Fabry Disease Treatment Market to grow with a CAGR of 6.30%
Increasing
healthcare infrastructure and a supportive regulatory environment are the major
drivers for the Global Fabry Disease Treatment Market.
According
to TechSci Research report, “Global Fabry Disease Treatment Market Industry
Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028”, Global Fabry Disease Treatment
Market has valued at USD 1862.50 million in 2022 and is anticipated to witness
an impressive growth in the forecast period with a CAGR of 6.30% through 2028. This
can be due to collaborations and partnerships among leading companies with a
diverse approach to merge the expertise of individual companies and to
strengthen their position in the market.
Technological advancements have played a
significant role in improving the diagnosis and treatment of Fabry disease.
These advancements have contributed to earlier diagnosis, more effective
therapies, and better patient management. Telemedicine and telehealth
technologies have expanded access to Fabry disease specialists, allowing
patients to receive consultations and diagnostic evaluations remotely. This is
especially important for individuals in underserved or remote areas. Advances
in data analytics and electronic health records have improved the management of
Fabry disease patients. Healthcare providers can track disease progression,
treatment responses, and outcomes more effectively. Innovations in drug
delivery technology have enhanced the convenience and effectiveness of Fabry
disease treatments. For example, advancements in enzyme replacement therapy
(ERT) delivery methods can make it easier for patients to receive treatment.
Technologies used in clinical trials, such as virtual patient registries and
remote monitoring, have improved the efficiency of drug development and the
collection of real-world data on Fabry disease. Mobile applications and
wearable devices can help patients manage their disease and track important
health parameters. These technologies promote self-monitoring and adherence to
treatment plans. Advances in laboratory techniques, such as CRISPR-Cas9 gene
editing and stem cell technology, have facilitated research into potential gene
therapies and disease mechanisms.
Fabry
disease, also known as Anderson-Fabry disease, Fabry's disease, or alpha-galactosidase-A
deficiency, is a rare genetic disorder characterized by insufficient production
of the enzyme alpha-galactosidase A (alpha-GAL). This enzyme plays a crucial
role in breaking down sphingolipids, a fatty substance, and preventing its harmful
accumulation in blood vessels and tissues. Fabry disease belongs to a group of
conditions known as lysosomal storage disorders. The deficiency of functional
alpha-GAL enzymes leads to the harmful buildup of sphingolipids in the body's
blood vessels and tissues. This condition has a significant impact on various
organs and systems, including the heart, kidneys, brain, central nervous
system, and skin.
In
May 2023, The FDA has approved isaralgagene civaparvovec, also known as ST-920,
a wholly owned gene therapy product candidate for the treatment of Fabry
disease, according to Sangamo Therapeutics, Inc., a genomic medicine business.
A novel therapy that is designed to treat serious or life-threatening illnesses
and that has the potential to meet unmet medical needs is given Fast Track
classification to facilitate its development and quicken its evaluation. An
opportunity for more regular engagement with the FDA is provided to companies
who receive this classification. These clinical programs could also be
qualified to apply for Priority Review and Accelerated Approval provided the
necessary requirements are satisfied. Prior until now, the FDA designated ST-920
as an orphan drug.
Long-term treatment monitoring is a
significant challenge in the Global Fabry Disease Treatment Market, as it poses
several complexities and considerations for both patients and healthcare
providers. Fabry disease is a lifelong condition that requires continuous
treatment and monitoring. Patients typically need to adhere to their treatment
regimens and undergo regular medical assessments to manage the disease
effectively. Fabry disease can affect multiple organs, including the heart,
kidneys, nervous system, and skin. Monitoring these different aspects of the
disease's progression and managing associated complications requires a
coordinated and comprehensive approach. The symptoms of Fabry disease can vary
widely among individuals. Some patients may experience mild symptoms, while
others may have severe complications. Monitoring and managing these variable
symptoms over the long term can be challenging. Ensuring that patients adhere
to their prescribed treatment regimens, which often involve enzyme replacement
therapy (ERT) and other medications, is essential for disease management.
Non-adherence can lead to disease progression and complications. Regular
assessment of organ function, such as kidney and cardiac function, is crucial
for detecting early signs of organ damage and adjusting treatment plans
accordingly. These assessments may involve various tests and imaging studies.
Browse
over XX market data Figures and spread through 110 Pages and an in-depth TOC
on "Fabry Disease Treatment Market.”
Global Fabry Disease Treatment Market is segmented based
on Drugs, Treatment, Route of Administration, Distribution Channel, and by
region. Based on
Treatment, Global Fabry Disease
Treatment Market is segmented into Enzyme Replacement Therapy (ERT), Chaperone
Treatment, Substrate Reduction Therapy (SRT), and others. Substrate
Reduction Therapy (SRT) is a therapeutic approach used in the treatment of
certain lysosomal storage disorders, including Gaucher disease and Niemann-Pick
disease. These disorders are characterized by the accumulation of specific
substances, or substrates, within the lysosomes of cells. Lysosomes are
cellular structures responsible for breaking down various molecules, and when
they don't function properly, it can lead to the buildup of harmful substances.
SRT aims to reduce the accumulation of these substances by inhibiting their
production within the body. It works by targeting and inhibiting the enzyme
responsible for synthesizing the accumulated substrate. SRT is generally
tailored to the specific lysosomal storage disorder being treated and the
enzyme involved. Different disorders require the development of specific
inhibitors.
Based on Region, North America dominated the Global Fabry Disease Treatment Market. Patients
in North America have relatively good access to Fabry disease treatments,
including enzyme replacement therapy (ERT) and other emerging therapies. Health
insurance coverage and reimbursement policies in the region can support
patients’ access to these treatments. North America has active patient advocacy
groups and organizations dedicated to rare diseases like Fabry disease. These
groups play a crucial role in raising awareness, advocating for patients, and
supporting research efforts. North America has some of the highest healthcare
spending levels in the world. This financial investment in healthcare
contributes to research and development efforts for rare diseases. The region
is home to many leading pharmaceutical companies with a focus on rare diseases.
These companies invest in research and development of Fabry disease treatments,
leading to the availability of a variety of therapeutic options. Healthcare
professionals in North America receive comprehensive education and training in
the diagnosis and treatment of rare diseases. This expertise contributes to
better patient care.
Asia-pacific region to witness fastest growth in the
Global Fabry Disease Treatment Market.
Over the years, there has been a growing awareness of rare diseases like Fabry
disease in the Asia-Pacific region. Improved disease awareness has led to
increased diagnosis rates and a greater demand for treatment. Many countries in
the Asia-Pacific region have experienced significant economic growth, leading
to increased healthcare expenditure. With more resources allocated to
healthcare, there is greater potential for investment in rare disease
treatments, including Fabry disease. The pharmaceutical market in the
Asia-Pacific region has been expanding, driven by factors such as a growing
population, urbanization, and increased access to healthcare. This growth has
made it an attractive market for pharmaceutical companies to introduce and
market Fabry disease treatments. Several countries in the Asia-Pacific region,
such as China and India, are considered emerging markets for healthcare. As
healthcare infrastructure improves and access to medical services increases,
more patients with rare diseases are likely to be diagnosed and seek treatment.
Some
of the major companies operating in the Global
Fabry Disease Treatment Market include:
- Sanofi
(Genzyme Corporation)
- Takeda
Pharmaceutical Company Limited
- Amicus
Therapeutics, Inc
- ISU
ABXIS Co Ltd.
- JCR
Pharmaceuticals Co., Ltd.
- Protalix
BioTherapeutics Inc.
- Chiesi
Farmaceutici S.p.A.
- Freeline
Therapeutics Holdings PLC
- Yuhan
Corporation
- M6P
Therapeutics
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“Certain areas, particularly in North
America, are projected to exert significant demand for Fabry Disease Treatment.
The growth in the competitive landscape and the presence of well-established
companies in the market, committed to enhance the overall wellbeing of people each
year, are expected to contribute to a remarkable growth of the Global Fabry
Disease Treatment Market in the forecast period," said Mr. Karan Chechi,
Research Director with TechSci Research, a research-based Global management
consulting firm.
Fabry Disease Treatment Market – Global Industry Size, Share, Trends, Opportunity,
and Forecast, 2018-2028 Segmented by Drugs (Agalsidase Beta, Migalastat, others), By Treatment (Enzyme Replacement Therapy (ERT),
Chaperone Treatment, Substrate
Reduction Therapy (SRT), others), By Route of Administration (Oral, Parenteral,
others), By Distribution Channel (Hospital pharmacies, Retail pharmacies,
Online Pharmacies), by region, and Competition evaluated
the future growth potential of Global Fabry Disease Treatment Market and
provides statistics & information on market size, structure, and future
market growth. The report intends to provide innovative market intelligence and
help decision makers take sound investment decisions. Besides, the report also
identifies and analyzes the emerging trends along with essential drivers,
challenges, and opportunities in Global Fabry Disease Treatment Market.
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