Fabry Disease - Pipeline Review, H1 2018

  • ID: 4473122
  • Report
  • 84 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Genzyme Corp
  • greenovation Biotech GmbH
  • iBio Inc
  • Idorsia Ltd
  • Pharming Group NV
  • MORE
Fabry Disease - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H1 2018, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.

Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused due to mutations in GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A (this enzyme is responsible for breaking down of globotriaosylceramide). Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.

Report Highlights:

Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H1 2018, 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 2, 1, 4, 1 and 5 respectively. Similarly, the Universities portfolio in Unknown stages comprises 1 molecules, 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 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 Purchase:
  • 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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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Genzyme Corp
  • greenovation Biotech GmbH
  • iBio Inc
  • Idorsia Ltd
  • Pharming Group NV
  • MORE
Introduction

Report Coverage

Fabry Disease - Overview

Fabry Disease - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

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

iBio Inc

Idorsia Ltd

JCR Pharmaceuticals Co Ltd

Pharming Group NV

Protalix BioTherapeutics Inc

Resverlogix Corp

Sangamo Therapeutics Inc

Fabry Disease - Drug Profiles

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

apabetalone - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ATB-101 + migalastat hydrochloride - 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

Gene Therapy to Activate GLA 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

pegunigalsidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PGN-005 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ST-920 - 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

Feb 12, 2018: U.S. FDA Files New Drug Application Under Priority Review for Migalastat for Treatment of Fabry Disease

Jan 31, 2018: Protalix BioTherapeutics' pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

Jan 29, 2018: JCR to Present Data On JR-051 at the 14th Annual WORLD Symposium 2018

Jan 29, 2018: Canadian Drug Expert Committee Grants Positive Recommendation for Reimbursement of Galafold (migalastat) for Fabry Disease in Canada

Jan 29, 2018: Protalix BioTherapeutics to Participate in the 14th Annual WORLDSymposium 2018

Jan 17, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Treatment of Fabry Disease in Spain

Dec 27, 2017: Protalix BioTherapeutics’ PRX-102 Granted Orphan Drug Designation by the European Commission

Dec 19, 2017: JCR Signs MoU with the Grand Duchy of Luxembourg on the Leasing of Industrial Site

Dec 14, 2017: Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

Nov 27, 2017: Protalix BioTherapeutics Presents Positive Results from the Phase I/II Open-Label Extension Trial for PRX-102 at the New Horizons for Fabry Disease Conference

Nov 20, 2017: Protalix BioTherapeutics Announces Presentation to be made at the New Horizons in Fabry Disease Conference

Nov 13, 2017: Protalix BioTherapeutics Receives Positive Opinion for Orphan Designation for PRX-102 for the Treatment of Fabry Disease in the European Union

Oct 17, 2017: Sangamo Therapeutics Announces Presentations At 2017 Annual Congress Of The European Society Of Gene And Cell Therapy

Sep 19, 2017: U.S. FDA Grants Fast Track Designation for Amicus Therapeutics Migalastat for Treatment of Fabry Disease

Sep 14, 2017: Amicus Therapeutics Announces Approval for Galafold (Migalastat) for Treatment of Fabry Disease in Canada

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 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Fabry Disease - Pipeline by Amicus Therapeutics Inc, H1 2018

Fabry Disease - Pipeline by Genzyme Corp, H1 2018

Fabry Disease - Pipeline by greenovation Biotech GmbH, H1 2018

Fabry Disease - Pipeline by iBio Inc, H1 2018

Fabry Disease - Pipeline by Idorsia Ltd, H1 2018

Fabry Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018

Fabry Disease - Pipeline by Pharming Group NV, H1 2018

Fabry Disease - Pipeline by Protalix BioTherapeutics Inc, H1 2018

Fabry Disease - Pipeline by Resverlogix Corp, H1 2018

Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H1 2018

Fabry Disease - Dormant Projects, H1 2018

Fabry Disease - Discontinued Products, H1 2018

List of Figures:

Number of Products under Development for Fabry Disease, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Amicus Therapeutics Inc
  • Genzyme Corp
  • greenovation Biotech GmbH
  • iBio Inc
  • Idorsia Ltd
  • JCR Pharmaceuticals Co Ltd
  • Pharming Group NV
  • Protalix BioTherapeutics Inc
  • Resverlogix Corp
  • Sangamo Therapeutics Inc
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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