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Fabry Disease-Pipeline Review, H2 2016

Fabry Disease-Pipeline Review, H2 2016

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Executive Summary

Fabry Disease-Pipeline Review, H2 2016


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.


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 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 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

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

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Actelion Ltd

Alexion Pharmaceuticals Inc

Amicus Therapeutics, Inc.

Genzyme Corporation

Greenovation Biotech GmbH


JCR Pharmaceuticals Co., Ltd.

Neuraltus Pharmaceuticals, Inc.

Pharming Group N.V.

Protalix BioTherapeutics, Inc.

Sangamo BioSciences, Inc.

Shire Plc

Fabry Disease Therapeutic Products under Development, Key Players in Fabry Disease Therapeutics, Fabry Disease Pipeline Overview, Fabry Disease Pipeline, Fabry Disease Pipeline Assessment

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