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    Fabry Disease - Pipeline Review, H1 2017

    Fabry Disease - Pipeline Review, H1 2017

    • Report Code ID: RW0001709485
    • Category Pharmaceuticals
    • No. of Pages 67
    • Publication Month Mar-17
    • Publisher Name Global Markets Direct
    Fabry Disease - Pipeline Review, H1 2017

    Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H1 2017, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.

    Fabry disease is an inherited disorder. Fabry disease results from abnormal deposits of a particular fatty substance (called globotriaosylceramide) in blood vessel walls throughout the body. Symptoms include pain, diarrhea, nausea, kidney problems, tinnitus, irregular heartbeat, and leaky heart valves. Treatment includes enzyme replacement therapy (ERT) .

    Report Highlights

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Fabry Disease (Genetic Disorders) , complete with analysis by stage of development, drug target, mechanism of action (MoA) , route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

    The Fabry Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fabry Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I and Preclinical stages are 4, 2, 2 and 4 respectively.

    Fabry Disease (Genetic Disorders) pipeline guide 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. The guide is built using data and information sourced from Publisher'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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

    Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

    Scope

    - The pipeline guide provides a snapshot of the global therapeutic landscape of Fabry Disease (Genetic Disorders) .
    - The pipeline guide reviews pipeline therapeutics for Fabry Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
    - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
    - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
    - The pipeline guide reviews key companies involved in Fabry Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
    - The pipeline guide evaluates Fabry Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA) , drug target, route of administration (RoA) and molecule type.
    - The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
    - The pipeline guide reviews latest news related to pipeline therapeutics for Fabry Disease (Genetic Disorders)

    Reasons to buy

    - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
    - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
    - Find and recognize significant and varied types of therapeutics under development for Fabry Disease (Genetic Disorders) .
    - Classify potential new clients or partners in the target demographic.
    - Develop tactical initiatives by understanding the focus areas of leading companies.
    - Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
    - Formulate corrective measures for pipeline projects by understanding Fabry Disease (Genetic Disorders) 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.
    - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
    Table of Contents
    List of Tables
    List of Figures
    Introduction
    Publisher Report Coverage
    Fabry Disease - Overview
    Fabry Disease - Therapeutics Development
    Pipeline Overview
    Pipeline by Companies
    Products under Development by Companies
    Fabry Disease - Therapeutics Assessment
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Fabry Disease - Companies Involved in Therapeutics Development
    Actelion Ltd
    Amicus Therapeutics Inc
    Genzyme Corp
    greenovation Biotech GmbH
    JCR Pharmaceuticals Co Ltd
    Neuraltus Pharmaceuticals Inc
    Pharming Group NV
    Protalix BioTherapeutics Inc
    Sangamo Therapeutics Inc
    Shire Plc
    Fabry Disease - 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
    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
    pegunigalsidase alfa - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    PGN-005 - 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
    Mar 08, 2017: Amicus Therapeutics Launches Galafold (Migalastat) for Treatment of Fabry Disease in Italy
    Feb 27, 2017: Amicus Therapeutics Launches Galafold (Migalastat) for Treatment of Fabry Disease in the United Kingdom
    Feb 16, 2017: Gene therapy used to treat Fabry disease - a world first
    Feb 14, 2017: Amicus Therapeutics Highlights New Fabry Program Data at WORLDSymposium 2017
    Jan 04, 2017: National Institute for Health and Care Excellence Issues Final Positive Recommendation for Galafold (migalastat) for Fabry Disease in England
    Nov 28, 2016: Amicus Therapeutics Announces U.S. Regulatory Pathway for Migalastat for Fabry Disease
    Nov 11, 2016: Journal of Medical Genetics Publishes Pivotal Phase 3 ATTRACT Study of Migalastat for Patients with Fabry Disease
    Oct 25, 2016: Protalix BioTherapeutics Doses First Patient in Global Phase III Clinical Trial of PRX-102 for the Treatment of Fabry Disease
    Sep 07, 2016: Protalix BioTherapeutics Announces Presentation of Results from the Phase I/II Clinical Trial of PRX-102 for the Treatment of Fabry Disease at the Society for the Study of Inborn Errors of Metabolism
    Aug 31, 2016: Amicus Therapeutics to Highlight Fabry Disease Program at Society for the Study of Inborn Errors of Metabolism Annual Symposium
    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
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development for Fabry Disease, H1 2017
    Number of Products under Development by Companies, H1 2017
    Products under Development by Companies, H1 2017
    Number of Products by Stage and Target, H1 2017
    Number of Products by Stage and Mechanism of Action, H1 2017
    Number of Products by Stage and Route of Administration, H1 2017
    Number of Products by Stage and Molecule Type, H1 2017
    Fabry Disease - Pipeline by Actelion Ltd, H1 2017
    Fabry Disease - Pipeline by Amicus Therapeutics Inc, H1 2017
    Fabry Disease - Pipeline by Genzyme Corp, H1 2017
    Fabry Disease - Pipeline by greenovation Biotech GmbH, H1 2017
    Fabry Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017
    Fabry Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H1 2017
    Fabry Disease - Pipeline by Pharming Group NV, H1 2017
    Fabry Disease - Pipeline by Protalix BioTherapeutics Inc, H1 2017
    Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H1 2017
    Fabry Disease - Pipeline by Shire Plc, H1 2017
    Fabry Disease - Dormant Projects, H1 2017
    Fabry Disease - Discontinued Products, H1 2017

    List of Figures

    Number of Products under Development for Fabry Disease, H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products by Targets, H1 2017
    Number of Products by Stage and Targets, H1 2017
    Number of Products by Mechanism of Actions, H1 2017
    Number of Products by Stage and Mechanism of Actions, H1 2017
    Number of Products by Routes of Administration, H1 2017
    Number of Products by Stage and Routes of Administration, H1 2017
    Number of Products by Molecule Types, H1 2017
    Number of Products by Stage and Molecule Types, H1 2017
    Actelion Ltd
    Amicus Therapeutics Inc
    Genzyme Corp
    greenovation Biotech GmbH
    JCR Pharmaceuticals Co Ltd
    Neuraltus Pharmaceuticals Inc
    Pharming Group NV
    Protalix BioTherapeutics Inc
    Sangamo Therapeutics Inc
    Shire Plc

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