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Fabry Disease - Pipeline Review, H2 2016

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


    Introduction
    Global Markets Direct Report Coverage
    Fabry Disease Overview
    Therapeutics Development
    Pipeline Products for Fabry Disease - Overview
    Fabry Disease - Therapeutics under Development by Companies
    Fabry Disease - Pipeline Products Glance
    Late Stage Products
    Clinical Stage Products
    Early Stage Products
    Fabry Disease - Products under Development by Companies
    Fabry Disease - Companies Involved in Therapeutics Development
    Actelion Ltd
    Alexion Pharmaceuticals Inc
    Amicus Therapeutics, Inc.
    Genzyme Corporation
    Greenovation Biotech GmbH
    ISU ABXIS Co.,Ltd.
    JCR Pharmaceuticals Co., Ltd.
    Neuraltus Pharmaceuticals, Inc.
    Pharming Group N.V.
    Protalix BioTherapeutics, Inc.
    Sangamo BioSciences, Inc.
    Shire Plc
    Fabry Disease - Therapeutics Assessment
    Assessment by Monotherapy Products
    Assessment by Combination Products
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Drug Profiles
    agalsidase alfa - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    agalsidase alfa - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    agalsidase beta biosimilar - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    AVR-02 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Enzyme Replacement Therapy + migalastat hydrochloride Biobetter - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ibiglustat - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    lucerastat - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    migalastat hydrochloride - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    MOSS-AGAL - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    NP-003 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PRX-102 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Alpha-Galactosidase A Replacement for Fabry Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SBLSD-4 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Fabry Disease - Dormant Projects
    Fabry Disease - Discontinued Products
    Fabry Disease - Product Development Milestones
    Featured News & Press Releases
    Aug 10, 2016: New England Journal of Medicine Publishes Pivotal Phase 3 FACETS Study of Migalastat for Patients with Fabry Disease
    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
    Aug 03, 2016: Amicus Therapeutics to Submit Japanese New Drug Application for Migalastat for Fabry Disease in 1H17
    Aug 02, 2016: Almac Group’s Integrated Development to Commercialisation Services Support Amicus Therapeutics’ First Commercial Orphan Drug Product
    Jun 06, 2016: Protalix BioTherapeutics Initiates PRX-102 Global Phase III Clinical Trial of Fabry Disease to Support United States and European Filings
    May 31, 2016: Amicus Therapeutics Announces European Commission Approval for Galafold (Migalastat) in Patients with Fabry Disease in European Union
    Apr 01, 2016: Amicus Therapeutics Receives Positive CHMP Opinion for Approval of Migalastat in Patients with Fabry Disease in European Union
    Apr 01, 2016: First oral treatment for Fabry disease recommended for approval in the EU
    Mar 22, 2016: Amicus Therapeutics Announces Expanded Access Program for Migalastat
    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
    Mar 01, 2016: Amicus Therapeutics Highlights New Phase 3 Fabry Data at WORLDSymposium 2016
    Feb 22, 2016: Protalix BioTherapeutics to Participate in the SunTrust Robinson Humphrey Orphan Drug Day and the 12th Annual WORLDSymposium 2016
    Feb 10, 2016: Amicus Therapeutics Announces Presentations on Migalastat at 12th Annual WORLDSymposium 2016
    Jan 11, 2016: Amicus Therapeutics Provides Update On Migalastat for Fabry Disease
    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
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer
    List of Tables
    Number of Products under Development for Fabry Disease, H2 2016
    Number of Products under Development by Companies, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Development, H2 2016
    Products under Development by Companies, H2 2016
    Fabry Disease - Pipeline by Actelion Ltd, H2 2016
    Fabry Disease - Pipeline by Alexion Pharmaceuticals Inc, H2 2016
    Fabry Disease - Pipeline by Amicus Therapeutics, Inc., H2 2016
    Fabry Disease - Pipeline by Genzyme Corporation, H2 2016
    Fabry Disease - Pipeline by greenovation Biotech GmbH, H2 2016
    Fabry Disease - Pipeline by ISU ABXIS Co.,Ltd., H2 2016
    Fabry Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2016
    Fabry Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2016
    Fabry Disease - Pipeline by Pharming Group N.V., H2 2016
    Fabry Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2016
    Fabry Disease - Pipeline by Sangamo BioSciences, Inc., H2 2016
    Fabry Disease - Pipeline by Shire Plc, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Assessment by Combination Products, H2 2016
    Number of Products by Stage and Target, H2 2016
    Number of Products by Stage and Mechanism of Action, H2 2016
    Number of Products by Stage and Route of Administration, H2 2016
    Number of Products by Stage and Molecule Type, H2 2016
    Fabry Disease - Dormant Projects, H2 2016
    Fabry Disease - Discontinued Products, H2 2016
    List of Figures
    Number of Products under Development for Fabry Disease, H2 2016
    Number of Products under Development by Companies, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Targets, H2 2016
    Number of Products by Stage and Top 10 Targets, H2 2016
    Number of Products by Mechanism of Actions, H2 2016
    Number of Products by Stage and Mechanism of Actions, H2 2016
    Number of Products by Routes of Administration, H2 2016
    Number of Products by Stage and Routes of Administration, H2 2016
    Number of Products by Molecule Types, H2 2016
    Number of Products by Stage and Molecule Types, H2 2016

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