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Mitochondrial disorders are inherited chronic disorders characterized by a wide range of biochemical and genetic mitochondrial defects and mutations. The disorder affects multiple organs, which includes brain, heart, liver, skeletal muscles, kidney, and respiratory systems. The patients with mitochondrial disorders often suffer from muscle weakness, exercise intolerance, and fatigue due to reduction in mitochondrial respiration, which is caused due to mitochondrial DNA mutations leading to reduced ability to produce cellular adenosine triphosphate (ATP). Despite major advances in understanding of the molecular mechanisms, currently there are no effective treatments for the disease. The treatment of mitochondrial disorders is mostly performed with different vitamins, co-factors, off-label drugs approved for other indications, and nutritional supplements. However, the research organizations and pharmaceutical companies are involved in development of novel drugs with technology advancements including the use of biomarkers, replacement therapies and sophisticated trial designs, thus creating a lucrative opportunity for the market growth.

Mitochondrial Disorders Treatment Market: Market Dynamics

Increasing prevalence of mitochondrial disorders causing significant morbidity and mortality combined with the higher potential of genetic transmission to the next generation are the factors driving the market growth. According to the Wellcome Center for Mitochondrial Research, 2015, about 10 people per 100,000 have a mitochondrial disorders, which often express first in early childhood. According to the Genetic and Rare Disease Information Center, women with mitochondrial genetic disorders have 50% chance with each pregnancy of passing along the altered gene to the offspring, thus indicating the rising burden of the disease. Also, the mitochondrial disorders treatment market is expected to gain significant traction in the future, as the pharmaceutical drugs approved for the treatment of primary mitochondrial disease have orphan drug status, thereby having a greater potential for market approval than conventional pharmaceuticals. Moreover, the approval of novel drug molecules from the regulatory agencies is expected to create a lucrative opportunity for the growth of mitochondrial disorders treatment market. For instance, in April 2018, NeuroVive Pharmaceutical AB received orphan drug designation to KL1333 by the US Food and Drug administration (FDA) for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

Also, the presence of various clinical trials undertaken by various research institutes to address the unmet need of the patients suffering with the mitochondrial disorders is expected to augment market growth. For instance, in April, 2018, GenSight Biologics, a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced results from the REVERSE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in for treating a mitochondrial disease- Leber Hereditary Optic Neuropathy (LHON).

Higher R&D investments by the organizations supporting the researchers for the development of efficient therapies to address the unmet medical needs of the patients is expected to upsurge the growth of mitochondrial disorders treatment market in the near future. For instance, the Foundation for Mitochondrial Medicine (FMM) supports and accelerates the development for most promising mitochondrial research and treatments to treat various forms of mitochondrial disease and disorders by funding targeted therapeutic discovery project. In 2014, (FMM) collaborated with the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support a research project at the Department of Neuroscience at Mayo Clinic for investigating a mitochondria-targeted therapeutic approach to treating Parkinson’s disease. Also, the United Mitochondria Disease Foundation (UMDF) along with the National Institute of Child Health and Human Development (NICHD) launched the Mitochondrial Disease Sequence Data Resource (MSeqDR) Consortium. The goals of this consortium is to facilitate deposition, curation, annotation and integrated analysis of genomic data for mitochondrial diseases for clinical and research communities.

However, higher R&D cost, stringent regulations for the approval of novel therapies, lack of awareness and understanding of the disease are the factors restraining the market growth.

Mitochondrial Disorders Treatment Market: Regional Dynamics

Regional segmentation of the global mitochondrial disorders treatment market by Coherent Market Insights comprises North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America is expected to hold a dominant position in the global mitochondrial disorders treatment market over the forecast period, owing to increasing prevalence of the disease and the rising number of initiatives to spread awareness and support novel drug development. According to the Institute of Medicine, U.S., 2015, an estimated 30,000 Americans are suffering from inherited defects in their mitochondria. Also, according to the Foundation for Mitochondrial Medicine, an estimated 1,000 children per year are born with some form of mitochondrial disease in the U.S. Moreover, the North American Mitochondrial Disease Consortium (NAMDC), a part of Rare Disease Clinical Research Network (RDCRN), specially works towards collecting information from mitochondrial disease patients in a clinical patient registry, thereby helping researchers to identify and recruit patients for future studies.

Mitochondrial Disorders Treatment Market: Competitive Landscape

The key players operating in the market are adopting strategic collaborations with research organizations to advance drugs for rare diseases through clinical development. For instance, in February 2018, NeuroVive Pharmaceutical AB in collaboration with Karolinska Institute developed a compound NV556 to treat patients with mitochondrial myopathy. The company is expected to initiate the clinical trials for the compound with the effective outcomes from its preclinical study.

Key players operating in the global mitochondrial disorders treatment market include Centogene AG, GeneDx, Ixchel Pharma, Khondrion BV, Mitobridge, NeuroVive Pharmaceutical AB, Reata Pharmaceuticals Inc., and Stealth Biotherapeutics.

Mitochondrial Disorders Treatment Market: Market Taxonomy

On the basis of therapy type, the global mitochondrial disorders treatment market is segmented into:

  • Dietary Therapy
  • Vitamin and Supplement Therapy
  • Pipeline Therapies
    • NV556
    • KL1333
    • Others

On the basis of distribution channel, the global mitochondrial disorders treatment market is segmented into:

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

On the basis of geography, the global mitochondrial disorders treatment market is segmented into:

  • North America
  • Latin America
  • Europe
  • Asia Pacific
  • Middle East
  • Africa

Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.

Data Triangulation Methodology | Coherent Market Insights

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.

Market Analysis | Coherent Market Insights

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users

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