Fabry Disease – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Fabry Disease – Pipeline Review, H1 2017’, 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

Companies mentioned

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

Table of Contents

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 Tables

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

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

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