Market Research Logo

Fabry Disease - Pipeline Review, H2 2018

Fabry Disease - Pipeline Review, H2 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 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

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H2 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 and Preclinical stages are 2, 2, 4 and 7 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 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.


Introduction
Global Markets Direct 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
Avrobio Inc
Biosidus SA
Chiesi Farmaceutici SpA
Genzyme Corp
greenovation Biotech GmbH
iBio Inc
Idorsia Pharmaceutical Ltd
JCR Pharmaceuticals Co Ltd
Moderna Therapeutics Inc
Pharming Group NV
Resverlogix Corp
Sangamo Therapeutics Inc
Fabry Disease - Drug Profiles
(ATB-101 + migalastat hydrochloride) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
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
mRNA-3630 - 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
Aug 10, 2018: FDA approves Galafold (migalastat) for the treatment of certain adult patients with Fabry Disease
May 30, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Fabry Disease in Japan
May 25, 2018: Protalix BioTherapeutics Announces Poster Presentation on Baseline Characteristics for Fabry Disease Patients Screened in the Phase III BALANCE Study of Pegunigalsidase Alfa at the 55th ERA-EDTA Congress
May 16, 2018: Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option for patients with Fabry disease
Mar 22, 2018: Amicus Therapeutics Announces Approval of Galafold (Migalastat) for Fabry Disease in Japan
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
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 2018
Number of Products under Development by Companies, H2 2018
Number of Products under Development by Universities/Institutes, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Universities/Institutes, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2018
Fabry Disease - Pipeline by Avrobio Inc, H2 2018
Fabry Disease - Pipeline by Biosidus SA, H2 2018
Fabry Disease - Pipeline by Chiesi Farmaceutici SpA, H2 2018
Fabry Disease - Pipeline by Genzyme Corp, H2 2018
Fabry Disease - Pipeline by greenovation Biotech GmbH, H2 2018
Fabry Disease - Pipeline by iBio Inc, H2 2018
Fabry Disease - Pipeline by Idorsia Pharmaceutical Ltd, H2 2018
Fabry Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2018
Fabry Disease - Pipeline by Moderna Therapeutics Inc, H2 2018
Fabry Disease - Pipeline by Pharming Group NV, H2 2018
Fabry Disease - Pipeline by Resverlogix Corp, H2 2018
Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H2 2018
Fabry Disease - Dormant Projects, H2 2018
Fabry Disease - Discontinued Products, H2 2018
List of Figures
Number of Products under Development for Fabry Disease, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018

Download our eBook: How to Succeed Using Market Research

Learn how to effectively navigate the market research process to help guide your organization on the journey to success.

Download eBook

Share this report