Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 2019, 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 2019, 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, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 5, 1, 7 and 2 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.
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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
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
4D Molecular Therapeutics Inc
Abeona Therapeutics Inc
Amicus Therapeutics Inc
AVROBIO Inc
Biosidus SA
Chiesi Farmaceutici SpA
Freeline Therapeutics Ltd
Genzyme Corp
Greenovation Biotech GmbH
Idorsia Pharmaceutical Ltd
Moderna Therapeutics Inc
Pharming Group NV
Resverlogix Corp
Sangamo Therapeutics Inc
Sigilon Therapeutics Inc
UniQure NV
Yuhan Corp
Fabry Disease - Drug Profiles
4D-310 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
agalsidase beta biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AMT-190 - 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
FLT-190 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 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
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
Sig-007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ST-920 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
venglustat malate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
YHNCE-10 - 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
Nov 18, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici announce successful pre-BLA meeting with FDA for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
Oct 17, 2019: Protalix BioTherapeutics announces positive 12-month interim data from the BRIDGE phase III open label switch-over study of Pegunigalsidase Alfa for the treatment of Fabry Disease
Sep 25, 2019: Protalix and Chiesi close enrolment in Phase III Fabry disease trial
Aug 02, 2019: Amicus Therapeutics receives marketing authorization for galafold (migalastat) for fabry disease in Argentina
Jul 15, 2019: AVROBIO announces 87% substrate reduction in first kidney biopsy and additional positive data from clinical trials of AVR-RD-01 investigational gene therapy in Fabry Disease
Jul 02, 2019: Handok releases Fabry disease treatment
Jun 17, 2019: Protalix Biotherapeutics completes enrollment in the phase III bright clinical trial of Pegunigalsidase Alfa (PRX 102) for the treatment of Fabry disease
Jun 06, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici to apply for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
May 30, 2019: 4D Molecular Therapeutics announces presentation of preclinical data at the 6th International Update on Fabry Disease and provides clinical update
May 02, 2019: uniQure announces new preclinical data on fabry disease in oral presentations at the 22nd ASGCT Annual Meeting
May 01, 2019: Avrobio to incorporate plato platform into FAB-201 Phase II trial
Apr 15, 2019: uniQure to present data on Gene Therapy for Fabry Disease at ASGCT 2019
Mar 18, 2019: Resverlogix proudly announces funding for phase 2 trial evaluating Apabetalone in Pulmonary Arterial Hypertension led by Quebec Heart and Lung Institute - Laval University researcher
Feb 20, 2019: Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
Feb 06, 2019: AVROBIO announces updated clinical data from ongoing phase 1 and phase 2 studies for AVR-RD-01 gene therapy for fabry disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Fabry Disease, H2 2019
Table 2: Number of Products under Development by Companies, H2 2019
Table 3: Number of Products under Development by Universities/Institutes, H2 2019
Table 4: Products under Development by Companies, H2 2019
Table 5: Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Table 6: Products under Development by Universities/Institutes, H2 2019
Table 7: Number of Products by Stage and Target, H2 2019
Table 8: Number of Products by Stage and Mechanism of Action, H2 2019
Table 9: Number of Products by Stage and Route of Administration, H2 2019
Table 10: Number of Products by Stage and Molecule Type, H2 2019
Table 11: Fabry Disease - Pipeline by 4D Molecular Therapeutics Inc, H2 2019
Table 12: Fabry Disease - Pipeline by Abeona Therapeutics Inc, H2 2019
Table 13: Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2019
Table 14: Fabry Disease - Pipeline by AVROBIO Inc, H2 2019
Table 15: Fabry Disease - Pipeline by Biosidus SA, H2 2019
Table 16: Fabry Disease - Pipeline by Chiesi Farmaceutici SpA, H2 2019
Table 17: Fabry Disease - Pipeline by Freeline Therapeutics Ltd, H2 2019
Table 18: Fabry Disease - Pipeline by Genzyme Corp, H2 2019
Table 19: Fabry Disease - Pipeline by Greenovation Biotech GmbH, H2 2019
Table 20: Fabry Disease - Pipeline by Idorsia Pharmaceutical Ltd, H2 2019
Table 21: Fabry Disease - Pipeline by Moderna Therapeutics Inc, H2 2019
Table 22: Fabry Disease - Pipeline by Pharming Group NV, H2 2019
Table 23: Fabry Disease - Pipeline by Resverlogix Corp, H2 2019
Table 24: Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H2 2019
Table 25: Fabry Disease - Pipeline by Sigilon Therapeutics Inc, H2 2019
Table 26: Fabry Disease - Pipeline by UniQure NV, H2 2019
Table 27: Fabry Disease - Pipeline by Yuhan Corp, H2 2019
Table 28: Fabry Disease - Dormant Projects, H2 2019
Table 29: Fabry Disease - Discontinued Products, H2 2019
List of Figures
Figure 1: Number of Products under Development for Fabry Disease, H2 2019
Figure 2: Number of Products under Development by Companies, H2 2019
Figure 3: Number of Products by Targets, H2 2019
Figure 4: Number of Products by Stage and Targets, H2 2019
Figure 5: Number of Products by Mechanism of Actions, H2 2019
Figure 6: Number of Products by Stage and Mechanism of Actions, H2 2019
Figure 7: Number of Products by Routes of Administration, H2 2019
Figure 8: Number of Products by Stage and Routes of Administration, H2 2019
Figure 9: Number of Products by Molecule Types, H2 2019
Figure 10: Number of Products by Stage and Molecule Types, H2 2019
