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.
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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.
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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.
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