Fabry Disease Pipeline, Clinical and Non-Clinical Stage Products, and FDA Approvals 2023 (Updated)
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.
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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
- 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
For further information on the Fabry Disease pipeline therapeutics, reach out to Fabry Disease Unmet Needs and Analyst Views
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