Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Drugs In Development, 2022, 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 - Drugs In Development, 2022, 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, IND/CTA Filed, Preclinical and Discovery stages are 1, 3, 3, 2, 1, 15 and 5 respectively. Similarly, the Universities portfolio in Preclinical, Discovery and Unknown stages comprises 1, 1 and 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
Fabry Disease - Drug Profiles
Fabry Disease - Dormant Projects
Fabry Disease - Discontinued Products
Fabry Disease - Product Development Milestones
Featured News & Press Releases
Apr 04, 2022: Protalix BioTherapeutics and Chiesi Global Rare Diseases announce topline results from the 24-month Phase III BALANCE clinical trial of PRX-102 for the treatment of Fabry disease
Mar 24, 2022: Freeline announces updated development plan and timelines for FLT190 for people with Fabry disease
Mar 21, 2022: Protalix, Chiesi report data from Phase III Fabry disease trial
Feb 24, 2022: Protalix BioTherapeutics and Chiesi Global Rare Diseases announce the submission of a marketing authorization application to the European Medicines Agency for PRX-102 for the treatment of Fabry Disease
Feb 09, 2022: 4D Molecular Therapeutics announces updated interim results from the 4D-310 phase 1/2 clinical trial in patients with Fabry Disease at the 18th Annual WORLDSymposium
Feb 08, 2022: Freeline presents on its Fabry disease AAV-based gene therapies at the 18th Annual WORLDSymposium
Feb 07, 2022: Sangamo Therapeutics announces updated preliminary phase 1/2 data showing tolerability and sustained elevated a-Gal a enzyme activity in patients With Fabry Disease
Feb 03, 2022: Protalix BioTherapeutics to participate in the 18th Annual WORLDSymposium 2022
Feb 02, 2022: 4D Molecular Therapeutics to present updated clinical data with 4D-310 in Fabry Disease patients in platform presentation at the 18th Annual WORLDSymposium
Jan 27, 2022: Freeline to present on its fabry disease AAV-based gene therapies at the 18th Annual WORLDSymposium
Dec 13, 2021: Idorsia to further characterize lucerastat for the treatment of Fabry disease by continuing the open-label extension of the Phase 3 MODIFY study
Nov 04, 2021: Sangamo Therapeutics announces preliminary phase 1/2 data showing tolerability and sustained elevated a-Gal A enzyme activity in patients with Fabry disease
Oct 25, 2021: 4D Molecular Therapeutics reports interim results from the 4D-310 phase 1/2 clinical trial in patients with fabry disease
Oct 15, 2021: Protalix Biotherapeutics and Chiesi Global Rare Diseases announce final dosing of last patient in phase III BALANCE clinical trial PRX-102 for the treatment of Fabry disease
Oct 11, 2021: Idorsia announces the results of MODIFY, a Phase 3 study of lucerastat in Fabry disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Fabry Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Fabry Disease - Pipeline by 4D Molecular Therapeutics Inc, 2022
Fabry Disease - Pipeline by AceLink Therapeutics Inc, 2022
Fabry Disease - Pipeline by Amicus Therapeutics Inc, 2022
Fabry Disease - Pipeline by Bioasis Technologies Inc, 2022
Fabry Disease - Pipeline by Biosidus SA, 2022
Fabry Disease - Pipeline by CANbridge Life Sciences Ltd, 2022
Fabry Disease - Pipeline by CellGenTech Inc, 2022
Fabry Disease - Pipeline by Chiesi Farmaceutici SpA, 2022
Fabry Disease - Pipeline by Equaly SA, 2022
Fabry Disease - Pipeline by Freeline Therapeutics Holdings Plc, 2022
Fabry Disease - Pipeline by GC Pharma, 2022
Fabry Disease - Pipeline by Hanmi Pharmaceuticals Co Ltd, 2022
Fabry Disease - Pipeline by Idorsia Pharmaceutical Ltd, 2022
Fabry Disease - Pipeline by M6P Therapeutics, 2022
Fabry Disease - Pipeline by Pharming Group NV, 2022
Fabry Disease - Pipeline by Resverlogix Corp, 2022
Fabry Disease - Pipeline by Sangamo Therapeutics Inc, 2022
Fabry Disease - Pipeline by Sanofi, 2022
Fabry Disease - Pipeline by Sigilon Therapeutics Inc, 2022
Fabry Disease - Pipeline by Takeda Pharmaceutical Co Ltd, 2022
Fabry Disease - Pipeline by UniQure NV, 2022
Fabry Disease - Pipeline by Yuhan Corp, 2022
Fabry Disease - Dormant Projects, 2022
Fabry Disease - Discontinued Products, 2022
List of Figures
Number of Products under Development for Fabry Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022
