Fabry Disease - Pipeline Review, H1 2015

Published: Feb, 2015 | Pages: 72 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Fabry Disease - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Fabry Disease - Pipeline Review, H1 2015', 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

Reason 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
Synageva BioPharma Corp. 25
Fabry Disease - Therapeutics Assessment 26
Assessment by Monotherapy Products 26
Assessment by Combination Products 27
Assessment by Target 28
Assessment by Mechanism of Action 30
Assessment by Route of Administration 32
Assessment by Molecule Type 34
Drug Profiles 36
(migalastat hydrochloride + agalsidase alfa) - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
agalsidase alfa - 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
agalsidase beta biosimilar - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Alpha-Galactosidase - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Genz-682452 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
GZ-402671 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
JR-051 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
migalastat hydrochloride - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Next Generation biologics for Pompe, Fabry and Hunter - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
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
Jan 26, 2015: Amicus Therapeutics Announces Presentations and Posters at Lysosomal Disease Network WORLD Symposium 58
Jan 08, 2015: Amicus Therapeutics Announces Positive Phase 3 Data on Cardiac Endpoints From Fabry Monotherapy Study 011 and Long Term Extension Study 59
Dec 22, 2014: Amicus Therapeutics to Present Program Updates at 33rd Annual J.P. Morgan Healthcare Conference 60
Nov 15, 2014: Amicus Therapeutics Announces Positive Phase 3 Data on Cardiac and Composite Endpoints from Fabry Monotherapy Study 012 at American Society of Nephrology 61
Oct 19, 2014: Amicus Therapeutics Announces Additional Positive Phase 3 Data From Fabry Monotherapy Study 011 62
Sep 29, 2014: First GMP-C ompliant Batch of a Biologic Drug Substance from a Moss Expression System Produced for Clinical Use 63
Aug 20, 2014: Amicus Therapeutics Announces Positive Phase 3 Data From Fabry Monotherapy Study 012 63
Jun 30, 2014: Amicus Therapeutics Provides Updates and Final Analysis Plan for Phase 3 Fabry Monotherapy Study 012 65
Apr 29, 2014: Amicus Therapeutics Announces Positive 12- and 24-Month Data From Phase 3 Fabry Monotherapy Study 011 66
Feb 12, 2014: Amicus Therapeutics Highlights Data Featured at Lysosomal Disease Network WORLD Symposium 2014 69
Appendix 71
Methodology 71
Coverage 71
Secondary Research 71
Primary Research 71
Expert Panel Validation 71
Contact Us 71
Disclaimer 72

List of Tables
Number of Products under Development for Fabry Disease, H1 2015 8
Number of Products under Development for Fabry Disease - Comparative Analysis, H1 2015 9
Number of Products under Development by Companies, H1 2015 10
Comparative Analysis by Late Stage Development, H1 2015 11
Comparative Analysis by Clinical Stage Development, H1 2015 12
Comparative Analysis by Early Stage Development, H1 2015 13
Products under Development by Companies, H1 2015 14
Fabry Disease - Pipeline by Amicus Therapeutics, Inc., H1 2015 15
Fabry Disease - Pipeline by Biosidus S.A., H1 2015 16
Fabry Disease - Pipeline by Genzyme Corporation, H1 2015 17
Fabry Disease - Pipeline by greenovation Biotech GmbH, H1 2015 18
Fabry Disease - Pipeline by iBio, Inc., H1 2015 19
Fabry Disease - Pipeline by ISU ABXIS Co.,Ltd., H1 2015 20
Fabry Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H1 2015 21
Fabry Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H1 2015 22
Fabry Disease - Pipeline by Pharming Group N.V., H1 2015 23
Fabry Disease - Pipeline by Protalix BioTherapeutics, Inc., H1 2015 24
Fabry Disease - Pipeline by Synageva BioPharma Corp., H1 2015 25
Assessment by Monotherapy Products, H1 2015 26
Assessment by Combination Products, H1 2015 27
Number of Products by Stage and Target, H1 2015 29
Number of Products by Stage and Route of Administration, H1 2015 33
Number of Products by Stage and Molecule Type, H1 2015 35
Fabry Disease Therapeutics - Recent Pipeline Updates, H1 2015 51
Fabry Disease - Dormant Projects, H1 2015 57