Fabry Disease - Pipeline Review, H2 2016

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Fabry Disease - Pipeline Review, H2 2016 Summary Global Markets Directs, 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 Directs 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 Directs 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 its 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 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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