United States Friedreich's Ataxia Market Set for Strong Growth with Breakthrough Therapies & Expanding Research Investments 2025 | Top key players - Biogen (Reata Pharmaceuticals, Inc.)

Market Size and Growth:

The Global Friedreich's Ataxia Market size reached US$ 0.96 Billion in 2024 and is expected to reach US$ 2.86 Billion by 2033, growing at a CAGR of 12.9% during the forecast period 2025-2033.

The Friedreichs Ataxia Market report by DataM Intelligence provides insights into the latest trends and developments in the market. This report identifies the key growth opportunities in the market and provides recommendations for market participants to capitalize on these opportunities. Overall, the Friedreichs Ataxia market report is an essential resource for market participants who are looking to gain a comprehensive understanding of the market and identify opportunities for growth.

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The Friedreich's Ataxia Market refers to the global industry focused on developing and commercializing therapies, diagnostics, and supportive care for Friedreich's Ataxia, a rare inherited neurodegenerative disorder. It encompasses pharmaceutical research, clinical trials, genetic testing, treatment innovations, and healthcare services aimed at managing symptoms, slowing disease progression, and improving quality of life for affected patients.

Recent Key Developments of United States (U.S):

FDA Rejection of Vatiquinone: On August 19, 2025, the FDA issued a Complete Response Letter (CRL) to PTC Therapeutics for their drug vatiquinone, stating that "substantial evidence of efficacy was not demonstrated" in the clinical trials.

Pediatric Clinical Trial of Omaveloxolone: Biogen announced the start of its global Phase 3 "BRAVE" study for omaveloxolone (Skyclarys) in children aged 2 to under 16 years. This follows the drug's initial approval for adolescents and adults aged 16 and older. The first patient was dosed on June 18, 2025.

Lexeo Therapeutics Gene Therapy Trial: In April 2025, Lexeo Therapeutics reported encouraging interim results from its LX2006 gene therapy trial for Friedreich's Ataxia cardiomyopathy.

Novel Genetic Research: A University of Oklahoma researcher, with a new $2.8 million grant from the U.S. Department of Defense, discovered "spelling errors" in the FXN gene's GAA repeat, a finding that could change how the condition is diagnosed and studied. The news was published on July 21, 2025.

Long-Term Study Data for Omaveloxolone: Ongoing studies continue to gather long-term safety and efficacy data for the approved drug, omaveloxolone (Skyclarys), with a focus on its effects on physical function, coordination, and safety over an extended period.

Key Players:

Biogen (Reata Pharmaceuticals, Inc.)

Emerging Key Players:

Ionis Pharmaceuticals
KalVista Pharmaceuticals, Inc.

This Report Covers:

✔ Go-to-market Strategy.

✔ Neutral perspective on the market performance.

✔Development trends, competitive landscape analysis, supply side analysis, demand side analysis, year-on-year growth, competitive benchmarking, vendor identification, and other significant analysis, as well as development status.

✔Customized regional/country reports as per request and country level analysis.

✔ Potential & niche segments and regions exhibiting promising growth covered.

✔ Analysis of Market Size (historical and forecast), Total Addressable Market (TAM), Serviceable Available Market (SAM), Serviceable Obtainable Market (SOM), Market Growth, Technological Trends, Market Share, Market Dynamics, Competitive Landscape and Major Players (Innovators, Start-ups, Laggard, and Pioneer).

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Market Segmentation:

By Disease Type: Late-onset Friedreich's Ataxia (LOFA), Very late-onset Friedreich's Ataxia (VLOFA).

By Medication: Antioxidants, Others.

Regional Analysis:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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Chapter Outline

⏩ Market Overview: It contains five chapters, as well as information about the research scope, major manufacturers covered, market segments, Friedreichs Ataxia market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Global Friedreichs Ataxia Market is evaluated here in terms of value, turnover, revenues, and market share by organization, as well as market rate, competitive landscape, and recent developments, transaction, growth, sale, and market shares of top companies.

⏩ Companies Profiles: The global Friedreichs Ataxia market's leading players are studied based on sales, main products, gross profit margin, revenue, price, and growth production.

⏩ Market Outlook by Region: The report goes through gross margin, sales, income, supply, market share, CAGR, and market size by region in this segment. North America, Europe, Asia Pacific, Middle East & Africa, and South America are among the regions and countries studied in depth in this study.

⏩ Market Segments: It contains the deep research study which interprets how different end-user/application/type segments contribute to the Friedreichs Ataxia Market.

⏩ Market Forecast: Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

⏩ Research Findings: This section of the report showcases the findings and analysis of the report.

⏩ Conclusion: This portion of the report is the last section of the report where the conclusion of the research study is provided.

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