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Published: Aug, 2017 | Pages:
81 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Fabry Disease - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 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 Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H2 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 Pre-Registration, Phase III, Phase II, Phase I and Preclinical stages are 1, 3, 3, 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 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. 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 Global Markets Direct 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 Amicus Therapeutics Inc Genzyme Corp greenovation Biotech GmbH Idorsia Ltd JCR Pharmaceuticals Co Ltd Neuraltus Pharmaceuticals Inc Pharming Group NV Protalix BioTherapeutics Inc Resverlogix Corp 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 apabetalone - Drug Profile Product Description Mechanism Of Action R&D Progress AVRRD-01 - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate Alpha Galactosidase A for Fabry Disease - 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 Jul 11, 2017: U.S. FDA Confirms Amicus Therapeutics May Submit New Drug Application for Migalastat for Fabry Disease Jun 28, 2017: Amicus Therapeutics Submits Japanese New Drug Application for Migalastat for Fabry Disease Jun 01, 2017: Protalix BioTherapeutics Announces FDA Approval to Operate its Current Facility as a Multi-Product Facility May 30, 2017: Resverlogix Receives Approval From Health Canada To Proceed With Fabry Disease Clinical Trial With Lead Compound Apabetalone May 09, 2017: Protalix Announces FDA Investigational New Drug Clearance to Commence Once-Monthly Dosing Study of pegunigalsidase alfa (PRX-102) for the Treatment of Fabry Disease May 02, 2017: Amicus Therapeutics Launches Galafold for Treatment of Fabry Disease in France Apr 18, 2017: Protalix BioTherapeutics Announces New Preclinical Results Demonstrating a Positive Effect of pegunigalsidase alfa (PRX-102) on Small-fiber Neuropathy in Fabry Disease Models Compared to Commercially Available Enzyme Replacement Therapies 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 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 2017 Number of Products under Development by Companies, H2 2017 Products under Development by Companies, H2 2017 Number of Products by Stage and Target, H2 2017 Number of Products by Stage and Mechanism of Action, H2 2017 Number of Products by Stage and Route of Administration, H2 2017 Number of Products by Stage and Molecule Type, H2 2017 Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2017 Fabry Disease - Pipeline by Genzyme Corp, H2 2017 Fabry Disease - Pipeline by greenovation Biotech GmbH, H2 2017 Fabry Disease - Pipeline by Idorsia Ltd, H2 2017 Fabry Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017 Fabry Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H2 2017 Fabry Disease - Pipeline by Pharming Group NV, H2 2017 Fabry Disease - Pipeline by Protalix BioTherapeutics Inc, H2 2017 Fabry Disease - Pipeline by Resverlogix Corp, H2 2017 Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H2 2017 Fabry Disease - Pipeline by Shire Plc, H2 2017 Fabry Disease - Dormant Projects, H2 2017 Fabry Disease - Discontinued Products, H2 2017
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