Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum

Yahoo

05-06-2025

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period.
LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Friedreich's Ataxia Market Report
According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034.
The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals.
Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing.
Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years.
The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others.
Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report
Friedreich's Ataxia Market Dynamics
The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology.
The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification.
Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM.
However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies.
Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth.
Friedreich's Ataxia Treatment Market
Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected.
The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective.
Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles.
Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation.
SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects.
It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone.
To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication
Friedreich's Ataxia Pipeline Therapies and Key Companies
Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others.
Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025.
RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021.
Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition.
The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials
Recent Developments in the Friedreich's Ataxia Market
In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression.
In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review.
In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia.
Friedreich's Ataxia Overview
Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination.
The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease.
In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech.
Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis.
Friedreich's Ataxia Epidemiology Segmentation
The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Diagnosed Prevalent Cases of Friedreich's Ataxia
Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia
Total Treated Cases of Friedreich's Ataxia
Friedreich's Ataxia Market Report Metrics
Details
Study Period
2020–2034
Coverage
7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan].
Key Friedreich's Ataxia Companies
Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, Reata Pharmaceuticals, and others
Key Friedreich's Ataxia Therapies
RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, SKYCLARYS, and others
Scope of the Friedreich's Ataxia Market Report
Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies
Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook
Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement
Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market
Table of Contents
1.
Friedreich's Ataxia Market Key Insights
2.
Friedreich's Ataxia Market Report Introduction
3.
Friedreich's Ataxia Market Overview at a Glance
4.
Friedreich's Ataxia Market Executive Summary
5.
Disease Background and Overview
6.
Friedreich's Ataxia Treatment and Management
7.
Friedreich's Ataxia Epidemiology and Patient Population
8.
Patient Journey
9.
Friedreich's Ataxia Marketed Drugs
10.
Friedreich's Ataxia Emerging Drugs
11.
Seven Major Friedreich's Ataxia Market Analysis
12.
Friedreich's Ataxia Market Outlook
13.
Potential of Current and Emerging Therapies
14.
KOL Views
15.
Unmet Needs
16.
SWOT Analysis
17.
Appendix
18.
DelveInsight Capabilities
19.
Disclaimer
20.
About DelveInsight
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