Mitochondrial Disorders Treatment Market Development Analysis Contributing Top Vendor Landscape and Economic Growth 2028
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.
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