Friedreich’s Ataxia Drug Market

Friedreich’s Ataxia Drug Market Outlook (2024 to 2034)

According to a newly published research report by Fact.MR, revenue from the sales of Friedreich’s ataxia drugs is estimated to reach US$ 1 billion in 2024. The global Friedreich’s ataxia drug market has been projected to expand at a double-digit CAGR of 13.2% and reach US$ 3.49 billion by the end of 2034.

Increasing cases of Friedreich’s ataxia due to genetic mutations are resulting in a declining production and mitochondrial protein expression, addressed as frataxin. It is closely working for the regulation of iron-sulfur cluster enzymes within human body cells. Further, insufficient frataxin levels are resulting in reduced functioning of several mitochondrial enzymes, which are involved in the transportation of electron chain, subsequently leading to impaired mitochondrial metabolism, such as reduced production of ATP (adenosine triphosphate), insufficient anti-oxidant response, and iron accumulation in mitochondria dysfunction.

These abovementioned factors hamper patients’ abilities to coordinate movements and perform daily activities, such as running, walking, and others. Some popular pharmacology therapy approaches utilized for the treatment or management of Friedreich’s ataxia, such as the use of several types of drugs, including beta blockers, ACE (angiotensin-converting enzyme) inhibitors, diuretics, skeletal muscle relaxants immunomodulators, anti-epileptic drugs, skeletal muscle relaxants, nuclear factor-erythroid factor 2-related Nrf2 inducer, and several others are widening opportunities for market players. Moreover, the use of advanced gene therapy against the treatment of Friedreich’s ataxia is resulting in the production of frataxin protein in the human body. These factors are contributing to Friedreich’s ataxia drug market growth.

• Demand for Friedreich’s Ataxia drugs in India is projected to increase at a CAGR of 18.6% and reach a market value of US$ 312.6 million by 2034-end.
• East Asia is forecasted to capture a share of 18.7% of global market revenue by 2034-end.
• Worldwide demand for Friedreich’s Ataxia drugs for use in hospitals is evaluated to increase at a CAGR of 13.1% and reach a market value of US$ 1.56 billion by the end of 2034.
• Sales of Friedreich’s ataxia drugs in Japan are projected to rise at a CAGR of 6.3% from 2024 to 2034.
• Demand for Friedreich’s Ataxia drugs in Canada is forecasted to increase at 16.1% CAGR through 2034.

Report Attribute	Detail
Friedreich’s Ataxia Drug Market Size (2024E)	US$ 1 Billion
Forecasted Market Value (2034F)	US$ 3.49 Billion
Global Market Growth Rate (2024 to 2034)	13.2% CAGR
Market Share of Small Molecule Therapy (2034F)	38.3%
North America Market Share (2034F)	36.7%
South Korea Market Growth Rate (2024 to 2034)	17.6% CAGR
Key Companies Profiled	Reata Pharmaceuticals, Inc.; Biogen Inc.; Retrotope Inc.; Intas Pharmaceuticals Ltd.; Minoryx; Torrent Pharmaceuticals Ltd.; PTC Therapeutics; Aurobindo Pharma Ltd.; Design Therapeutics, Inc.; GlaxoSmithKline Plc.; Larimar Therapeutics, Inc.; Sun Pharmaceutical Industries Ltd.; Cipla Limited; Jupiter Neurosciences, Inc.; Zydus Lifesciences Ltd.; Lexeo Therapeutics; Neurocrine Biosciences Inc.

Why is Demand for Friedreich’s Ataxia Drugs Rising at a Fast Pace?

“Crucial Role of Friedreich’s Ataxia Drugs in Treating Worsening Muscle Coordination”

Friedreich’s ataxia is a rare genetic disorder, which results in progressive damage to movement difficulties and the nervous system. This condition generally starts in childhood, thereby resulting in worsened muscle coordination with time. It arises from FXN gene mutation, which encodes protein frataxin. Kids inherit two gene defective copies, which results in disease development. Two defective gene copies are inherited in individuals, leading to Friedreich’s ataxia, thereby generating demand for effective drugs.

What is Adversely Impacting Demand Growth for Friedreich Ataxia Drugs?

“Challenges Associated with Designing and Conducting Clinical Trials for Friedreich’s Ataxia”

Several challenges are encountered during the designing and conducting of clinical trials against developing Friedreich’s ataxia drugs. Designing trials against rare neurodegenerative diseases, including Friedreich’s ataxia, is a bit tricky with a variable and slow progression rate. These factors make it a bit difficult to identify reliable outcome measures for monitoring the progression of Friedreich’s ataxia.

For instance:

• The Orphanet Journal of Rare Diseases came up with a report in August 2020. As per the report, it is a bit difficult to identify the symptoms of Friedreich’s ataxia, thereby resulting in a longer diagnosis time, which ultimately creates limited scope for drug development and demand.

What is the Contribution of Start-ups to Market Expansion?

“Expenditure on R&D Activities to Develop Genetic Testing Offering Definite Diagnosis”

Start-ups need to focus more on investing in R&D activities for developing Friedreich’s ataxia drugs through clinical examination. A thorough physical examination for balance issues, absence of reflexes, loss of joint sensation, and several other neurological symptoms. Development of genetic testing is offering definite diagnosis facilities for ensuring effective treatment. These factors are contributing to the expansion of the Friedreich’s ataxia drug market size.

Country-wise Insights

As opined in the newly published analysis by Fact.MR, a market research and competitive intelligence provider, North America is evaluated to account for 36.7% of the global Friedreich’s ataxia drug market share by the end of 2034. The presence of a well-established healthcare infrastructure in the region is opening wide opportunities for market players.

What Makes the United States a Key Market for Friedreich Ataxia Drug Manufacturers?

“Development and FDA Approval for First Treatment for Friedreich Ataxia”

Attribute	United States
Market Value (2024E)	US$ 331 Million
Growth Rate (2024 to 2034)	12.3% CAGR
Projected Value (2034F)	US$ 1.06 Billion

The United States is analyzed to hold 82.5% share of North America by 2034. Skyclarys has been approved by the United States FDA as the first effective treatment against Friedreich’s ataxia, which is a rare, degenerative, and inherited disease damaging the nervous system. Through this approved treatment, patients in the United States are consuming oral capsules as per the recommendation by healthcare professionals, which is a key Friedreich’s ataxia drug market trend.

Why is There Surging Demand for Friedreich Ataxia Drugs in China?

“Market Benefiting from Increased Research and Development Initiatives”

Attribute	China
Market Value (2024E)	US$ 66.2 Million
Growth Rate (2024 to 2034)	20.3% CAGR
Projected Value (2034F)	US$ 419.7 Million

China is evaluated to hold a share of 64.3% of the East Asia market by the end of 2034. Certain studies are reported about the clinical or genetic characteristics of Friedreich’s Ataxia patients, which is further calling for effective treatment options. Subsequently, lucrative opportunities are created for market players in the country.

Category-wise Insights

As reported in Fact.MR analysis, increased demand for Friedreich’s ataxia drugs in hospitals is attributed to their ability to offer effective treatment solutions with minimal chances of adverse health impacts with the presence of several effective healthcare providers.

Which Type of Therapy is Used More Against Friedreich’s Ataxia?

“Surging Use of Small Molecule Therapies for Treating Friedreich’s Ataxia”

Attribute	Small Molecule Therapy
Segment Value (2024E)	US$ 376.8 Million
Growth Rate (2024 to 2034)	13.5% CAGR
Projected Value (2034F)	US$ 1.34 Billion

Small molecule-based therapy is forecasted to occupy a share of 38.3% of the global market by 2034. There is only one approved treatment by the FDA against Friedreich’s ataxia, which is further opening new doors for small molecule therapy. This therapy aims to keep a check on the progress made in treating Friedreich’s ataxia. Certain agents, such as catalytic antioxidants, Nrf2 activators, and other novel methods are used in genetic modulation, thereby helping in more effective treatment, which is further creating a positive impact on Friedreich’s Ataxia drug market trends.

Which End User Will Generate High Revenue for Companies?

“Accelerating Demand for Friedreich’s Ataxia Drugs in Hospitals”

Attribute	Hospitals
Market Value (2024E)	US$ 455.9 Million
Growth Rate (2024 to 2034)	13.1% CAGR
Projected Value (2034F)	US$ 1.56 Billion

Hospitals are analyzed to capture 44.8% share of global market revenue by 2034-end. More patients of Friedreich’s ataxia are reaching hospitals for effective treatment although there are comparatively fewer chances of recovery. Certain treatment studies are conducted to offer effective treatment options in the future.

Currently, available treatments, such as physical therapy and surgery are aimed at keeping a check on patients at hospitals. Moreover, several hospitals are more equipped with advanced healthcare facilities and service providers, so in case of acute emergency, situations are controlled from getting worse.

Competitive Landscape

Key players in Friedreich’s ataxia drugs industry are offering more advanced and effective solutions for use in the healthcare sector. They are focusing on offering drugs for use against Friedreich’s ataxia matching set regulatory norms.

For instance:

• Design Therapeutics, Inc. is one of the popular biotechnology companies operating in the United States. In February 2022, the company announced the conduction of phase 1 of a clinical trial of drug DT-216, for its use in Friedreich’s ataxia treatment. As per the projections, the trial study was completed in December 2022.

Fact.MR provides detailed information about the price points of leading providers of Friedreich’s ataxia drugs positioned across the world, sales growth, production capacity, and speculative technological expansion, in this newly published market report.

Segmentation of Friedreich’s Ataxia Drug Market Research

• By Drug Class :

  • ACE Inhibitors
  • Beta Blockers
  • Diuretics
  • Nrf2 Activators
  • Para-Benzoquinone
  • Immunomodulators
  • Skeletal Muscle Relaxants
  • Anti-Epileptic Drugs
  • Others
    • Vitamin E
    • Other Supplements

• By Mechanism of Action :

  • Enzyme Activators
  • Enzyme Replacement
  • Enzyme Inhibitors
  • Protein & Peptide Activators
  • Protein & Peptide Inhibitors

• By Therapy :

  • Small Molecule
  • Gene Therapy
  • Gene-Modified Cell Therapy
  • Recombinant Protein
  • Cell Therapy

• By Route of Administration :

  • Oral
  • Injectable

• By Distribution Channel :

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

• By Patient Pool :

  • Pediatrics
  • Young Adults [10 to 15]
  • Adults & Seniors

• By Region :

  • North America
  • Latin America
  • Western Europe
  • Eastern Europe
  • East Asia
  • South Asia & Pacific
  • MEA