Pompe Disease Therapeutic Market Key Players, Competitive Status and Trend, The Technology Trends in Future to 2026
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
government initiatives is expected to augment growth of the pompe
disease therapeutic market
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. 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.
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.
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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Disease Therapeutic Market
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