Fabry Disease - Pipeline Review, H2 2016

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Date: 31-Aug-2016
No. of pages: 81
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Fabry Disease - Pipeline Review, H2 2016

Summary

Global Markets Direct's, ‘Fabry Disease - Pipeline Review, H2 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

Fabry Disease - Pipeline Review, H2 2016

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
Fabry Disease - Therapeutics under Development by Companies 9
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
ISU ABXIS Co.,Ltd. 20
JCR Pharmaceuticals Co., Ltd. 21
Neuraltus Pharmaceuticals, Inc. 22
Pharming Group N.V. 23
Protalix BioTherapeutics, Inc. 24
Sangamo BioSciences, Inc. 25
Shire Plc 26
Fabry Disease - Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Combination Products 28
Assessment by Target 29
Assessment by Mechanism of Action 31
Assessment by Route of Administration 33
Assessment by Molecule Type 35
Drug Profiles 37
agalsidase alfa - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
agalsidase alfa - 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
AVR-02 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Enzyme Replacement Therapy + migalastat hydrochloride Biobetter - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
ibiglustat - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
lucerastat - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
migalastat hydrochloride - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
MOSS-AGAL - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
NP-003 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
PRX-102 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Recombinant Alpha-Galactosidase A Replacement for Fabry Disease - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
SBLSD-4 - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
Fabry Disease - Dormant Projects 64
Fabry Disease - Discontinued Products 65
Fabry Disease - Product Development Milestones 66
Featured News & Press Releases 66
Aug 10, 2016: New England Journal of Medicine Publishes Pivotal Phase 3 FACETS Study of Migalastat for Patients with Fabry Disease 66
Aug 10, 2016: Protalix BioTherapeutics Announces Additional Positive Data from its Phase I/II Clinical Trial for PRX-102 for the Treatment of Fabry Disease 67
Aug 03, 2016: Amicus Therapeutics to Submit Japanese New Drug Application for Migalastat for Fabry Disease in 1H17 68
Aug 02, 2016: Almac Group's Integrated Development to Commercialisation Services Support Amicus Therapeutics' First Commercial Orphan Drug Product 69
Jun 06, 2016: Protalix BioTherapeutics Initiates PRX-102 Global Phase III Clinical Trial of Fabry Disease to Support United States and European Filings 69
May 31, 2016: Amicus Therapeutics Announces European Commission Approval for Galafold (Migalastat) in Patients with Fabry Disease in European Union 70
Apr 01, 2016: Amicus Therapeutics Receives Positive CHMP Opinion for Approval of Migalastat in Patients with Fabry Disease in European Union 72
Apr 01, 2016: First oral treatment for Fabry disease recommended for approval in the EU 73
Mar 22, 2016: Amicus Therapeutics Announces Expanded Access Program for Migalastat 74
Mar 03, 2016: Protalix BioTherapeutics Presents Positive Six and Twelve Month Interim Clinical Data on PRX-102 for the Treatment of Fabry Disease at the 12th Annual WORLDSymposium 2016 74
Mar 01, 2016: Amicus Therapeutics Highlights New Phase 3 Fabry Data at WORLDSymposium 2016 75
Feb 22, 2016: Protalix BioTherapeutics to Participate in the SunTrust Robinson Humphrey Orphan Drug Day and the 12th Annual WORLDSymposium 2016 76
Feb 10, 2016: Amicus Therapeutics Announces Presentations on Migalastat at 12th Annual WORLDSymposium 2016 76
Jan 11, 2016: Amicus Therapeutics Provides Update On Migalastat for Fabry Disease 77
Nov 16, 2015: Protalix BioTherapeutics to Conduct Phase III Clinical Trial for PRX-102 for the Treatment of Fabry Disease Following a Successful End-of-Phase II Meeting With FDA 78
Appendix 80
Methodology 80
Coverage 80
Secondary Research 80
Primary Research 80
Expert Panel Validation 80
Contact Us 80
Disclaimer 81

List of Tables
Number of Products under Development for Fabry Disease, H2 2016 8
Number of Products under Development by Companies, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 11
Comparative Analysis by Clinical Stage Development, H2 2016 12
Comparative Analysis by Early Stage Development, H2 2016 13
Products under Development by Companies, H2 2016 14
Fabry Disease - Pipeline by Actelion Ltd, H2 2016 15
Fabry Disease - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 16
Fabry Disease - Pipeline by Amicus Therapeutics, Inc., H2 2016 17
Fabry Disease - Pipeline by Genzyme Corporation, H2 2016 18
Fabry Disease - Pipeline by greenovation Biotech GmbH, H2 2016 19
Fabry Disease - Pipeline by ISU ABXIS Co.,Ltd., H2 2016 20
Fabry Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2016 21
Fabry Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2016 22
Fabry Disease - Pipeline by Pharming Group N.V., H2 2016 23
Fabry Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2016 24
Fabry Disease - Pipeline by Sangamo BioSciences, Inc., H2 2016 25
Fabry Disease - Pipeline by Shire Plc, H2 2016 26
Assessment by Monotherapy Products, H2 2016 27
Assessment by Combination Products, H2 2016 28
Number of Products by Stage and Target, H2 2016 30
Number of Products by Stage and Mechanism of Action, H2 2016 32
Number of Products by Stage and Route of Administration, H2 2016 34
Number of Products by Stage and Molecule Type, H2 2016 36
Fabry Disease - Dormant Projects, H2 2016 64
Fabry Disease - Discontinued Products, H2 2016 65

List of Figures
Number of Products under Development for Fabry Disease, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Comparative Analysis by Clinical Stage Development, H2 2016 12
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Targets, H2 2016 29
Number of Products by Stage and Top 10 Targets, H2 2016 29
Number of Products by Mechanism of Actions, H2 2016 31
Number of Products by Stage and Mechanism of Actions, H2 2016 31
Number of Products by Routes of Administration, H2 2016 33
Number of Products by Stage and Routes of Administration, H2 2016 33
Number of Products by Molecule Types, H2 2016 35
Number of Products by Stage and Molecule Types, H2 2016 35
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