Frataxin, a mitochondrial protein required for controlling iron-sulfur cluster enzymes in body cells, is one of the genetic abnormalities that results in Friedreich’s ataxia. Frataxin deficiency reduces the activity of various mitochondrial enzymes involved in the electron transport chain, affecting adenosine triphosphate synthesis, lowering ATP (adenosine triphosphate) generation, and limiting the ability of the mitochondria to react to free radicals. People with Friedreich’s ataxia gradually lose the ability to coordinate their movements and do basic daily functions like walking, jogging, etc. because of mitochondrial dysfunction, which causes neurons to die. Therefore, therapeutic approaches for the management or treatment of Friedreich’s ataxia patients attempt to reverse some of the aftereffects of frataxin deficiency or to restore adequate levels of frataxin in levels in the cell.
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Friedreich’s ataxia does not yet have a suitable treatment option for sufferers. Angiotensin-converting enzyme (ACE) inhibitors, deuterated polyunsaturated fatty acids, beta-blockers, histone deacetylase (HDAC) inhibitors, diuretics, para-benzoquinone, nuclear factor-erythroid factor 2-related factor 2 (Nrf2) inducers, immunomodulators, anti-epileptic medications, skeletal muscle relaxants, and trans-activators of t-cell receptors are some of the medications used in the future, enhanced gene therapy will be used to add a proper copy of the gene in charge of the body’s creation of the frataxin protein as a treatment for Friedreich’s ataxia.
The market for medications to treat Friedreich’s ataxia is anticipated to increase at a CAGR of 13.0% over the forecast period, reaching US$ 777.2 million in 2022. (2022-2030).
The high incidence of Friedreich’s ataxia is expected to fuel market expansion for the duration of the forecast period.
For instance, based on information provided by the Ataxia UK, a charity established in Scotland, England, and Wales in November 2020, it was predicted that the incidence of Friedreich’s ataxia throughout Europe would be 1:29,000 in 2020.
Increased research and development efforts for the introduction of cutting-edge Friedreich’s ataxia medications are predicted to fuel market expansion throughout the forecasted period.
For instance, troglitazone, an oral medication, was shown to be beneficial in treating Friedrich’s ataxia in cellular and animal models, according to a research published in the Neurobiology of Disease journal in January 2021. Minoryx Therapeutics, a clinical-stage biotechnology business with offices in Spain, is conducting advanced Phase 2/3 clinical research employing leriglitazone to treat Friedrich’s ataxia. Due to the lack of the mitochondrial protein frataxin, which is typically weakened in Friedreich’s ataxia, leriglitazone boosts mitochondrial function (FXN).
Additionally, a U.S.-based biotechnology company called Design Therapeutics, Inc. announced in February 2022 that it is presently running a Phase 1 clinical trial for its investigational drug DT-216, an intravenous (IV) injection for the treatment of Friedreich’s ataxia, and that the study is anticipated to be finished by December 2022.
The market’s major competitors are concentrating on winning regulatory clearance for their treatments for Friedreich’s ataxia, which is anticipated to propel the market’s expansion throughout the projected period.
The Cayman Islands-based biotechnology firm Stealth BioTherapeutics Inc. announced in March 2022 that their medicine Elamipretide has been designated as an orphan by the US Food and Drug Administration (US FDA) Office of Orphan Products Development.
The World Health Organization (WHO) proclaimed SARS-CoV-2 (Severe Acute Respiratory Syndrome Coronavirus) to be a worldwide pandemic after it was first identified in Wuhan, China, in December 2019. The WHO refers to this SARS-CoV-2 infection as a new Coronavirus (COVID-19). According to the World Health Organization’s Weekly Epidemiological Update on Coronavirus (COVID-19), COVID-19 has infected more than 519 million people worldwide and will cause more than 6.3 million fatalities by May 15, 2022. The COVID-19 outbreak has had a severe negative impact on the healthcare industry and has seriously impacted the whole supply chain, from raw materials to manufacture and distribution. Similar to COVID-19, the lockdowns and limitations brought on by the pandemic hindered the overall growth of the Friedreich’s ataxia market.
Due to the suspension of clinical studies to find treatments for Friedreich’s ataxia and the disrupted supply of Friedreich’s ataxia treatment medications during the lockdown, demand and supply for therapies for the condition were affected. Additionally, during the lockdown, all physical therapy, rehabilitation programmes, physical training, etc. for the management of Friedreich’s ataxia came to an end.
According to an electronic survey of Italian Friedreich ataxia (FA) patients that was published in the open access Cerebellum & Ataxias journal in January 2021, the COVID-19 lockout resulted in a 72.2% decrease in the number of patients receiving physiotherapy. Over 60% of Friedreich ataxia sufferers reportedly reported worsening health conditions due to lack of physiotherapy during the COVID-19 lockdown, according to the same source.
The factors that are anticipated to restrain the growth of the global Friedreich’s ataxia market over the forecast period include difficulties encountered during the design and execution of clinical studies for the development of Friedreich’s ataxia treatments.
Due to the limited patient population, heterogeneous nature of the disease, and slow and variable rate of progression, it is challenging to develop clinical trial designs for rare neurodegenerative diseases like Friedreich’s ataxia. These factors make it challenging to choose appropriate outcome measures to track the progression of Friedreich’s ataxia.
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For instance, Friedreich’s ataxia is a hereditary, neurological movement condition with a typical age of start (the age at which the disease’s symptoms first manifest) between 10 and 15 years, according to an article published by the Orphanet Journal of Rare Diseases in August 2020. As a result, it takes a while for the condition to be diagnosed once it has manifested. The same source also predicted that it takes 5 years on average to diagnose uncommon diseases like Friedreich’s ataxia.
Intas Pharmaceuticals Ltd., Reata Pharmaceuticals, Inc., Torrent Pharmaceuticals Ltd., Retrotope Inc., Sun Pharmaceutical Industries Ltd., Minoryx, Aurobindo Pharma Ltd., PTC Therapeutics, GlaxoSmithKline Plc., Design Therapeutics, Cipla Limited, Larimar Therapeutics, Zydus Lifesciences Ltd., Jupiter Neurosciences, Inc., and Lexeo Therapeutics, are significant market participants in the global Friedreich’s ataxia market.
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Reasons to buy this Friedreich’s Ataxia Market Report
☑ It attempts an analysis of the competing scenario.
☑ The current and exceptional product revenue market.
☑ An in-depth data on the regional investigation and competitive landscape structure.
☑ It benefits in creating an awareness of the important key product segments.
☑ The marketing strategies, opportunities, and development factors are explained.
☑ Friedreich’s Ataxia market size estimation and recent advancements in the industry are explained.
The Study Objectives are:
✔ A comprehensive insight into key players operating in the Friedreich’s Ataxia Market and their corresponding data.
✔ It includes product portfolio, annual revenue, expenditure on research and development, geographical presence, key developments in recent years, and growth strategies.
✔ Regional analysis, which includes insight into the dominant market and corresponding market share.
✔ It also includes various socio-economic factors affecting the evolution of the market in the region.
✔ The report offers a comprehensive insight into different individuals from value chains such as raw materials suppliers, distributors, and stockholders.
Table of Content
Chapter 1 Industry Overview
1.3 Research Scope
1.4 Market Analysis by Regions
1.5 Friedreich’s Ataxia Market Size Analysis from 2022 to 2028
11.6 COVID-19 Outbreak: Friedreich’s Ataxia Industry Impact
Chapter 2 Global Friedreich’s Ataxia Competition by Types, Applications, and Top Regions and Countries
2.1 Global Friedreich’s Ataxia (Volume and Value) by Type
2.3 Global Friedreich’s Ataxia (Volume and Value) by Regions
Chapter 3 Production Market Analysis
3.1 Global Production Market Analysis
3.2 Regional Production Market Analysis
Chapter 4 Global Friedreich’s Ataxia Sales, Consumption, Export, Import by Regions (2017-2022)
Chapter 5 North America Friedreich’s Ataxia Market Analysis
Chapter 6 East Asia Friedreich’s Ataxia Market Analysis
Chapter 7 Europe Friedreich’s Ataxia Market Analysis
Chapter 8 South Asia Friedreich’s Ataxia Market Analysis
Chapter 9 Southeast Asia Friedreich’s Ataxia Market Analysis
Chapter 10 Middle East Friedreich’s Ataxia Market Analysis
Chapter 11 Africa Friedreich’s Ataxia Market Analysis
Chapter 12 Oceania Friedreich’s Ataxia Market Analysis
Chapter 13 South America Friedreich’s Ataxia Market Analysis
Chapter 14 Company Profiles and Key Figures in Friedreich’s Ataxia Business
Chapter 15 Global Friedreich’s Ataxia Market Forecast (2022-2028)
Chapter 16 Conclusions
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