Friedreich’s Ataxia Market to Grow at a Substantial Growth Rate During the Forecast Period | Reata Pharmaceuticals, Aavantibio, Minoryx, Retrotope

June 13 08:15 2024
Friedreich’s Ataxia Market to Grow at a Substantial Growth Rate During the Forecast Period | Reata Pharmaceuticals, Aavantibio, Minoryx, Retrotope
Friedreich’s Ataxia Market
As per DelveInsight, Friedreich’s Ataxia Market is anticipated to grow in the coming years owing to the rise in prevalence and expedited R&D activities of the pharma giants towards developing new drug therapy options to treat Friedreich’s Ataxia. Currently, key players such as Reata Pharmaceuticals (Omaveloxolone), Retrotope (RT001), PTC Therapeutics (Vatiquinone), Minoryx Therapeutics (Leriglitazone), among others, are actively involved in the development of novel therapeutic products.

DelveInsight’s “Friedreich’s Ataxia Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Friedreich’s Ataxia market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The Friedreich’s Ataxia market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market. 

Friedreich’s Ataxia Overview

The primary treatment goals for Friedreich’s Ataxia focus on managing symptoms, slowing disease progression, and enhancing the quality of life for patients. This often requires a multidisciplinary approach, including:

  • Physical Therapy: To improve strength, coordination, and balance, and to alleviate symptoms such as muscle stiffness and unsteady gait.

  • Occupational Therapy: To assist with daily activities and improve functional abilities.

  • Speech Therapy: To address issues like slurred speech and to improve communication skills.

  • Medications: To manage symptoms such as muscle stiffness, loss of reflexes, and fatigue.

Current and Future Therapies

Currently, there is no medication specifically approved to treat the genetic cause of Friedreich’s Ataxia. However, medications can be prescribed to help manage symptoms and improve quality of life for those affected by the condition.

Research and Development

Ongoing research aims to develop therapies that target the underlying genetic cause of Friedreich’s Ataxia, with the ultimate goal of finding a cure. Scientists are exploring various approaches to correct or mitigate the effects of the defective FXN gene responsible for the disease.

Recent advancements in the Friedreich’s Ataxia Market:

  • In February 2023, the US FDA approved the use of SKYCLARYS (omaveloxolone) to treat Friedreich’s Ataxia. It is a significant milestone as the first and only FDA-approved treatment for Friedreich’s Ataxia in individuals aged 16 years old and above. With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, Friedreich’s Ataxia market in the 7MM is expected to change significantly during the study period 2020–2034.

Key Highlights from the Friedreich’s Ataxia Market Report:

  • The Friedreich’s Ataxia market size is projected to experience growth at a consistent CAGR during the forecast period (2024-2034). The growth in market revenue is primarily driven by the increasing research and development activities and the development of novel treatments.

  • Friedreich’s Ataxia is one of the most common forms of inherited ataxia, with the condition typically manifesting in childhood or adolescence and progressing over time. Despite its rarity, it presents significant challenges due to its progressive nature and debilitating symptoms.

  • The current treatment regime is restrictive with only one approved product, and there is a critical need for disease-modifying therapies that can address the underlying genetic defect and provide meaningful improvements in the quality of life for individuals.

  • Increased focus on drug development, advances in genetics and genomics, and a growing market for precision medicine, among other factors may change the market scenario in times to come. Several companies are in the market race as their drugs are in the pipeline to excel in the coming years like vatiquinone (PTC Therapeutics), MIB-626 (Metro International Biotech), leriglitazone (Minoryx Therapeutics), and others.

Friedreich’s Ataxia Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current and forecasted Friedreich’s Ataxia market size by analyzing the impact of current and emerging pipeline therapies. It also thoroughly assesses the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete detail of the Friedreich’s Ataxia market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Friedreich’s Ataxia Epidemiology Assessment 

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders. 

  • As per research findings, Friedreich’s ataxia is the most common hereditary ataxia that appears to affect males and females equally. The prevalence of Friedreich’s ataxia is approximately 1 in 40,000 to 50,000. This disease frequency has been estimated at 1 in every 50,000 individuals in the United States.

  • A study reported the analyzed Friedreich’s ataxia prevalence which is the most common of the inherited ataxias across most of Europe. Its prevalence is highest in Western Europe, with more than 1 case per 30,000 individuals.

  • According to the research study, the prevalence of Friedreich’s ataxia in Japan is 13.1 per 100,000 individuals.

The Report Covers the Friedreich’s Ataxia Epidemiology, Segmented as –

  • Total Prevalent Cases of Friedreich’s Ataxia [2020–2034]

  • Diagnosed and Treatable Population of Friedreich’s Ataxia [2020–2034]

  • Prevalence of Friedreich’s Ataxia based on onset types [2020–2034]

Friedreich’s Ataxia Drugs Uptake and Pipeline Development Activities

The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Friedreich’s Ataxia market or expected to be launched during the study period. The analysis covers Friedreich’s Ataxia market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Friedreich’s Ataxia pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Learn How the Friedreich’s Ataxia Market Will Evolve and Grow by 2034 @

Friedreich’s Ataxia Therapeutics Analysis

  • Friedreich’s Ataxia Marketed Drugs

SKYCLARYS (omaveloxolone): Reata Pharmaceuticals

SKYCLARYS, developed by Reata Pharmaceuticals, is a groundbreaking therapy that Skyclarys is designed to activate nuclear factor erythroid 2-related factor 2 (NrF2), a type of protein called a transcription factor whose signaling is impaired in FA patients. NrF2 works to activate genes that promote mitochondrial function, boost antioxidant responses, and prevent inflammation.

Friedreich’s Ataxia Emerging Drugs

  • Vatiquinone: PTC Therapeutics

Vatiquinone is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress and inflammation response pathways that underpin neurological disease pathology. In March 2014, the US FDA granted fast-track designation to Vatiquinone for the treatment of Friedreich’s Ataxia. It has also received orphan drug designation from the US FDA for the treatment of Friedreich’s Ataxia. The company recently announced results from the Phase III MOVE-FA study evaluating vatiquinone (PTC743) in children and young adults with Friedreich’s ataxia and held a meeting with the US FDA. Based on the US FDA feedback from the Live Type C Meeting, the company plans to submit an NDA for vatiquinone for the treatment of Friedreich’s ataxia in late 2024.

  • In addition, LX2006 (Lexeo Therapeutics) and elamipretide (Stealth BioTherapeutics) are in the early phase of clinical development for the treatment of Friedreich’s ataxia.

Leading Companies in the Friedreich’s Ataxia Therapeutics Market Include:

  • AavantiBio

  • Design Therapeutics

  • Exicure

  • Lacerta Therapeutics

  • Larimar Therapeutics

  • LEXEO Therapeutics

  • Metro International Biotech

  • Minoryx Therapeutics

  • PTC Therapeutics

  • Reata Pharmaceuticals

  • Retrotope

  • StrideBio

  • Voyager Therapeutics

Friedreich’s Ataxia Emerging and Marketed Drugs Covered in the Report Include:

  • CTI-1601: Larimar Therapeutics

  • FXN gene therapy: AavantiBio

  • Leriglitazone: Minoryx Therapeutics

  • MIB-626: Metro International Biotech

  • Omaveloxolone: Reata Pharmaceuticals

  • RT001: Retrotope

  • SynTEFs: Design Therapeutics

  • Vatiquinone: PTC Therapeutics

The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @

Table of Contents

1. Key Insights

2. Executive Summary 

3. Friedreich’s Ataxia Competitive Intelligence Analysis

4. Friedreich’s Ataxia Market Overview at a Glance

5. Friedreich’s Ataxia Disease Background and Overview

6. Friedreich’s Ataxia Patient Journey

7. Friedreich’s Ataxia Epidemiology and Patient Population (In the US, EU5, and Japan)

8. Friedreich’s Ataxia Treatment Algorithm, Current Treatment, and Medical Practices

9. Friedreich’s Ataxia Unmet Needs

10. Key Endpoints of Friedreich’s Ataxia Treatment

11. Friedreich’s Ataxia Marketed Products

12. Friedreich’s Ataxia Emerging Drugs and Latest Therapeutic Advances

13. Friedreich’s Ataxia Seven Major Market Analysis

14. Attribute Analysis

15. Friedreich’s Ataxia Market Outlook (In US, EU5, and Japan)

16. Friedreich’s Ataxia Access and Reimbursement Overview

17. KOL Views on the Friedreich’s Ataxia Market

18. Friedreich’s Ataxia Market Drivers

19. Friedreich’s Ataxia Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

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