Friedreich’s Ataxia Pipeline Assessment, 2024 Updates: FDA, EMA, and PMDA Approvals, Emerging Drugs, Clinical Trials, Therapeutic Analysis, and Growth Prospects | AbbVie, Minoryx Therapeutics, Takeda

May 08 09:02 2024
Friedreich’s Ataxia Pipeline Assessment, 2024 Updates: FDA, EMA, and PMDA Approvals, Emerging Drugs, Clinical Trials, Therapeutic Analysis, and Growth Prospects | AbbVie, Minoryx Therapeutics, Takeda
(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, 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 analyzes DelveInsight.

The Friedreich’s Ataxia Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.


Friedreich’s Ataxia Pipeline Insight, 2024 report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Friedreich’s Ataxia Market.


Some of the key takeaways from the Friedreich’s Ataxia Pipeline Report:

  • Companies across the globe are diligently working toward developing novel Friedreich’s Ataxia treatment therapies with a considerable amount of success over the years. 

  • Friedreich’s Ataxia companies working in the treatment market are Astellas Gene Therapies, Solid Biosciences, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, Metro International Biotech, LLC, PTC Therapeutics, Retrotope, and others, are developing therapies for the Friedreich’s Ataxia treatment 

  • Emerging Friedreich’s Ataxia therapies in the different phases of clinical trials are- ASP 2016, AVB-202, DT-216, CTI-1601, MIN-102, MIB-626, Vatiquinone, RT001, and others are expected to have a significant impact on the Friedreich’s Ataxia market in the coming years.  

  • In February 2024, Larimar Therapeutics, headquartered in the United States, has released encouraging top-line findings from a Phase II exploratory study of the Friedreich’s ataxia treatment, nomlabofusp (CTI-1601). CEO Dr. Carole Ben-Maimon has expressed intentions to initiate a confirmatory trial for the medication, with plans to submit a biologics license application (BLA) to the US Food and Drug Administration (FDA) in the second half of 2025.

  • In September 2023, Vatiquinone (PTC-743), previously recognized as EPI-743, is an experimental oral therapy aimed at mitigating neuroinflammation and nerve cell deterioration in individuals with Friedreich’s ataxia (FA). This treatment has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration for FA treatment, alongside orphan drug status in the European Union. These designations are intended to expedite clinical progress. PTC Therapeutics is spearheading its development after its acquisition from BioElectronTechnology Corporation in 2019. Moreover, investigations are underway to explore its efficacy in managing seizures among individuals with mitochondrial diseases.

  • In February 2023, The US FDA has granted approval to Reata Pharmaceuticals’ omaveloxolone, marketed as SKYCLARYS, for treating Friedreich’s Ataxia in individuals aged 16 years and older.


Friedreich’s Ataxia Overview

Friedreich’s ataxia (FA) is a rare genetic disorder characterized by progressive damage to the nervous system, leading to symptoms such as difficulty with balance and coordination (ataxia), muscle weakness, loss of sensation in the extremities, and impaired speech. It is caused by a mutation in the FXN gene, which leads to a deficiency of frataxin, a protein involved in mitochondrial function.


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Emerging Friedreich’s Ataxia Drugs Under Different Phases of Clinical Development Include:

  • ASP 2016: Astellas Gene Therapies

  • AVB-202: Solid Biosciences

  • DT-216: Design Therapeutics

  • CTI-1601: Larimar Therapeutics

  • MIN-102: Minoryx Therapeutics

  • MIB-626: Metro International Biotech, LLC

  • Vatiquinone: PTC Therapeutics

  • RT001: Retrotope


Friedreich’s Ataxia Route of Administration

Friedreich’s Ataxia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as 

  • Infusion

  • Intradermal

  • Intramuscular

  • Intranasal

  • Intravaginal

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Molecule Type


Friedreich’s Ataxia Molecule Type

Friedreich’s Ataxia Products have been categorized under various Molecule types, such as

  • Vaccines

  • Monoclonal Antibody

  • Peptides

  • Polymer

  • Small molecule

  • Product Type


Friedreich’s Ataxia Pipeline Therapeutics Assessment

  • Friedreich’s Ataxia Assessment by Product Type

  • Friedreich’s Ataxia By Stage and Product Type

  • Friedreich’s Ataxia Assessment by Route of Administration

  • Friedreich’s Ataxia By Stage and Route of Administration

  • Friedreich’s Ataxia Assessment by Molecule Type

  • Friedreich’s Ataxia by Stage and Molecule Type


DelveInsight’s Friedreich’s Ataxia Report covers around 10+ products under different phases of clinical development like

  • Late-stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage product (Phase I)

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates

  • Route of Administration


Further Friedreich’s Ataxia product details are provided in the report. Download the Friedreich’s Ataxia pipeline report to learn more about the emerging Friedreich’s Ataxia therapies


Some of the key companies in the Friedreich’s Ataxia Therapeutics Market include:

Key companies developing therapies for Friedreich’s Ataxia are – AbbVie, Minoryx Therapeutics S.L., Takeda, Reata Pharmaceuticals Inc., Design Therapeutics Inc., ApoPharma, Metro International Biotech LLC, PTC Therapeutics, Neurocrine Biosciences, Larimar Therapeutics Inc., Veristat Inc., Retrotope Inc., and others.


Friedreich’s Ataxia Pipeline Analysis:

The Friedreich’s Ataxia pipeline report provides insights into 

  • The report provides detailed insights about companies that are developing therapies for the treatment of Friedreich’s Ataxia with aggregate therapies developed by each company for the same.

  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Friedreich’s Ataxia Treatment.

  • Friedreich’s Ataxia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • Friedreich’s Ataxia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Friedreich’s Ataxia market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.


Download Sample PDF Report to know more about Friedreich’s Ataxia drugs and therapies


Friedreich’s Ataxia Pipeline Market Drivers

  • Rapid research in the field of ataxia therapeutics, increasing collaborations and acquisitions among key players are some of the important factors that are fueling the Friedreich’s Ataxia Market.


Friedreich’s Ataxia Pipeline Market Barriers

  • However, there are currently no approved therapies for the treatment of patients with Friedreich’s ataxia and other factors are creating obstacles in the Friedreich’s Ataxia Market growth.


Scope of Friedreich’s Ataxia Pipeline Drug Insight    

  • Coverage: Global

  • Key Friedreich’s Ataxia Companies: Astellas Gene Therapies, Solid Biosciences, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, Metro International Biotech, LLC, PTC Therapeutics, Retrotope, and others

  • Key Friedreich’s Ataxia Therapies: ASP 2016, AVB-202, DT-216, CTI-1601, MIN-102, MIB-626, Vatiquinone, RT001, and others

  • Friedreich’s Ataxia Therapeutic Assessment: Friedreich’s Ataxia current marketed and Friedreich’s Ataxia emerging therapies

  • Friedreich’s Ataxia Market Dynamics: Friedreich’s Ataxia market drivers and Friedreich’s Ataxia market barriers 


Request for Sample PDF Report for Friedreich’s Ataxia Pipeline Assessment and clinical trials


Table of Contents

1. Friedreich’s Ataxia Report Introduction

2. Friedreich’s Ataxia Executive Summary

3. Friedreich’s Ataxia Overview

4. Friedreich’s Ataxia- Analytical Perspective In-depth Commercial Assessment

5. Friedreich’s Ataxia Pipeline Therapeutics

6. Friedreich’s Ataxia Late Stage Products (Phase II/III)

7. Friedreich’s Ataxia Mid Stage Products (Phase II)

8. Friedreich’s Ataxia Early Stage Products (Phase I)

9. Friedreich’s Ataxia Preclinical Stage Products

10. Friedreich’s Ataxia Therapeutics Assessment

11. Friedreich’s Ataxia Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Friedreich’s Ataxia Key Companies

14. Friedreich’s Ataxia Key Products

15. Friedreich’s Ataxia Unmet Needs

16 . Friedreich’s Ataxia Market Drivers and Barriers

17. Friedreich’s Ataxia Future Perspectives and Conclusion

18. Friedreich’s Ataxia Analyst Views

19. Appendix

20. About DelveInsight



About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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