The report titled “Fabry Disease Pipeline Insight 2023” by DelveInsight offers extensive information on more than 18+ companies and 18+ pipeline drugs in the field of Fabry Disease. It presents detailed profiles of these drugs, encompassing both Fabry Disease clinical trials and nonclinical stage products. The Fabry Disease pipeline report also includes a thorough assessment of therapeutics based on product type, stage, route of administration, and molecule type. Additionally, it highlights the inactive pipeline products in this area.
The Fabry Disease Pipeline report provides an elaborate account of the drug, encompassing its mechanism of action, Fabry Disease clinical trials studies, Fabry Disease NDA approvals (if any), and product development endeavors such as technology advancements, collaborations pertaining to Fabry Disease licensing, mergers and acquisitions, funding, designations, and other relevant details concerning the product.
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Key Takeaways from the Fabry Disease Pipeline Report
- Over 18+ Fabry Disease companies are evaluating 18+ Fabry Disease pipeline therapies in various stages of development, and their anticipated acceptance in the Fabry Disease market would significantly increase market revenue.
- The leading Fabry Disease Companies are working in the market include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others
- Promising Fabry Disease Pipeline therapies in various stages of development include Recombinant human alpha galactosidase A (agalsidase beta), Venglustat (GZ402671), Agalsidase beta (GZ419828), Migalastat, Lucerastat, and others
- Venglustat (also known as ibiglustat) (Sanofi Genzyme) is an orally administered small molecule being developed for several rare diseases. It is an inhibitor of an enzyme called glucosylceramide synthase (GCS) and modifies the enzyme substrates. GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. When venglustat inhibits GCS, it prevents the synthesis of GL-1, thereby reducing the substrate of the following reactions that lead to the formation of Gb3 and its accumulation in the absence of α-galactosidase A. Due to this mechanism of action venglustat is called a substrate reduction therapy. The molecule is developed by Sanofi Genzyme and is in the Phase III (NCT05280548; NCT05206773) stage of development for FD. The company expects to file submission by 2025+.
- ST-920 (Sangamo Therapeutics) is a gene therapy that comprises an AAV vector carrying a GLA gene construct driven by a proprietary liver-specific promoter. It is designed to enable a patient’s liver to produce a long-lasting and continuous supply of the α-Gal A enzyme. Preclinical studies of ST-920 showed a positive response, and one-time administration in mice led to substantial increases in the activity of enzyme α-Gal A and marked reductions in the levels of the fatty molecules that accumulate in patients with this disease. Based on the positive response, the FDA accepted the IND application of the gene therapy for the treatment of the FD, and the company initiated the Phase I/II clinical study of the product for the treatment of FD. ST-920 was generally well tolerated in Phase I/II. Based on these data, Sangamo has initiated Phase III planning.
Fabry Disease Overview
Fabry disease is an inherited lysosomal storage disease caused by a nonfunctional or partially functional enzyme, alpha-galactosidase A (α-Gal A). Decreased activity of α-Gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout the body.
For further information, refer to the detailed Fabry Disease Unmet Needs, Fabry Disease Market Drivers, and Fabry Disease Market Barriers, click here for Fabry Disease Ongoing Clinical Trial Analysis
Fabry Disease Emerging Drugs Profile
- Pegunigalsidase Alfa: Protalix Biotherapeutics
- Venglustat: Sanofi
- 4D 310: 4D Molecular Therapeutics
Fabry Disease Pipeline Therapeutics Assessment
There are approx. 18+ key companies which are developing the therapies for Fabry Disease. The companies which have their Fabry Disease drug candidates in the most advanced stage, i.e. Preregistration include Protalix Biotherapeutics.
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Some of the companies in the Fabry Disease Therapeutics Market include-
Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others
Fabry Disease Pipeline Therapeutics Assessment
- Fabry Disease Assessment by Product Type
- Fabry Disease by Stage and Product Type
- Fabry Disease Assessment by Route of Administration
- Fabry Disease by Stage and Route of Administration
- Fabry Disease Assessment by Molecule Type
- Fabry Disease by Stage and Molecule Type
Dive deep into rich insights for drugs for Fabry Disease Pipeline, click here @ Fabry Disease Unmet Needs and Analyst Views
Scope of the Fabry Disease Pipeline Report
- Coverage- Global
- Fabry Disease Companies- Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others
- Fabry Disease Pipeline Therapies- Recombinant human alpha galactosidase A (agalsidase beta), Venglustat (GZ402671), Agalsidase beta (GZ419828), Migalastat, Lucerastat, and others.
- Fabry Disease Pipeline Segmentation: Product Type, Molecule Type, Route of Administration
Got Queries? Find out the related information on Fabry Disease Mergers and acquisitions, Fabry Disease Licensing Activities @ Fabry Disease Emerging Drugs, and Recent Trends
Table of Content
- Introduction
- Executive Summary
- Fabry Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Fabry Disease – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Fabry Disease Collaboration Deals
- Late Stage Products (Preregistration)
- Pegunigalsidase Alfa: Protalix Biotherapeutics
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Venglustat: Sanofi
- Drug profiles in the detailed report…..
- Early stage products (Phase I/II)
- 4D 310: 4D Molecular Therapeutics
- Drug profiles in the detailed report…..
- Inactive Products
- Fabry Disease Key Companies
- Fabry Disease Key Products
- Fabry Disease- Unmet Needs
- Fabry Disease- Market Drivers and Barriers
- Fabry Disease- Future Perspectives and Conclusion
- Fabry Disease Analyst Views
- Fabry Disease Key Companies
- Appendix
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