Fabry Disease-Pipeline Review, H1 2017

Fabry Disease-Pipeline Review, H1 2017


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  • Pages: 67
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Executive Summary

Fabry Disease-Pipeline Review, H1 2017

Summary

Global Markets Direct'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

Global Markets Direct'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 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.



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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

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 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

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

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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 Therapeutic Products under Development, Key Players in Fabry Disease Therapeutics, Fabry Disease Pipeline Overview, Fabry Disease Pipeline, Fabry Disease Pipeline Assessment

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