DelveInsight’s, “Fabry Disease Pipeline Insight 2023” report provides comprehensive insights about 18+ companies and 18+ pipeline drugs in the Fabry Disease pipeline landscape. It covers the Fabry Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Fabry Disease pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key Takeaways from the Fabry Disease Pipeline Report

• DelveInsight’s Fabry Disease pipeline report depicts a robust space with 18+ active players working to develop 18+ pipeline therapies for Fabry Disease treatment.
• The leading companies working in the Fabry Disease 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 the various stages of development include PRX-102, migalastat, Lucerastat, Enzyme replacement therapy (ERT), 4D-310, Recombinant human alpha galactosidase A (agalsidase beta), and others.
• January 2023: 4D Molecular Therapeutics announced a study of phase 1 & 2 clinical trials for 4D-310. This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement.
• September 2023: Protalix announced a study of phase 3 clinical trials for Pegunigalsidase Alfa. This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase alfa or agalsidase beta for at least 3 years. Safety and efficacy exploratory endpoints will be evaluated throughout the study period and pharmacokinetics will be obtained on Day 1 and Week 52.

 

Request a sample and discover the recent advances in Fabry Disease Treatment Drugs @ Fabry Disease Pipeline Report

 

In the Fabry Disease pipeline report, a detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Fabry Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Fabry Disease Overview

Fabry Disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that the body’s cells can use.

 

Find out more about Fabry Disease Therapeutics Assessment @ Fabry Disease Preclinical and Discovery Stage Products

 

Fabry Disease Emerging Drugs Profile

• Pegunigalsidase Alfa: Protalix Biotherapeutics
• 4D 310: 4D Molecular Therapeutics
• Venglustat: Sanofi

 

Fabry Disease Pipeline Therapeutics Assessment

There are approx. 18+ key companies which are developing the Fabry Disease therapies. The Fabry Disease companies which have their Fabry Disease drug candidates in the most advanced stage, i.e. Preregistration include Protalix Biotherapeutics.

 

Learn more about the emerging Fabry Disease Pipeline Therapies @ Fabry Disease Clinical Trials Assessment

 

Scope of the Fabry Disease Pipeline Report

• Coverage- Global
• Fabry Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Fabry Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• 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- PRX-102, migalastat, Lucerastat, Enzyme replacement therapy (ERT), 4D-310, Recombinant human alpha galactosidase A (agalsidase beta), and others.

 

Dive deep into rich insights for new drugs for Fabry Disease treatment, Visit @ Fabry Disease Market Drivers and Barriers, and Future Perspective

 

Table of Content

1. Introduction
2. Executive Summary
3. Fabry Disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Fabry Disease – DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Fabry Disease Collaboration Deals
9. Late Stage Products (Preregistration)
10. Pegunigalsidase Alfa: Protalix Biotherapeutics
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. Venglustat: Sanofi
14. Drug profiles in the detailed report…..
15. Early stage products (Phase I/II)
16. 4D 310: 4D Molecular Therapeutics
17. Drug profiles in the detailed report…..
18. Inactive Products
19. Fabry Disease Key Companies
20. Fabry Disease Key Products
21. Fabry Disease- Unmet Needs
22. Fabry Disease- Market Drivers and Barriers
23. Fabry Disease- Future Perspectives and Conclusion
24. Fabry Disease Analyst Views
25. Fabry Disease Key Companies
26. Appendix

 

For further information on the Fabry Disease pipeline therapeutics, reach out to Fabry Disease Unmet Needs and Analyst Views

 

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