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
List of Figures
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 Products, H1 2016 13 Assessment by Monotherapy Products, H1 2016 28 Number of Products by Targets, H1 2016 30 Number of Products by Stage and Targets, H1 2016 30 Number of Products by Mechanism of Actions, H1 2016 32 Number of Products by Stage and Mechanism of Actions, H1 2016 32 Number of Products by Routes of Administration, H1 2016 34 Number of Products by Stage and Routes of Administration, H1 2016 34 Number of Products by Molecule Types, H1 2016 36 Number of Products by Stage and Molecule Types, H1 2016 36
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