
Fabry Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Description
Fabry Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
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
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)
- 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.
Table of Contents
98 Pages
- 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
- AceLink Therapeutics Inc
- Amicus Therapeutics Inc
- Bioasis Technologies Inc
- Biosidus SA
- CANbridge Life Sciences Ltd
- CellGenTech Inc
- Chiesi Farmaceutici SpA
- Equaly SA
- Freeline Therapeutics Holdings Plc
- GC Pharma
- Hanmi Pharmaceuticals Co Ltd
- Idorsia Pharmaceutical Ltd
- M6P Therapeutics
- Pharming Group NV
- Resverlogix Corp
- Sangamo Therapeutics Inc
- Sanofi
- Sigilon Therapeutics Inc
- Takeda Pharmaceutical Co Ltd
- UniQure NV
- Yuhan Corp
- Fabry Disease – Drug Profiles
- 4D-310 – Drug Profile
- agalsidase alfa – Drug Profile
- agalsidase beta biosimilar – Drug Profile
- AL-1 – Drug Profile
- AL-2 – Drug Profile
- AMT-191 – Drug Profile
- apabetalone – Drug Profile
- ATGTX-701 – Drug Profile
- CAN-104 – Drug Profile
- CAN-201 – Drug Profile
- CDX-6311 – Drug Profile
- FLT-190 – Drug Profile
- GC-1134A – Drug Profile
- Gene Therapy to Activate GLA for Fabry Disease – Drug Profile
- HM-15421 – Drug Profile
- isaralgagene civaparvovec – Drug Profile
- lucerastat – Drug Profile
- M-051 – Drug Profile
- M-052 – Drug Profile
- M-053 – Drug Profile
- MG-2102A – Drug Profile
- pegunigalsidase alfa – Drug Profile
- PGN-005 – Drug Profile
- Recombinant Enzyme to Replace Alpha Galactosidase A for Fabry Disease – Drug Profile
- RPV-001 – Drug Profile
- SIG-007 – Drug Profile
- venglustat malate – Drug Profile
- XB-3004 – Drug Profile
- YH-36425 – Drug Profile
- YHC-1116 – Drug Profile
- YHNCE-10 – Drug Profile
- 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
- Table 1: Number of Products under Development for Fabry Disease, 2022
- Table 2: Number of Products under Development by Companies, 2022
- Table 3: Number of Products under Development by Universities/Institutes, 2022
- Table 4: Products under Development by Companies, 2022
- Table 5: Products under Development by Companies, 2022 (Contd..1)
- Table 6: Products under Development by Universities/Institutes, 2022
- Table 7: Number of Products by Stage and Target, 2022
- Table 8: Number of Products by Stage and Mechanism of Action, 2022
- Table 9: Number of Products by Stage and Route of Administration, 2022
- Table 10: Number of Products by Stage and Molecule Type, 2022
- Table 11: Fabry Disease – Pipeline by 4D Molecular Therapeutics Inc, 2022
- Table 12: Fabry Disease – Pipeline by AceLink Therapeutics Inc, 2022
- Table 13: Fabry Disease – Pipeline by Amicus Therapeutics Inc, 2022
- Table 14: Fabry Disease – Pipeline by Bioasis Technologies Inc, 2022
- Table 15: Fabry Disease – Pipeline by Biosidus SA, 2022
- Table 16: Fabry Disease – Pipeline by CANbridge Life Sciences Ltd, 2022
- Table 17: Fabry Disease – Pipeline by CellGenTech Inc, 2022
- Table 18: Fabry Disease – Pipeline by Chiesi Farmaceutici SpA, 2022
- Table 19: Fabry Disease – Pipeline by Equaly SA, 2022
- Table 20: Fabry Disease – Pipeline by Freeline Therapeutics Holdings Plc, 2022
- Table 21: Fabry Disease – Pipeline by GC Pharma, 2022
- Table 22: Fabry Disease – Pipeline by Hanmi Pharmaceuticals Co Ltd, 2022
- Table 23: Fabry Disease – Pipeline by Idorsia Pharmaceutical Ltd, 2022
- Table 24: Fabry Disease – Pipeline by M6P Therapeutics, 2022
- Table 25: Fabry Disease – Pipeline by Pharming Group NV, 2022
- Table 26: Fabry Disease – Pipeline by Resverlogix Corp, 2022
- Table 27: Fabry Disease – Pipeline by Sangamo Therapeutics Inc, 2022
- Table 28: Fabry Disease – Pipeline by Sanofi, 2022
- Table 29: Fabry Disease – Pipeline by Sigilon Therapeutics Inc, 2022
- Table 30: Fabry Disease – Pipeline by Takeda Pharmaceutical Co Ltd, 2022
- Table 31: Fabry Disease – Pipeline by UniQure NV, 2022
- Table 32: Fabry Disease – Pipeline by Yuhan Corp, 2022
- Table 33: Fabry Disease – Dormant Projects, 2022
- Table 34: Fabry Disease – Discontinued Products, 2022
- List of Figures
- Figure 1: Number of Products under Development for Fabry Disease, 2022
- Figure 2: Number of Products under Development by Companies, 2022
- Figure 3: Number of Products under Development by Universities/Institutes, 2022
- Figure 4: Number of Products by Targets, 2022
- Figure 5: Number of Products by Stage and Targets, 2022
- Figure 6: Number of Products by Mechanism of Actions, 2022
- Figure 7: Number of Products by Stage and Mechanism of Actions, 2022
- Figure 8: Number of Products by Routes of Administration, 2022
- Figure 9: Number of Products by Stage and Routes of Administration, 2022
- Figure 10: Number of Products by Molecule Types, 2022
- Figure 11: Number of Products by Stage and Molecule Types, 2022
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