Friedreich's Ataxia Market

Market Overview:

The 7 major Friedreich's ataxia markets reached a value of USD 660.4 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 1,825.0 Million by 2035, exhibiting a growth rate (CAGR) of 9.65% during 2025-2035.

The Friedreich's ataxia market has been comprehensively analyzed in IMARC's new report titled "Friedreich's Ataxia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Friedreich's ataxia is a rare recessive genetic disorder that causes progressive nervous system impairment and movement difficulties. It is caused by mutations in the FXN gene responsible for producing a protein called frataxin, which is necessary for the proper functioning of nerve cells. The symptoms of Friedreich's ataxia usually begin in childhood or adolescence and progressively worsen over time. The indications associated with this disorder are broad but consistently involve limb and gait ataxia, loss of lower limb reflexes, dysarthria, etc. Individuals suffering from Friedreich's ataxia may also experience trouble walking, tiredness, lack of sensation in the arms and legs, hearing loss, shortness of breath, heart palpitations, decreased vision, chest pain, etc. Diagnosing this ailment involves a combination of medical history and physical examination. Various tests, including electromyograms, electrocardiograms, nerve conduction studies, echocardiograms, genetic testing, etc., are adopted to detect the condition among patients. The healthcare professional may also perform MRI and CT scans of the spinal cord and brain to rule out other functional disorders.

The increasing prevalence of autosomal recessive genetic disorders and the rising clinical need for treatments that address the underlying genetic cause of such diseases are primarily driving the Friedreich's ataxia market. Apart from this, the widespread adoption of several medications, including diazoxide and dyclonine, to restore sufficient frataxin levels in the body and reduce the severity of the ailment is acting as another significant growth-inducing factor. Moreover, the escalating utilization of iron chelation therapy to decrease the accumulation of iron in cells, which further contributes to the development of the disorder, is also bolstering the market growth. Additionally, several key players are making extensive investments in introducing disease-modifying therapies that aim to slow the progression of the ailment by targeting oxidative stress, mitochondrial dysfunction, and iron accumulation in cells. This, in turn, is creating a positive outlook for the market. Furthermore, the ongoing development of reliable and accurate serum biomarkers that aid in improving the diagnosis and management of FA, allowing for earlier intervention and better outcomes for patients, is expected to drive the Friedreich's ataxia market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Friedreich’s ataxia market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Friedreich’s ataxia and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Friedreich’s ataxia market in any manner.

Recent Developments:

• In February 2024, PTC Therapeutics announced that the FDA has accepted NDA for Vatiquinone to treat Friedreich's ataxia (FA).
• In March 2024, Larimar Therapeutics, Inc. announced the dosing of the first patient in an open-label extension (OLE) trial assessing nomlabofusp subcutaneous injections at a dose of 25 mg per day for individuals with Friedreich's ataxia. Nomlabofusp (CTI-1601) is a new protein replacement therapy that targets the underlying cause of Friedreich's ataxia by delivering frataxin to the mitochondria.
• In March 2024, Lexeo published preliminary findings from a subset of their second dose cohort of the SUNRISE-FA trial, demonstrating a rise in frataxin levels three months after treatment with LX2006 compared to pre-treatment baseline levels for people with Friedreich's ataxia cardiomyopathy.
• In February 2024, Voyager Therapeutics, Inc. announced that the joint steering committee with Neurocrine Biosciences had chosen a lead development candidate for Friedreich's ataxia program. The candidate combines a frataxin (FXN) gene replacement payload with an intravenous, blood-brain barrier penetrant, new capsid from Voyager's TRACER capsid discovery platform.
• In February 2024, Biogen Inc. announced that the European Commission (EC) had approved SKYCLARYS (omaveloxolone) for the treatment of Friedreich's ataxia in adults and adolescents aged 16 and above. SKYCLARYS is the first medication approved in the European Union for this rare, genetically determined, progressive neurodegenerative disease.

Key Highlights:

• Friedreich's ataxia affects one in every 50,000 people in the United States, with Western Europeans having the highest prevalence.
• The worldwide prevalence of Friedreich's ataxia is 1 in 40,000.
• Friedreich's ataxia is a disease that affects young people, as the development of symptoms usually begins before the age of 20.
• Friedreich's ataxia is the most prevalent inherited ataxia in the Caucasian population. The estimated incidence in Caucasians is around 1 in 29,000, with a carrier chance of 1 in 85.
• Approximately 7% of patients with Friedreich's ataxia have diabetes.

Drugs:

Skyclarys (omaveloxolone) is FDA-approved medication in the United States for the treatment of Friedreich's ataxia in adults and adolescents aged 16 and above. The recommended dose is 150 mg to be administered orally once daily. Skyclarys is intended to stimulate nuclear factor erythroid 2-related factor 2 (NrF2), a transcription factor whose signaling is reduced in Friedreich's ataxia patients. NrF2 activates genes that improve mitochondrial function, increase antioxidant responses, and reduce inflammation.

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that regulates the energy and oxidative stress pathways that are disturbed in Friedreich's ataxia. Inhibiting 15-LO helps to reduce the effects of mitochondrial malfunction and oxidative stress, hence reducing ferroptosis and promoting neuronal survival.

Leriglitazone (MIN-102) is an orally bioavailable and selective PPARγ agonist with a potential best-in-class and first-in-class profile indicated for CNS diseases. It has demonstrated significant brain penetration and an acceptable safety profile. The medicine provided strong preclinical proof-of-concept in animal models of Friedreich's ataxia by changing pathways that lead to neuroinflammation, demyelination, and mitochondrial failure.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Friedreich's ataxia market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Friedreich's ataxia market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Friedreich's ataxia marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Friedreich's ataxia market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the Friedreich's ataxia market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the Friedreich's ataxia market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of Friedreich's ataxia across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Friedreich's ataxia by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Friedreich's ataxia by gender across the seven major markets?
• What is the number of prevalent cases (2019-2035) of Friedreich's ataxia by type across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with Friedreich's ataxia across the seven major markets?
• What is the size of the Friedreich's ataxia patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of Friedreich's ataxia?
• What will be the growth rate of patients across the seven major markets?
   

Friedreich's Ataxia: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Friedreich's ataxia drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Friedreich's ataxia market?
• What are the key regulatory events related to the Friedreich's ataxia market?
• What is the structure of clinical trial landscape by status related to the Friedreich's ataxia market?
• What is the structure of clinical trial landscape by phase related to the Friedreich's ataxia market?
• What is the structure of clinical trial landscape by route of administration related to the Friedreich's ataxia market?