabry Disease - Pipeline by greenovation Biotech GmbH, H2 2014 18
Fabry Disease - Pipeline by iBio, Inc., H2 2014 19
Fabry Disease - Pipeline by ISU ABXIS Co.,Ltd., H2 2014 20
Fabry Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2014 21
Fabry Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2014 22
Fabry Disease - Pipeline by Pharming Group N.V., H2 2014 23
Fabry Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2014 24
Assessment by Monotherapy Products, H2 2014 25
Assessment by Combination Products, H2 2014 26
Number of Products by Stage and Target, H2 2014 28
Number of Products by Stage and Mechanism of Action, H2 2014 30
Number of Products by Stage and Route of Administration, H2 2014 32
Number of Products by Stage and Molecule Type, H2 2014 34
Fabry Disease Therapeutics - Recent Pipeline Updates, H2 2014 51
Fabry Disease - Dormant Projects, H2 2014 57