Alport Syndrome Treatment Market to grow with a CAGR of 4.00%
Increasing
healthcare infrastructure and initiatives taken by the government are the major
drivers for the Global Alport Syndrome Treatment Market.
According
to TechSci Research report, “Global Alport Syndrome Treatment Market
Industry Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028”, Global Alport Syndrome
Treatment Market has valued at USD 8.90 million in 2022 and is anticipated to
witness an impressive growth in the forecast period with a CAGR of 4.00 %
through 2028. This can be ascribed 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. Advancements in
therapeutic approaches in Alport syndrome treatment are critical in addressing
the unmet medical needs of affected individuals. Advances in genetic research
have paved the way for precision medicine in Alport syndrome treatment.
Identifying specific genetic mutations allows for personalized treatment
approaches that target the underlying genetic cause of the disease. This
approach aims to develop therapies tailored to an individual's genetic subtype.
RNA-targeted genetic therapies, such as antisense oligonucleotides (ASOs) and
small interfering RNA (siRNA), are being investigated as potential treatments
for Alport syndrome.
These
therapies aim to modify or correct the expression of mutated genes responsible
for the condition. Exosomes, tiny vesicles secreted by cells, have shown
potential in delivering therapeutic molecules to target tissues. Researchers
are exploring exosome-based therapies to deliver genetic material or
therapeutic agents to affected kidney cells. The identification of specific
biomarkers associated with Alport syndrome can aid in early diagnosis and
treatment monitoring. Biomarker development is an active area of research in
the field. Inflammation plays a role in the progression of kidney damage in
Alport syndrome. Emerging therapies include the use of anti-inflammatory agents
to reduce kidney inflammation and slow disease progression.
Alport
syndrome is a rare genetic disorder characterized by kidney disease, hearing
impairment, and vision issues. It results from mutations in the COL4A3, COL4A4,
or COL4A5 genes, which are responsible for producing crucial proteins like type
IV Collagen. Individuals with Alport syndrome experience a gradual decline in
kidney function, often indicated by the presence of blood in the urine as a
sign of impaired kidney function. Many Alport syndrome patients also exhibit
elevated protein levels in their urine. With disease progression, kidney
function deteriorates, ultimately leading to End-Stage Renal Disease (ESRD).
Furthermore, Alport syndrome is associated with inner ear abnormalities,
resulting in sensorineural hearing loss, as well as vision impairment.
In February 2023, Eloxx Pharmaceuticals, Inc., a
pioneer in the development of genetic therapies focused on ribosomal RNA, has
commenced the dosing of the initial patients in its Phase 2 clinical trial for
ELX-02. This trial aims to assess the potential of ELX-02 as a treatment for
Alport syndrome in patients with nonsense mutations. In this Phase 2 trial, the
focus is on administering ELX-02 to a maximum of eight Alport syndrome patients
who possess nonsense mutations in the COL4 gene. These patients will undergo a
two-month dosing regimen, followed by a three-month monitoring period. In
addition to prioritizing safety as the primary endpoint, the study will also
closely monitor a significant secondary efficacy endpoint, which is the
measurement of proteinuria every two weeks. Proteinuria is a well-established
endpoint used in evaluating treatments for various kidney-related conditions
and serves as a reliable predictor of treatment effectiveness. Furthermore, for
eligible participants, the induction of COL4 will be assessed at the conclusion
of the two-month period.
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Limited treatment options pose a significant challenge
in the global Alport syndrome treatment market. There are currently no
disease-modifying treatments available for Alport syndrome. This means that
existing therapies primarily focus on symptom management and slowing disease
progression rather than addressing the underlying genetic cause of the
condition. The available treatments for Alport syndrome, such as blood pressure
control and medications to reduce proteinuria, have limited efficacy in preventing
kidney disease progression. While they can be beneficial, they may not halt the
disease's advance in many cases. Treatment options for hearing loss associated
with Alport syndrome are often limited to hearing aids or cochlear implants.
These devices can improve hearing to some extent but may not fully restore
normal auditory function. Alport syndrome patients and their families often
seek curative therapies that can address the genetic mutations responsible for
the disease. The absence of curative options can be frustrating and
disappointing. Advances in genetic research have raised hopes for personalized
medicine approaches to treat Alport syndrome. Limited treatment options hinder
the development of targeted therapies tailored to an individual's specific
genetic subtype.
Global Alport Syndrome Treatment Market
segmentation is based on Product Type, Disease
Type, End User, By Company, and Region.
Based on Disease Type, Global Alport Syndrome
Treatment Market is segmented into X-linked Alport Syndrome, Autosomal
Recessive Alport Syndrome, Autosomal Dominant Alport Syndrome. Autosomal
recessive Alport syndrome is one of the genetic inheritance patterns of Alport
syndrome, a rare genetic disorder that primarily affects the kidneys, ears, and
eyes. In autosomal recessive Alport syndrome, the condition is caused by
mutations in specific genes, and it follows an inheritance pattern that can
affect both males and females. Autosomal recessive Alport syndrome is inherited
when an individual inherits two mutated copies of the relevant genes, one from
each parent. These mutated genes are typically located on autosomal
chromosomes, which are non-sex chromosomes (i.e., not the X or Y chromosomes).
The progression of autosomal recessive
Alport syndrome can vary among individuals. It often leads to kidney
insufficiency or failure over time, necessitating kidney replacement therapies
such as dialysis or transplantation.
Based on Region, the North America segment is the
dominating segment of Global Alport Syndrome Treatment Market. North America is home to numerous
pharmaceutical and biotechnology companies with a focus on rare diseases. These
companies play a crucial role in developing and commercializing treatments for
Alport syndrome. The region's relatively large population allows for a
sufficient pool of patients for clinical trials and the establishment of
specialized clinics and centers of excellence for rare diseases. In countries
like the United States and Canada, health insurance coverage may help
facilitate access to costly treatments, reducing the financial burden on
patients and their families. North American researchers and organizations often
collaborate with international counterparts, contributing to a global effort to
advance Alport syndrome research and treatment. While healthcare access varies
within North America, the region generally provides relatively good access to
healthcare services, diagnostics, and specialized care for rare diseases.
Asia
Pacific is the fastest-growing region in the Global Alport Syndrome Treatment Market.
Awareness about rare diseases like Alport syndrome is growing in the Asia
Pacific region. As healthcare providers and patients become more informed,
there is greater recognition and diagnosis of the condition, leading to
increased demand for treatment. Many countries in the Asia Pacific have been
investing in expanding their healthcare infrastructure. This includes the
establishment of specialized centers and clinics for rare diseases, improving
access to diagnosis, treatment, and management of Alport syndrome. Advances in
genetic testing have made it easier to diagnose Alport syndrome and identify
specific genetic mutations. Improved access to genetic testing facilities in
the Asia Pacific has contributed to early diagnosis and targeted treatment. The
region has witnessed a growing interest in rare disease research, including
Alport syndrome. Pharmaceutical companies and research institutions are
conducting clinical trials and studies in the Asia Pacific, making novel
treatments and therapies more accessible.
Some
of the major companies operating in the Global
Alport Syndrome Treatment Market include:
- Retrophin,
Inc.
- Goldfinch
Bio, Inc.
- Regulus
Therapeutics Inc.
- Vertex
Pharmaceuticals Incorporated
- ChemoCentryx,
Inc.
- Reata
Pharmaceuticals, Inc.
- Sanofi
S.A.
- Ionis
Pharmaceuticals, Inc.
- Omeros
Corporation
- Akebia
Therapeutics, Inc.
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“Certain areas, particularly in North
America, are projected to exert significant demand of Alport Syndrome
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 Alport Syndrome Treatment Market in the forecast period," said Mr.
Karan Chechi, Research Director with TechSci Research, a research-based Global
management consulting firm.
Alport
Syndrome Treatment Market - Global Industry Size, Share, Trends, Opportunity,
and Forecast, 2018-2028 Segmented by
Product Type (Tablets, Capsules, Injectable, Others), By Disease Type (X-linked
Alport Syndrome, Autosomal Recessive Alport Syndrome, Autosomal Dominant Alport
Syndrome), By End User (Hospital Pharmacies, Retail Pharmacies, Specialty
Clinics), By Region, and Competition has
evaluated the future growth potential of Global Alport Syndrome 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 Alport Syndrome
Treatment Market.
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