Lysosomal Storage Diseases Therapeutics Market to grow with a CAGR of 7.20%
Increasing
healthcare infrastructure and a supportive regulatory environment are the major
drivers for the Global Lysosomal Storage Diseases Therapeutics Market.
According
to TechSci Research report, “Global Lysosomal Storage Diseases Therapeutics Market
Industry Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028”, Global Lysosomal Storage
Diseases Therapeutics Market has valued at USD 7.60 billion in 2022 and is
anticipated to witness an impressive growth in the forecast period with a CAGR
of 7.20% 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.
Emerging therapies play a significant role in
driving the demand for the Global Lysosomal Storage Diseases (LSDs)
Therapeutics Market. These emerging therapies represent the cutting-edge
advancements in the field of LSD research and offer the potential for more
effective treatments and improved outcomes for patients. Emerging therapies
often bring novel approaches to LSD treatment. They may include innovative
mechanisms of action, such as gene therapies, small molecule chaperone
therapies, and substrate reduction therapies. These new options expand the
range of treatment choices for both patients and healthcare providers. Many
emerging therapies aim to modify the course of the disease, rather than just
addressing symptoms. This is particularly important for LSDs, as it offers the
potential to slow or halt disease progression and prevent irreversible damage
to organs and tissues. Emerging therapies may offer improved efficacy compared
to existing treatments. They may target the underlying genetic mutations or
biochemical defects more precisely, resulting in better therapeutic outcomes
and quality of life for patients. Some emerging therapies are designed to be
personalized, where treatment is tailored to an individual patient's genetic
profile and disease characteristics. This approach aligns with the trend toward
precision medicine and can optimize treatment effectiveness. The development
and testing of emerging therapies often involve clinical trials. Patients and
healthcare providers are increasingly interested in participating in these
trials to access innovative treatments and contribute to scientific
advancements.
Lysosomal
storage diseases are metabolic conditions characterized by the accumulation of macromolecules,
such as toxic substances and undigested cell particles. This buildup occurs due
to a deficiency of enzymes in the lysosomes. These disorders can impact various
body systems, including the circulatory, central nervous, digestive, integumentary,
hepatic, ocular, skeletal, splenic, and renal systems. There are over 50
distinct types of lysosomal storage diseases, and this disorder affects
approximately one in 7,700 births.
In
December 2022, patients with Gaucher disease who received a single dose of the
investigational hematopoietic stem cell (HSC) gene therapy AVR-RD-02 showed
stabilization or reversal of multiple clinically relevant measures, according
to new interim pharmacokinetic, pharmacodynamic, and clinical efficacy data
released by AVROBIO, Inc., a leading clinical-stage gene therapy company.
Additionally, AVROBIO intends to start a worldwide, registrational Phase 2/3
clinical study for Gaucher disease type 3 (GD3) in the second half of 2023
following fruitful conversations with regulators. The most significant and
prevalent lysosomal condition is Gaucher disease. People with Gaucher disease
type 1 (GD1) often have a shorter life expectancy and may endure incapacitating
symptoms that greatly lower their quality of life, even when receiving enzyme
replacement therapy (ERT), the current gold standard of care. GD3 is a more
severe, progressive type of Gaucher disease that manifests as neurological
signs and symptoms as well as more extensive systemic manifestations that are
frequently resistant to routine medical treatments.
High development costs are a significant
challenge in the Global Lysosomal Storage Diseases (LSDs) Therapeutics Market,
as in many areas of pharmaceutical research and drug development. LSDs are
considered rare diseases, and the patient populations for each LSD subtype are
relatively small. Developing therapies for rare diseases can be financially
challenging because the potential market size is limited. This makes it
difficult for pharmaceutical companies to recoup their development costs
through sales alone. Developing effective therapies for LSDs involves extensive
research into the underlying genetic and molecular mechanisms of each disease
subtype. This research can be complex and time-consuming, requiring substantial
investments in laboratory work, preclinical studies, and clinical trials. Conducting
clinical trials for LSD therapeutics can be costly due to the need for
specialized medical expertise, patient recruitment and retention efforts, and
the extended duration of trials, as rare diseases often require longer
follow-up periods to assess long-term safety and efficacy. Producing therapies
for LSDs, especially biologic treatments like enzyme replacement therapy (ERT)
or gene therapy, can be technically challenging and expensive. Ensuring
consistent quality and safety of these treatments requires sophisticated
manufacturing processes.
Browse
over XX market data Figures and spread through 110 Pages and an in-depth TOC
on "Lysosomal Storage Diseases Therapeutics Market.”
Global Lysosomal Storage Diseases Therapeutics Market
is segmented based on Treatment, Indication, End-User, and by region.
Based on Treatment,
Global Lysosomal Storage Diseases Therapeutics Market is segmented into Enzyme
Replacement Therapy, Stem Cell Therapy, Substrate Reduction Therapy, others.
Substrate Reduction Therapy (SRT) is a therapeutic approach used to treat certain
lysosomal storage diseases (LSDs), which are rare genetic disorders
characterized by the accumulation of specific substances within lysosomes, the
cellular organelles responsible for breaking down various molecules. SRT is
designed to reduce the accumulation of these substances, known as substrates,
by inhibiting their production within the body. SRT targets the metabolic
pathway responsible for the synthesis of the substrate that accumulates in the
lysosomes. By inhibiting or reducing the production of this substrate, SRT aims
to slow down or prevent its buildup within the lysosomes. In some LSDs, the
accumulation of substrates occurs because of a deficiency in a lysosomal enzyme
that normally breaks down the substrate. SRT does not attempt to replace the
missing enzyme, as enzyme replacement therapy (ERT) does. Instead, it uses small
molecule compounds to inhibit an upstream enzyme in the metabolic pathway,
which reduces the production of the substrate. This approach does not require
the delivery of enzymes into lysosomes.
Based on Region, North America dominated the Global Lysosomal Storage Diseases Therapeutics Market.
North America has a well-established clinical trial infrastructure, making it
an attractive location for conducting clinical trials for LSD therapies. Access
to a diverse patient population is essential for testing the safety and
efficacy of these treatments. North America has strong patient advocacy
organizations that raise awareness about rare diseases, including LSDs. These
groups advocate for patients' needs, research funding, and access to therapies.
In countries like the United States, where healthcare insurance coverage plays
a crucial role, the availability of insurance for rare disease treatments can
be more favorable, making it easier for patients to access these therapies. North
America has a relatively larger population compared to many other regions,
which can make it an attractive market for pharmaceutical companies developing
LSD therapies. North American researchers and institutions often collaborate
with their counterparts in other regions, contributing to the global effort to
advance LSD therapeutics.
Asia-pacific region to witness fastest growth in the
Global Lysosomal Storage Diseases
Therapeutics Market. Over the years, there has been an increase in
awareness about rare diseases, including LSDs, in the Asia-Pacific region.
Healthcare professionals, patient advocacy groups, and government initiatives
have played a role in raising awareness. This heightened awareness has led to
more accurate diagnoses and increased demand for treatment options. Many
countries in the Asia-Pacific region have been investing in expanding and
improving their healthcare infrastructure. This includes the development of
specialized treatment centers, diagnostic facilities, and expertise in rare
disease management. Rising incomes and economic growth in several Asia-Pacific
countries have led to increased healthcare spending. Patients and their
families are more willing and able to seek specialized treatments for rare
diseases like LSDs. Some governments in the Asia-Pacific region have introduced
policies and initiatives to support patients with rare diseases. This support
includes funding for research, subsidies for medications, and incentives for
pharmaceutical companies to develop treatments for rare diseases.
Some
of the major companies operating in the Global
Lysosomal Storage Diseases Therapeutics Market include:
- Pfizer,
Inc.
- Sanofi
SA
- BioMarin
Pharmaceutical Inc
- Actelion
Ltd.
- Raptor
Pharmaceutical Corp.
- Protalix
Biotherapeutics Inc.
- Amicus
Therapeutics, Inc.
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“Certain areas, particularly in North
America, are projected to exert significant demand for Lysosomal Storage
Diseases Therapeutics. 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 Lysosomal Storage Diseases Therapeutics Market in the
forecast period," said Mr. Karan Chechi, Research Director with TechSci
Research, a research-based Global management consulting firm.
Lysosomal Storage Diseases Therapeutics Market – Global Industry Size,
Share, Trends, Opportunity, and Forecast, 2018-2028 Segmented by Treatment
(Enzyme Replacement Therapy, Stem Cell Therapy, Substrate Reduction Therapy,
others), By Indication (Gaucher's Disease, Fabry Disease, Pompe's Disease, Mucopolysaccharidosis, others), By End
User (Hospitals, Clinics), by region, and Competition evaluated
the future growth potential of Global Lysosomal Storage Diseases Therapeutics 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 Lysosomal Storage Diseases Therapeutics
Market.
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