Mitochondrial-based Therapeutics Market Size & Share, by Drug Type (Anti-Inflammatory Biologics, Non-Steroidal Anti-inflammatory Drugs, Corticosteroids); Indication (Mitochondrial Myopathy, Lebers heredity Leigh Syndrome, Mitochondrial DNA Depletion Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis, Myoclonic Epilepsy with Ragged Red Fibres): Route of Administration (Oral, Intravenous) - Global Supply & Demand Analysis, Growth Forecasts, Statistics Report 2024-2036

  • Report ID: 5876
  • Published Date: Mar 11, 2024
  • Report Format: PDF, PPT

Global Market Size, Forecast, and Trend Highlights Over 2024-2036

Mitochondrial-based Therapeutics Market size is expected to reach USD 3 Billion by the end of 2036, growing at a CAGR of 8% during the forecast period i.e., 2024-2036. In the year 2023, the industry size of mitochondrial-based therapeutic was over USD 500 Million. The dynamics of the market for mitochondrion-based medicines are being profoundly and multifacetedly influenced by a global demographic transition towards aging populations. The increasing incidence of age-related diseases, many of which are intricately linked to mitochondrial dysfunction, forming a central link that increases the urgency and potential of interventions directed at mitochondria, is a key consequence. The number of people over 60 in the world will rise to 1 in 6 between now and 2030. The proportion of the population aged 60 and over will increase from 1 billion in 2020 to 1.4 billion at this stage. The world's population of people 60 years and over is doubling by 2050.

Due to a number of genetic backgrounds and environmental factors, mitochondrial dysfunction is characterized by significant diversity in individuals. This diversity has led to the pursuit of carefully designed interventions that can address individual and distinctive mitochondrial deficiencies that arise in each patient. Sophisticated analyses of the individual mitochondria's DNA and functions have been facilitated by developments in technology, like Next Generation Sequencing. The advent of this technology facilitates the identification of accurate mitochondrial irregularity. Thus, healthcare professionals are in a position to devise tailored treatment strategies that address the root causes of mitochondrial dysfunction with increased precision.

Mitochondrial-based Therapeutics Market Overview
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Mitochondrial-based Therapeutics Market: Growth Drivers and Challenges

Growth Drivers

  • Growing Support by Market Players - Significant resources are being allocated to the development of mitochondrial-based therapeutics market by clinical-stage companies. For example, Alexion Pharmaceuticals and Stealth Biotherapeutics Corp. announced a collaboration in October 2019 to codevelop and market Elamipretide, a medicine for the treatment of mitochondrial dysfunction.
  • Prevalence of Mitochondrial Disorder - The demand for Mitochondrial Based Therapeutic Products is anticipated to increase with the increased prevalence of mitochondria disorders. The growth of the mitochondrial-based therapeutics market is anticipated to be driven by an increasing number of Joint Research and Development Activities between leading Biopharmaceutical Innovative Players. For example, in June 2020, the US Food and Drug Administration granted Reneo Pharmaceutical's lead drug candidate, RENOO1, an orphan drug for the treatment of primary mitochondrial myopathy.


  • Complexities in Mitochondrial Disease-Mitochondrial diseases are a diverse group of disorders with varying genetic and biochemical abnormalities. This complexity makes it difficult to develop one-size-fits-all treatments.
  • Lack of awareness is expected to pose limitation on the mitochondrial-based therapeutics market growth in the projected period
  • Strict regulations associated are set to hamper the market growth in the upcoming period.

Mitochondrial-based Therapeutics Market: Key Insights

Base Year


Forecast Year

2023 - 2036



Base Year Market Size

~USD 500 million

Forecast Year Market Size

~USD 3 billion

Regional Scope

  • North America (U.S., and Canada)
  • Latin America (Mexico, Argentina, Rest of Latin America)
  • Asia-Pacific (Japan, China, India, Indonesia, Malaysia, Australia, Rest of Asia-Pacific)
  • Europe (U.K., Germany, France, Italy, Spain, Russia, NORDIC, Rest of Europe)
  • Middle East and Africa (Israel, GCC North Africa, South Africa, Rest of the Middle East and Africa)
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Mitochondrial-based Therapeutics Segmentation

Indication (Mitochondrial Myopathy, Leber’s heredity Leigh Syndrome, Mitochondrial DNA Depletion Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis, Myoclonic Epilepsy with Ragged Red Fibres)

In terms of indication, the mitochondrial myopathy segment predicted to account for 20% share of the global mitochondrial-based therapeutics market by the end of 2036. The severity of the disease and the extent of the various organs affected ultimately determine the prognosis for people with mitochondrial myopathy, which can vary widely. Carnitine and arginine are the most commonly used medicinal products. A combination of 3-6 medicines taken by a doctor is used in the cocktail therapy. Increased cases of mitochondrial myopathies, continued clinical trials, more government initiatives, the ease of purchasing, research and development investments by pharmaceutical companies, and the availability of healthcare policies are expected to increase the market. The clinical trial for niacin has now been completed.

Route of Administration (Oral, Intravenous)

Based on route of administration, the oral segment in mitochondrial-based therapeutics market is anticipated to hold the largest revenue share of 60% during the forecast period. Their importance is driven by patients' preference for the convenience and nonintrusive nature of orally administered medicines, as well as a general acceptance and widespread use of existing formulations. This dominance is further strengthened by the ease of compliance, drug stability in oral form, and extensive experience from pharmaceutical companies that have developed oral formulations. They are more readily available to a wide range of patients because of regulatory approval pathways, dosing flexibility and the cost effectiveness of orally administered medicines. Consequently, a preferred and well-proven method of delivery of Mitochondrial Targeted Therapies is the orally administered route.

Our in-depth analysis of the global mitochondrial-based therapeutics market includes the following segments:

          Drug Type

  • Anti-Inflammatory Biologics
  • Non-Steroidal Anti-inflammatory Drugs
  •  Corticosteroids


  • Mitochondrial Myopathy
  • Leber’s heredity Leigh Syndrome
  • Mitochondrial DNA Depletion Syndrome
  •  Mitochondrial Encephalomyopathy
  •  Lactic Acidosis
  •  Myoclonic Epilepsy with Ragged Red Fibres

          Route of Administration

  • Oral
  • Intravenous

          Distribution Channel

  • Hospital Pharmacies
  •  Retail Pharmacies
  •  Drugstores
  • Online Pharmacies

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Mitochondrial-based Therapeutics Industry - Regional Synopsis

North American Market Forecasts

Mitochondrial-based Therapeutics Market in North America region is set to hold the largest revenue share of 35% during the projected time period. It is due to its sophisticated healthcare infrastructure, significant investments in R&D, and the presence of major companies with considerable resources that growth can be attributed. The United States has an overall score of 54.96 on the World Health Innovation Index, placing it in 4th place. The region also benefits from strong regulatory support, in particular from agencies such as the FDA and Health Canada, which promotes a favourable environment for approval and commercialization of innovative medicinal products. North America's importance is also enhanced by a high number of clinical trials, an increasing incidence of metabolic disorders and access to adequate funding. Moreover, continuous innovation in mitochondrial based treatments is driven by the region's leadership of the global pharmaceutical industry coupled with intense market competition and collaboration.

European Market Statistics

Mitochondrial-based Therapeutics Market in Europe region is set to grow significantly during the forecast timeframe. Advanced healthcare infrastructure, world-class research institutions, and a strong pharmaceutical sector can be attributed to the growth in the region. Europe is at the forefront of mitochondrial therapy developments, thanks to an active legal environment and strategic cooperation between academia and industry. Innovation and the development of efficient treatments are driven by the region's commitment to clinical trials as well as a large incidence of mitochondrial disorders.

Research Nester
Mitochondrial Based Therapeutic Market size
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Companies Dominating the Mitochondrial-Based Therapeutics Landscape

    • Novartis AG
      • Company Overview
      • Business Strategy
      • Key Product Offerings
      • Financial Performance
      • Key Performance Indicators
      • Risk Analysis
      • Recent Development
      • Regional Presence
      • SWOT Analysis
    • Pfizer Inc.
    • Amgen Inc.
    • Takeda Pharmaceuticals
    • NeuroVive Pharmaceutical AB
    • Reata Pharmaceuticals
    • Entogene AG
    • Intercept Pharmaceutics
    • Stealth Biotherapeutics
    • Ixchel Pharma


In The News

  • Stealth BioTherapeutics Inc., a clinical stage biotechnology company focused on the discovery, development and commercialization of novel treatments for diseases caused by mitochondrial dysfunction, announced in September 2023 that it had achieved its target to recruit patients with primary myopathy into the NuPOWER study.
  • a collaborative agreement was signed by Primera Therapeutics, the inaugural launch from the cell and gene therapy accelerator Mayflower BioVentures, with Cellectis. The two companies shall cooperate to develop methods for gene editing of mutations in mitochondrial DNA, which can be used as an in vivo therapy against mitochondria disease.

Author Credits:  Radhika Gupta, Shivam Bhutani

  • Report ID: 5876
  • Published Date: Mar 11, 2024
  • Report Format: PDF, PPT

Frequently Asked Questions (FAQ)

The growing prevalence of mitochondria disease and increasing demand for personalized medication are some of the major factors anticipated to drive the growth of the mitochondrial-based therapeutics market.

The market is anticipated to attain a CAGR of ~8% over the forecast period, i.e., 2024-2036.

The major players in the market are of NeuroVive Pharmaceutical AB, Reata Pharmaceuticals, Entogene AG, Intercept Pharmaceutics, Stealth Biotherapeutics, Ixchel Pharma, and others.

The oral segment is anticipated to garner the largest market size by the end of 2036 and display significant growth opportunities.

The market in North America is projected to hold the largest market share by the end of 2036 and provide more business opportunities in the future.
Mitochondrial-based Therapeutics Market Report Scope

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