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).
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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