Increasing government initiatives
is expected to augment growth of the pompe disease therapeutic market
Furthermore, initiatives from the
governments of economies such as Sweden, the Netherlands, and South Korea that
focus on approving guidelines for the reimbursement of individuals undergoing
enzyme replacement therapy (ERT) are also expected to boost growth of the Pompe
Disease Therapeutic Market in near future. Considering the public health
problems, government is taking continuous initiatives in promoting and
advancing the development of innovative products for the prevention and
diagnosis or treatment of rare diseases or conditions such as Pompe disease.
For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards
from 92 grant applications and provided funding and support to around 67
ongoing clinical study projects related to rare disease.
Pompe disease is an inherited
Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid named
alpha-glucosidase (GAA). This causes the glycogen to accumulate in different
kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac
muscle. It leads to muscle weakness, respiratory problems, and may also affects
the liver and heart. Pompe disease is inherited as an autosomal recessive
genetic trait. There are three major types of pompe disease, which differ in
severity and appear at different age, these types are known as classic
infantile-onset, which starts within a few months of birth, non-classic
infantile-onset Pompe disease- usually appears by age 1, and late-onset type of
Pompe disease that may shows the symptoms later in childhood, adolescence, or
adulthood. Pompe disease therapeutic market can exhibit immense growth
potential due to presence of limited number of manufacturers, which can
significantly enhance and expand their business potential and scope in this
market.
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Increasing prevalence of pompe
disease in many economies is expected to have a lucrative opportunities for
pharmaceutical manufacturers to come with a tailored solution for diagnosis and
treatment of disease. For instance, according to United Pompe Foundation (UPF),
2017, the estimated frequency occurrence of Pompe disease may vary among
different ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1
in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000
births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000
suffer from pompe disease. Gene therapy and enzyme replacement therapy may be
highly effective at reducing clinical manifestations of this rare disease.
Currently, there are two commercialized products in enzyme replacement therapy,
which are Myozyme for the treatment of infants and children with Pompe disease
and Lumizyme for late-onset non-infantile Pompe disease. Furthermore, various
pipeline products in phase 3 (NeoGAA GZ402666 (Sanofi), AT-982 (Audentes
therapeutics)) and in phase 2 (BMN 701(Biomarin)) clinical trial are also
expected to create a meteoric growth in this market during the forecast period.
However, the high cost associated with the treatment and long duration of the
treatment are the factors restraining growth of the pompe disease therapeutic
market. For instance, according to a research paper published by Harvard
School, in 2016, Lumizyme accounted for US$ 100,000 per year for children and
US$ 300,000 per year for adult treatments.
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Report: https://www.coherentmarketinsights.com/ongoing-insight/pompe-disease-therapeutic-market-1316
Increasing investments in
research and development by manufacturers is expected to propel the market
growth
Manufacturers are focusing on
finding new solutions by expanding their pipeline to cater the demand for pompe
disease therapeutics market. For instance, in September, 2017, Avrobio Inc.
expanded their products pipeline with gene therapy to treat pompe disease by
utilizing a proprietary lysosomal targeting sequence to deliver high levels of
enzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics received
orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of
Pompe disease. This novel treatment paradigm consists of ATB200, a unique
recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized
carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance
uptake, co-administered with AT2221, a pharmacological chaperone.
Key players operating in the
pompe disease therapeutic market include Amicus Therapeutics, Inc., Valerion
Therapeutics, LLC, Sanofi S.A., Oxyrane, and Sangamo Therapeutics, Inc.
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