Friedreichs ataxia market

Friedreich’s Ataxia Market, by Drug Class (ACE Inhibitors, Beta Blockers, Diuretics, Para-Benzoquinone, and Others (Vitamin E, Immunomodulators, Skeletal Muscle Relaxants, and Anti-Epileptic Drugs, among others)), by Route of Administration (Oral and Injectable), by Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2030

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Jun 2022
CMI5034
165 Pages
Excel & Pdf
Pharmaceutical

Summary

Friedreich’s ataxia is caused by genetic mutations resulting in decline in production and expression of a mitochondrial protein called frataxin, which is involved in regulating iron-sulphur cluster enzymes within cells of the body. Insufficient frataxin levels result in the reduced activity of several mitochondrial enzymes involved in the electron transport chain, which leads to impaired mitochondrial metabolism such as the accumulation of iron in mitochondria dysfunction of adenosine triphosphate synthesis, reduced ATP (Adenosine triphosphate) production, and inefficient anti-oxidant response. As a consequence of mitochondrial dysfunction, neurons progressively die and Friedreich’s ataxia patients progressively lose their ability to coordinate movement and perform daily activities such as walking, running, etc. effectively. Thus, therapeutic strategies for the management or treatment of Friedreich’s ataxia patients aim at correcting some of the downstream consequences of frataxin deficiency or at restoring sufficient frataxin levels in the cell.

However, an adequate cure for Friedreich’s ataxia is not yet available for patients. Some of the pharmacology therapy approaches used for the management or treatment of Friedreich’s ataxia patients include usage of drugs such as angiotensin-converting enzyme (ACE) inhibitors, beta blockers, diuretics, para-benzoquinone, immunomodulators, skeletal muscle relaxants, anti-epileptic drugs, nuclear factor-erythroid factor 2-related factor 2 (Nrf2) inducer, histone deacetylase (HDAC) inhibitors, deuterated polyunsaturated fatty acids, trans-activators of transcription (TAT)-frataxin, iron chelators, etc. Future therapeutics of Friedreich’s ataxia involve advanced gene therapy to add a correct copy of the gene responsible for the production of frataxin protein in the body.

The global Friedreich’s ataxia market is estimated to be valued at US$ 777.2 million in 2022 and is expected to exhibit a CAGR of 13.0% over the forecast period (2022-2030).

Figure 1. Global Friedreich’s Ataxia Market Share (%), By Region, 2022

High burden of Friedreich’s ataxia is expected to drive growth of the global Friedreich’s ataxia market over the forecast period

For instance, according to data published by the Ataxia UK, a charity registered in Scotland and England & Wales in November 2020, it was estimated that the incidence of Friedreich’s ataxia based on carrier frequency of 1 in 85 was 1:29,000 in Europe in 2020.

Friedreich’s Ataxia Market Report Coverage

Report Coverage	Details
Base Year:	2021	Market Size in 2022:	US$ 777.2 Mn
Historical Data for:	2017 to 2020	Forecast Period:	2022 to 2030
Forecast Period 2022 to 2030 CAGR:	13.0%	2030 Value Projection:	US$ 2,062.9 Mn
Geographies covered:	North America: U.S. and Canada Latin America: Brazil, Argentina, Mexico, and Rest of Latin America Europe: Germany, U.K., Spain, France, Italy, Russia, and Rest of Europe Asia Pacific: China, India, Japan, Australia, South Korea, ASEAN, and Rest of Asia Pacific Middle East: GCC Countries, Israel, and Rest of Middle East Africa: South Africa, North Africa, and Central Africa
Segments covered:	By Drug Class: ACE Inhibitors, Beta Blockers, Diuretics, Para-Benzoquinone, Others (Vitamin E, Immunomodulators, Skeletal Muscle Relaxants, and Anti-Epileptic Drugs, among others) By Route of Administration: Oral, Injectable By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Online Pharmacies
Companies covered:	Reata Pharmaceuticals, Inc., Retrotope Inc., Minoryx, PTC Therapeutics, Design Therapeutics, Inc., Larimar Therapeutics, Inc., Jupiter Neurosciences, Inc., Lexeo Therapeutics, Zydus Lifesciences Ltd., Cipla Limited, GlaxoSmithKline Plc., Aurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., and Intas Pharmaceuticals Ltd.
Growth Drivers:	Rapid research and development activities for development of novel therapeutics for Friedreich's ataxia treatment Increasing inorganic business growth strategies such as collaborations, acquisitions, and others among market players
Restraints & Challenges:	Challenges faced during designing and conducting clinical trials for development of Freidreich's ataxia treatment

Figure 2. Global Friedreich’s Ataxia Market Value (US$ Mn), By Route of Administration, 2022

Increasing research and development activities for introduction of novel Friedreich’s ataxia therapeutics is expected to drive the market growth over the forecast period.

For instance, according to an article published in the Neurobiology of Disease journal, in January 2021, an oral drug called leriglitazone had shown benefits in treating Friedrich's ataxia in cellular and animal models. Leriglitazone is manufactured by Minoryx Therapeutics, a Spain-based clinical-stage biotechnology company, and is currently in advanced Phase 2/3 of clinical trials for the treatment of Friedrich's ataxia. Leriglitazone restores mitochondrial function which is generally hampered due to the absence of mitochondrial protein frataxin (FXN) in Friedreich's ataxia disease.

Furthermore, in February 2022, Design Therapeutics, Inc., a U.S.-based biotechnology company, announced that it is currently conducting Phase 1 clinical trial of its investigational drug DT-216, an intravenous (IV) injection for the treatment of Friedreich's Ataxia and this clinical trial study is expected to be completed by December 2022.

Key players operating in market are focusing on obtaining regulatory approval of their Friedreich’s ataxia drugs, which is expected to drive growth of the global Friedreich’s ataxia market over the forecast period.

In March 2022, Stealth BioTherapeutics Inc., a Cayman Islands-based biotechnology company, announced that it received Orphan Drug Designation from the U.S. Food and Drug Administration (US FDA) Office of Orphan Products Development for its drug Elamipretide, which is indicated for the treatment of Friedreich's ataxia.

Global Friedreich’s Ataxia Market – Impact of Coronavirus (Covid-19) Pandemic

SARS-CoV-2 (Severe acute respiratory syndrome coronavirus) was first reported from Wuhan, China in December 2019, and was declared as a global pandemic by the World Health Organization (WHO), which designates this SARS-CoV-2 infection as novel Coronavirus (COVID-19). According to the Coronavirus (COVID-19), Weekly Epidemiological Update by the World Health Organization, COVID-19 has spread across the globe infecting over 519 million population leading to more than 6.3 million deaths as of May 15, 2022. The negative impact of COVID-19 on the healthcare domain is huge and it has significantly disrupted the healthcare industries’ entire supply chain, from raw materials to manufacturing and delivery. Similarly, the COVID-19 pandemic had a negative impact on the overall growth of the Friedreich’s ataxia market, owing to movement restrictions and lockdowns.

Similarly, demand and supply for Friedreich’s ataxia therapeutics was disrupted, owing to halt of clinical trials aimed at developing treatment drugs for Freidreich’s ataxia, as well as disrupted supply of Friedreich’s ataxia treatment drugs during the lockdown. Moreover, physiotherapy, rehabilitation programs, physical training, etc. for management of Friedreich’s ataxia was at halt during the lockdown.

For instance, according to an electronic survey conducted amongst Friedreich ataxia (FA) patients in Italy and published in the Cerebellum & Ataxias journal, an open access scientific journal in January 2021, it was found that the number of patients undergoing physiotherapy was reduced by 72.2% due to the COVID-19 lockdown. According to the same source, over 60% of Friedreich ataxia patients reported worsening of their health in absence of physiotherapy during COVID-19 lockdown.

Global Friedreich’s Ataxia Market - Restraint

Challenges faced during designing and conducting clinical trials for development of Freidreich's ataxia treatment are the factors that are expected to hinder growth of the global Friedreich’s ataxia market over the forecast period.

Designing clinical trials for rare neurodegenerative diseases such as Friedreich's ataxia is tricky due to the insufficient number of Friedreich's ataxia patients, heterogeneous nature of the disease with a slow and variable progression rate, which makes it difficult to identify reliable outcome measures to monitor the progression of Friedreich's ataxia.

For instance, according to an article published by the Orphanet Journal of Rare Diseases, in August 2020, Friedreich's ataxia is a genetic, neurodegenerative movement disorder, with a typical age of onset (the age at which the symptoms of a disease first appear) between 10 and 15 years. Thus, the diagnosis of the disease takes a long time since its initial stage. The same source also estimated that the average time taken for the diagnosis of rare disorders such as Friedreich's ataxia is 5 years.

Key Players

Major players operating in the global Friedreich’s ataxia market include Reata Pharmaceuticals, Inc., Retrotope Inc., Minoryx, PTC Therapeutics, Design Therapeutics, Inc., Larimar Therapeutics, Inc., Jupiter Neurosciences, Inc., Lexeo Therapeutics, Zydus Lifesciences Ltd., Cipla Limited, GlaxoSmithKline Plc., Aurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., and Intas Pharmaceuticals Ltd.

Table of Content

Table of Contents

*Browse 49 market data tables and 35 figures on "Global Friedreich’s Ataxia Market” - forecast to 2030

Segmentation

Detailed Segmentation:

- Frequently Asked Questions -

What is the current market size and compound annual growth rate (CAGR) of the global Friedreich’s ataxia market during the forecast period (2022-2030)?

Global Friedreich’s ataxia market size is estimated to be valued at US$ 777.2 million in 2022 and is expected to exhibit a CAGR of 13.0% between 2022 and 2030.

What are the major factors driving the market growth?

High burden of Friedreich's ataxia and rapid research and development activities for development of novel therapeutics for Friedreich's ataxia treatment are expected to drive the market growth over the forecast period.

Which is the leading route of administration segment in the market?

Oral segment is expected to hold the major market share in the market.

Which region holds the largest market share in the market?

North America holds the largest market share in the market.

What are the key factors hampering growth of the market?

Major factors hampering growth of the market include challenges faced during designing and conducting clinical trials for development of Freidreich's ataxia treatment

Which are the major players operating in the market?

Major players operating in the market include Reata Pharmaceuticals, Inc., Retrotope Inc., Minoryx, PTC Therapeutics, Design Therapeutics, Inc., Larimar Therapeutics, Inc., Jupiter Neurosciences, Inc., Lexeo Therapeutics, Zydus Lifesciences Ltd., Cipla Limited, GlaxoSmithKline Plc., Aurobindo Pharma Ltd., Sun Pharmaceutical Industries Ltd., Torrent Pharmaceuticals Ltd., and Intas Pharmaceuticals Ltd.