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Fabry Disease - Pipeline Review, H2 2013

Published By :

Global Markets Direct

Published Date : Nov 2013

Category :

Metabolic Disorders

No. of Pages : 68 Pages


Global Markets Directs, \'Fabry Disease Pipeline Review, H2 2013\', provides an overview of the indications therapeutic pipeline. This report provides information on the therapeutic development for Fabry Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Fabry Disease. Fabry Disease Pipeline Review, Half Year is built using data and information sourced from Global Markets Directs 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 Directs team.

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

Scope

  • A snapshot of the global therapeutic scenario for Fabry Disease.
  • 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.
  • Coverage of products based on various stages of development ranging from discovery till registration stages.
  • A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
  • Coverage of the Fabry Disease pipeline on the basis of route of administration and molecule type.
  • Key discontinued pipeline projects.
  • Latest news and deals relating to the products.

Reasons to buy

  • Identify and understand important and diverse types of therapeutics under development for Fabry Disease.
  • Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
  • Plan mergers and acquisitions effectively by identifying 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
An Overview of Pipeline Products for Fabry Disease 8
Fabry Disease Therapeutics under Development by Companies 10
Late Stage Products 12
Comparative Analysis 12
Mid Clinical Stage Products 13
Comparative Analysis 13
Early Clinical Stage Products 14
Comparative Analysis 14
Pre-Clinical Stage Products 15
Comparative Analysis 15
Fabry Disease Therapeutics - Products under Development by Companies 16
Companies Involved in Fabry Disease Therapeutics Development 17
Genzyme Corporation 17
Sanofi-Aventis 18
GlaxoSmithKline plc 19
Amicus Therapeutics, Inc. 20
Protalix BioTherapeutics, Inc. 21
Green Cross Corporation 22
JCR Pharmaceuticals Co., Ltd. 23
greenovation Biotech GmbH 24
iBio, Inc. 25
Neuraltus Pharmaceuticals, Inc. 26
ISU ABXIS Co.,Ltd. 27
Bio Sidus S.A. 28
Fabry Disease - Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Combination Products 30
Assessment by Route of Administration 31
Assessment by Molecule Type 33
Drug Profiles 35
migalastat hydrochloride - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
PRX-102 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
JR-051 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
NP-003 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
migalastat hydrochloride + Enzyme Replacement Therapy - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Fabrazyme Biosimilar - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Alpha-galactosidase A - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
GC-1119 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Genz-682452 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
GZ-402671 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
(migalastat hydrochloride + agalsidase alfa) - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Alpha-Galactosidase - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Fabry Disease Therapeutics - Drug Profile Updates 49
Fabry Disease Therapeutics - Dormant Products 55
Fabry Disease - Product Development Milestones 56
Featured News & Press Releases 56
Oct 15, 2013: Genzyme to Invest $80 Million in Framingham, Mass. Site to Expand Manufacturing Capacity for Fabrazyme 56
Jun 17, 2013: Amicus Therapeutics Delays US Regulatory Filing For Migalastat HCl For Treatment Of Fabry Disease 56
Feb 15, 2013: Amicus Therapeutics Presents Additional Results From Phase III Fabry Monotherapy Study At LDN World Symposium 56
Feb 13, 2013: Amicus Therapeutics Presents Additional Results From Phase II Chaperone-enzyme Replacement Therapy Study For Fabry Disease At LDN World Symposium 58
Feb 07, 2013: Amicus Therapeutics To Present Data On Its Pharmacological Chaperones At Lysosomal Disease Network WORLD Symposium 60
Dec 19, 2012: Amicus Therapeutics And GSK Announce Top Line Six-Month Primary Treatment Period Results From First Phase III Fabry Monotherapy Study With Migalastat HCl 60
Nov 08, 2012: Amicus Therapeutics Announces Additional Preliminary Results From Ongoing Phase II Chaperone-Enzyme Replacement Therapy Study For Fabry Disease 62
Nov 05, 2012: Amicus Therapeutics Announces Updated Results From Phase II Extension Study Of Migalastat HCl For Fabry Disease 63
Oct 22, 2012: Amicus Therapeutics Achieves Target Enrollment In Second Phase III Fabry Monotherapy Study 65
Oct 16, 2012: Amicus Therapeutics Announces Data Presentations On Migalastat At Upcoming Scientific Meetings 66

Appendix 67
Methodology 67
Coverage 67
Secondary Research 67
Primary Research 67
Expert Panel Validation 67
Contact Us 68
Disclaimer 68

List of Table


Number of Products Under Development for Fabry Disease, H2 2013 8
Products under Development for Fabry Disease - Comparative Analysis, H2 2013 9
Number of Products under Development by Companies, H2 2013 11
Comparative Analysis by Late Stage Development, H2 2013 12
Comparative Analysis by Mid Clinical Stage Development, H2 2013 13
Comparative Analysis by Early Clinical Stage Development, H2 2013 14
Comparative Analysis by Pre-Clinical Stage Development, H2 2013 15
Products under Development by Companies, H2 2013 16
Genzyme Corporation, H2 2013 17
Sanofi-Aventis, H2 2013 18
GlaxoSmithKline plc, H2 2013 19
Amicus Therapeutics, Inc., H2 2013 20
Protalix BioTherapeutics, Inc., H2 2013 21
Green Cross Corporation, H2 2013 22
JCR Pharmaceuticals Co., Ltd., H2 2013 23
greenovation Biotech GmbH, H2 2013 24
iBio, Inc., H2 2013 25
Neuraltus Pharmaceuticals, Inc., H2 2013 26
ISU ABXIS Co.,Ltd., H2 2013 27
Bio Sidus S.A., H2 2013 28
Assessment by Monotherapy Products, H2 2013 29
Assessment by Combination Products, H2 2013 30
Assessment by Stage and Route of Administration, H2 2013 32
Assessment by Stage and Molecule Type, H2 2013 34
Fabry Disease Therapeutics - Drug Profile Updates 49
Fabry Disease Therapeutics - Dormant Products 55

List of Chart

List of Figures
Number of Products under Development for Fabry Disease, H2 2013 8
Products under Development for Fabry Disease - Comparative Analysis, H2 2013 9
Products under Development by Companies, H2 2013 10
Late Stage Products, H2 2013 12
Mid Clinical Stage Products, H2 2013 13
Early Clinical Stage Products, H2 2013 14
Pre-Clinical Stage Products, H2 2013 15
Assessment by Monotherapy Products, H2 2013 29
Assessment by Combination Products, H2 2013 30
Assessment by Route of Administration, H2 2013 31
Assessment by Stage and Route of Administration, H2 2013 32
Assessment by Molecule Type, H2 2013 33
Assessment by Stage and Molecule Type, H2 2013 34

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