Fabry Disease - Pipeline Review, H1 2016

Published: Mar, 2016 | Pages: 76 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Fabry Disease - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Fabry Disease - Pipeline Review, H1 2016', provides an overview of the Fabry Disease pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Fabry Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Fabry Disease and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Fabry Disease
- The report reviews pipeline therapeutics for Fabry Disease by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Fabry Disease therapeutics and enlists all their major and minor projects
- The report assesses Fabry Disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Fabry Disease

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Fabry Disease
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- 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 6
Introduction 7
Global Markets Direct Report Coverage 7
Fabry Disease Overview 8
Therapeutics Development 9
Pipeline Products for Fabry Disease - Overview 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
Actelion Ltd 15
Alexion Pharmaceuticals, Inc. 16
Amicus Therapeutics, Inc. 17
Genzyme Corporation 18
greenovation Biotech GmbH 19
iBio, Inc. 20
ISU ABXIS Co.,Ltd. 21
JCR Pharmaceuticals Co., Ltd. 22
Neuraltus Pharmaceuticals, Inc. 23
Pharming Group N.V. 24
Protalix BioTherapeutics, Inc. 25
Sangamo BioSciences, Inc. 26
Shire Plc 27
Fabry Disease - Therapeutics Assessment 28
Assessment by Monotherapy Products 28
Assessment by Combination Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
agalsidase alfa - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
agalsidase alfa - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
agalsidase alfa - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
agalsidase beta biosimilar - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
AVR-02 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Enzyme Replacement Therapy + migalastat hydrochloride Biobetter - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
ibiglustat - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
lucerastat - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
migalastat hydrochloride - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
MOSS-AGAL - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
NP-003 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
PRX-102 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Recombinant Alpha-Galactosidase A Replacement for Fabry's Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
SBLSD-4 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Fabry Disease - Recent Pipeline Updates 56
Fabry Disease - Dormant Projects 65
Fabry Disease - Discontinued Products 66
Fabry Disease - Product Development Milestones 67
Featured News & Press Releases 67
Mar 01, 2016: Amicus Therapeutics Highlights New Phase 3 Fabry Data at WORLD Symposium 2016 67
Feb 10, 2016: Amicus Therapeutics Announces Presentations on Migalastat at 12th Annual WORLD Symposium 2016 68
Jan 11, 2016: Amicus Therapeutics Provides Update On Migalastat for Fabry Disease 68
Oct 02, 2015: Amicus Therapeutics Provides U.S. Regulatory Update for Migalastat Monotherapy 69
Sep 15, 2015: Amicus Therapeutics Plans to Submit New Drug Application for Migalastat for Fabry Disease Following Positive Pre-NDA Meeting With FDA 70
Jun 25, 2015: Amicus Therapeutics Announces Galafold Marketing Authorization Application Validated by European Medicines Agency 70
Jun 03, 2015: Amicus Therapeutics Submits Marketing Authorization Application for Full Approval of Fabry Monotherapy Galafold (Migalastat) in European Union 71
May 26, 2015: Amicus Therapeutics Fabry Monotherapy Granted Accelerated Assessment by European Regulators 72
May 26, 2015: Amicus Therapeutics' Fabry Monotherapy Granted Accelerated Assessment by European Regulators 73
Apr 28, 2015: Sanofi: FDA Grants Fast Track Designation To Genzyme's Investigational Substrate Reduction Therapy For The Treatment Of Fabry Disease 73
Appendix 75
Methodology 75
Coverage 75
Secondary Research 75
Primary Research 75
Expert Panel Validation 75
Contact Us 75
Disclaimer 76

List of Tables

Number of Products under Development for Fabry Disease, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Comparative Analysis by Late Stage Development, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Fabry Disease - Pipeline by Actelion Ltd, H1 2016 15
Fabry Disease - Pipeline by Alexion Pharmaceuticals, Inc., H1 2016 16
Fabry Disease - Pipeline by Amicus Therapeutics, Inc., H1 2016 17
Fabry Disease - Pipeline by Genzyme Corporation, H1 2016 18
Fabry Disease - Pipeline by greenovation Biotech GmbH, H1 2016 19
Fabry Disease - Pipeline by iBio, Inc., H1 2016 20
Fabry Disease - Pipeline by ISU ABXIS Co.,Ltd., H1 2016 21
Fabry Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H1 2016 22
Fabry Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H1 2016 23
Fabry Disease - Pipeline by Pharming Group N.V., H1 2016 24
Fabry Disease - Pipeline by Protalix BioTherapeutics, Inc., H1 2016 25
Fabry Disease - Pipeline by Sangamo BioSciences, Inc., H1 2016 26
Fabry Disease - Pipeline by Shire Plc, H1 2016 27
Assessment by Monotherapy Products, H1 2016 28
Assessment by Combination Products, H1 2016 29
Number of Products by Stage and Target, H1 2016 31
Number of Products by Stage and Mechanism of Action, H1 2016 33
Number of Products by Stage and Route of Administration, H1 2016 35
Number of Products by Stage and Molecule Type, H1 2016 37
Fabry Disease Therapeutics - Recent Pipeline Updates, H1 2016 56
Fabry Disease - Dormant Projects, H1 2016 65
Fabry Disease - Discontinued Products, H1 2016 66