A recent report published by
Infinium Global Research on pompe disease market provides in-depth analysis of
segments and sub-segments in the global as well as regional pompe disease
market. The study also highlights the impact of drivers, restraints, and macro
indicators on the global and regional pompe disease market over the short term
as well as long term. The report is a comprehensive presentation of trends,
forecast and dollar values of the global pompe disease market. According to the
report, the global pompe disease market is projected to grow at a CAGR of over
4% over the forecast period of 2022-2028.
The global pompe disease market was
valued at about USD 730 Million in 2022 and is expected to reach nearly USD 950
Million in 2028, with a CAGR of over 4% during the forecast period. Pompe
disease is an autosomal recessive metabolic disorder that damages muscle and
nerve cells throughout the body. It is caused by an accumulation of glycogen in
the lysosome due to a deficiency of the lysosomal acid alpha-glucosidase
enzyme. These diseases approximately affect one individual in 40,000 in the
U.S, however, the incidence varies among different ethnic groups. Pompe disease
can be treated in symptomatic, supportive, and disease-specific ways.
Increasing developments in gene therapy and enzyme replacement therapies are
the primary factors driving the growth of the pompe disease market. Moreover,
inflated costs associated with pompe disease therapy could hinder the market
growth opportunities.
The growth of the market is
basically attributed to the launch of novel drugs and therefore the continued
uptake of long-term therapies. Pompe disease is a rare disease classified as
early infantile or late-onset majorly affecting musculus, heart defects, and
breathing problems. As per the study by the National Institute of Health in
April 2022, the disease affects approximately 1 in 40,000 patients. Moreover,
consistent with the study titled "Carrier frequency and predicted genetic
prevalence of pompe disease supported a general population database"
genetic science and Metabolism report in June 2021, Pathogenic or likely
pathogenic variants (PPVs) were found in higher proportions in East Asian and
African patients with classic infantile-onset pompe disease. Within the general
population, total carrier frequency (CF) and predicted genetic prevalence (PGP)
was 1.3 % (1 in 77) and 1:23,232, respectively. Thus, the rising prevalence of pompe
disease is predicted to fuel the market growth. The launching of novel drugs
globally will propel the demand for treatment of this disorder and further
provide lucrative growth opportunities for the market. However, the high cost
of the treatment will restrain the expansion of the market during the forecast
period.
The COVID-19 epidemic had an
inconsequential effect on the pompe disease market during the forecast period.
The outbreak of the COVID-19 epidemic has affected the production and logistics
of medical products utilized for pompe disease patients. As per the study
titled "COVID-19 infection in patients with late-onset pompe disease"
published within the Muscles and Nerve in March 2022, in some pompe disease
patients, COVID-19 infection can have a benign outcome. On Moreover hand,
individuals with Pompe disease are still at high risk. Thus, COVID-19
significantly impacted the pompe disease market. Due to these factors, the
expansion of the market was restrained during the covid-19 pandemic.
The global pompe disease market is
segmented into the Asia Pacific, North America, Europe, the Middle East &
Africa, and South America. Globally, North America holds the largest market
share, of the market. However, product launches by the key market players with
reference to the segment are anticipated to drive the market over the forecast
period. As an example, In August 2021, The U.S. Food and Drug Administration
(FDA) approved a replacement enzyme replacement therapy, avalglucosidase alfa,
for patients aged 1 year and older with late-onset pompe disease. The European
region is anticipated to be the second-largest revenue-making region during the
forecast period. Experimental drugs prepared for pompe disease are gaining an
increasing number of approvals within the region. For example, the Marketing
Authorization Applications for AT-GAA, an investigational two-component
medication introduced by Amicus Therapeutics for the treatment of Pompe
disease, are verified by the ECU Medicines Agency (EMA). This will enhance the
market growth within the European region. However, the Asia Pacific is
anticipated to be the fastest growing region in pompe disease market during the
forecast period. The expansion of the region is followed by the expansion in
India and China because of the increased awareness of pompe disease and rising
genetic prevalence within the countries, which have resulted in the continuous
growth of the market.
The report on the global pompe disease
market covers segments such as therapy type, molecule type, and dosage form. On
the basis of therapy type, the sub-markets include enzyme replacement therapy,
and substrate reduction therapy. On the basis of molecule type, the sub-markets
include biologics, and small molecules. On the basis of dosage form, the
sub-markets include solid, and liquid.
The report provides profiles of
the companies in the market such as Amicus Therapeutics, BioMarin
Pharmaceuticals, Genzyme, Audentes Therapeutics, EPIVAX, OXYRANE, Valerion
Therapeutics, Sangamo Biosciences, Sanofi, and AVROBIO, Inc.
The report provides deep insights
into the demand forecasts, market trends, and micro and macro indicators. In
addition, this report provides insights into the factors that are driving and
restraining the growth in this market. Moreover, The IGR-Growth Matrix analysis
given in the report brings an insight into the investment areas that existing or
new market players can consider. The report provides insights into the market
using analytical tools such as Porter's five forces analysis and DRO analysis
of pompe disease market. Moreover, the study highlights current market trends
and provides forecast from 2022-2028. We also have highlighted future trends in
the market that will affect the demand during the forecast period. Moreover,
the competitive analysis given in each regional market brings an insight into
the market share of the leading players.
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