(Albany, United States) As per DelveInsight’s assessment, globally, the Friedreich’s Ataxia pipeline constitutes 10+ key companies continuously working towards developing 10+ Friedreich’s Ataxia treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analysis DelveInsight.
In the Friedreich’s Ataxia Pipeline Report, a detailed description of the drug is given which includes the mechanism of action of the drug, Friedreich’s Ataxia clinical trials studies, Friedreich’s Ataxia NDA approvals (if any), and product development activities comprising the technology, Friedreich’s Ataxia collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.
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Key Takeaways from the Friedreich’s Ataxia Pipeline Report
- DelveInsight’s Friedreich’s Ataxia Pipeline analysis depicts a robust space with 10+ active players working to develop 10+ pipeline treatment therapies.
- The leading Friedreich’s Ataxia companies working in the market include PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, and others
- Promising Friedreich’s Ataxia Pipeline Therapies in the various stages of development include TAK-831, Omaveloxolone Capsules, 2.5 mg, MIN-102, Deferiprone oral solution 100mg/mL, MIB-626, and others
- On June 2023, PTC Therapeutics announced a study of phase 3 clinical trials for Vatiquinone. The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.
- On March 2023, Design Therapeutics Inc announced a study of phase 1 clinical trials for DT-216. The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
Friedreich’s Ataxia Overview
Friedreich’s Ataxia (FRDA) is a genetic, progressive, neurodegenerative movement disorder, with a typical age of onset between 10 and 15 years. Initial symptoms may include unsteady posture, frequent falling, and progressive difficulty in walking due to impaired ability to coordinate voluntary movements (ataxia). Affected individuals often develop slurred speech (dysarthria), characteristic foot deformities, and an irregular curvature of the spine (scoliosis).
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Friedreich’s Ataxia Emerging Drugs Profile
- RT 001: Retrotope
- Leriglitazone: Minoryx Therapeutics
Friedreich’s Ataxia Therapeutics Assessment
There are approx. 10+ key companies which are developing the Friedreich’s Ataxia. The companies which have their Friedreich’s Ataxia drug candidates in the most advanced stage, i.e. Phase III include, Retrotope.
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Friedreich’s Ataxia Drugs and Companies
- TAK-831: Neurocrine Biosciences/Takeda
- Omaveloxolone Capsules, 2.5 mg: Reata Pharmaceuticals Inc
- MIN-102: Minoryx Therapeutics S.L
- Deferiprone oral solution 100mg/mL: ApoPharma
- MIB-626: Metro International Biotech LLC
Friedreich’s Ataxia Therapeutics Assessment
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
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Scope of the Friedreich’s Ataxia Pipeline Report
- Coverage- Global
- Friedreich’s Ataxia Companies- PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, and others
- Friedreich’s Ataxia Pipeline Therapies- TAK-831, Omaveloxolone Capsules, 2.5 mg, MIN-102, Deferiprone oral solution 100mg/mL, MIB-626, and others
- Friedreich’s Ataxia Segmentation: Product Type, Molecule Type, Route of Administration
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Table of Content
- Introduction
- Executive Summary
- Friedreich’s Ataxia: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Friedreich’s Ataxia– DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Friedreich’s Ataxia Collaboration Deals
- Late Stage Products (Phase III)
- RT 001: Retrotope
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Leriglitazone: Minoryx Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- CTI-1601: Larimar Therapeutics
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- LX-2006: LEXEO Therapeutics
- Drug profiles in the detailed report…..
- Inactive Products
- Friedreich’s Ataxia Key Companies
- Friedreich’s Ataxia Key Products
- Friedreich’s Ataxia- Unmet Needs
- Friedreich’s Ataxia- Market Drivers and Barriers
- Friedreich’s Ataxia- Future Perspectives and Conclusion
- Friedreich’s Ataxia Analyst Views
- Friedreich’s Ataxia Key Companies
- Appendix
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