The dynamics of the Alport syndrome market are anticipated
to change in the coming years owing to the improvement in the rise
in healthcare spending across the world.
LAS
VEGAS, April 17, 2024 /PRNewswire/ --
DelveInsight's Alport Syndrome Market Insights report
includes a comprehensive understanding of current treatment
practices, Alport syndrome emerging drugs, market share of
individual therapies, and current and forecasted market size from
2020 to 2034, segmented into 7MM [the
United States, the EU4 (Germany, France, Italy, and Spain), the United
Kingdom, and Japan].
Key Takeaways from the Alport Syndrome Market Report
- According to DelveInsight's analysis, the market size for
Alport syndrome reached USD 20
million in 2023 across the 7MM.
- Alport syndrome is an inherited disease, with X-linked being
the most common type of it, and accounts for approximately
85% of the total cases.
- DelveInsight's analysis reveals that the overall prevalent
population of Alport syndrome in the 7MM was reported as
159K in 2023.
- Leading Alport syndrome companies such as Eloxx
Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer,
Calliditas Therapeutics, Evotec, and others are developing
novel Alport syndrome drugs that can be available in the Alport
syndrome market in the coming years.
- Some promising Alport syndrome therapies in the pipeline
include ELX-02, Atrasentan, Finerenone, Setanaxib,
BAY3401016, and others.
- Over the next decade, improved early diagnosis and treatment
advancements are expected to raise the age at which kidney failure
manifests in individuals with Alport syndrome. Additional benefits
are anticipated from novel therapies that can complement ACE
inhibition. While safe and effective curative therapies are within
the realm of possibility, notable challenges must be addressed to
transform these possibilities into reality.
- ELX-02 can potentially become the first gene therapy for Alport
syndrome patients with nonsense mutations.
Discover which therapies are expected to grab the major Alport
syndrome market share @ Alport Syndrome Market
Report
Alport Syndrome Overview
Alport syndrome denotes a range of hereditary, diverse
conditions impacting the kidney's basement membranes, often with
effects on the cochlea and eyes. It is a genetic condition
resulting from mutations in the genes that code for alpha-3,
alpha-4, and alpha-5 of type 4 collagen (COL4A3, COL4A4, COL4A5),
also known as the collagen 4 α345 network. The disorder manifests
in different forms such as autosomal recessive Alport syndrome
(ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant
Alport syndrome (ADAS).
People with Alport syndrome may exhibit signs such as blood in
urine, excessive protein in urine, swelling, high blood pressure,
progressive deterioration in kidney function, and ultimately,
end-stage renal disease (ESRD). As time progresses, these symptoms
tend to worsen, with increasing protein in the urine, elevated
blood pressure, declining glomerular filtration rate (GFR), and
eventual development of ESRD. The diagnostic methods for confirming
Alport syndrome have been advancing in recent years. Diagnosis
typically involves recognizing characteristic symptoms, a thorough
review of the patient's medical history, and a comprehensive
clinical assessment. Suspicions of Alport syndrome are heightened
in individuals with a family history of the condition, unexplained
kidney failure, early onset of hearing loss, or the presence of
blood in urine.
Alport Syndrome Epidemiology Segmentation
Alport syndrome is more prevalent in the adult population with
~86% contribution in the 7MM, while the pediatric population
accounted for ~14% of cases.
The X-linked Alport syndrome (XLAS) is the most prevalent
subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal
dominant Alport syndrome (ADAS) is the least prevalent subtype of
Alport syndrome.
The Alport syndrome market report proffers epidemiological
analysis for the study period 2020–2034 in the 7MM segmented
into:
- Total Prevalent Cases of Alport Syndrome
- Total Diagnosed Prevalent Cases of Alport Syndrome
- Gender-specific Cases of Alport Syndrome
- Age-specific Cases of Alport Syndrome
- Type-specific Cases of Alport Syndrome
Alport Syndrome Treatment Market
Treatment for Alport syndrome typically focuses on managing
symptoms and slowing the progression of kidney damage. This often
involves a multidisciplinary approach with nephrologists,
otolaryngologists, and ophthalmologists. Medications such as ACE
inhibitors and angiotensin II receptor blockers (ARBs) are commonly
prescribed to help control high blood pressure and reduce
proteinuria, thus preserving kidney function. In cases of advanced
kidney failure, dialysis or kidney transplantation may be necessary
to replace lost kidney function and improve quality of life.
Ongoing research into potential gene therapies and other innovative
treatments holds promise for the future of Alport syndrome
management.
Additionally, supportive care plays a crucial role in managing
Alport syndrome. This includes regular monitoring of kidney
function, hearing, and vision, as well as lifestyle modifications
such as maintaining a healthy diet, staying hydrated, and avoiding
nephrotoxic substances. Genetic counseling is often recommended for
families to understand the inheritance pattern of Alport syndrome
and make informed decisions. By addressing both the medical and
emotional aspects of the condition, individuals with Alport
syndrome can work with their healthcare team to optimize their
treatment plan and lead fulfilling lives despite the challenges
posed by this rare genetic disorder.
To know more about Alport syndrome treatment guidelines, visit @
Alport Syndrome Management
Alport Syndrome Pipeline Therapies and Key Companies
- ELX-02: Eloxx Pharmaceuticals
- Atrasentan: Chinook Therapeutics/Novartis
- Finerenone: Bayer
- Setanaxib: Calliditas Therapeutics
- BAY3401016: Evotec/Bayer
Learn more about the FDA-approved drugs for Alport syndrome @
Drugs for Alport Syndrome Treatment
Alport Syndrome Market Dynamics
The dynamics of the Alport syndrome market are expected to
change in the coming years. With a deeper understanding of the
genetic basis of Alport syndrome, personalized treatment
strategies can be tailored to an individual's specific genetic
profile. ELX-02 is expected to garner the first mover
advantage in Alport syndrome apart from being the first gene
therapy with reduced frequency of administration (8 weeks), as an
increase in research and development has paved the way for
discovering novel MoA therapies such as dual NOX inhibitor
(setanaxib), semaphorin-3A blocker (BAY3401016), selective
endothelin A receptor antagonist (atrasentan), amidst the usage
of ACE/ARB inhibitors as standard of care.
Genetic advances may open the way for personalized treatment
tailored to the particular genetic profiles of individuals
with Alport syndrome, thus there is a huge opportunity for
precision medicine approaches, with patient advocacy groups
such as the Alport Syndrome Foundation playing a pivotal
role in shaping the landscape for Alport syndrome by directing the
majority of its resources to research and research-related
activities, resulting in groundbreaking knowledge and clinical
trials pertaining to Alport syndrome.
Furthermore, many potential therapies are being investigated for
the treatment of Alport syndrome, and it is safe to predict that
the treatment space will significantly impact the Alport
syndrome market during the forecast period. Moreover, the
anticipated introduction of emerging therapies with improved
efficacy and a further improvement in the diagnosis rate are
expected to drive the growth of the Alport syndrome market in
the 7MM.
However several factors may impede the growth of the Alport
syndrome market. The gene mutation causing Alport syndrome
might remain silent in a person's parents, complicating diagnosis,
often resulting in late-stage identification following significant
kidney damage. Over half of those under current therapies
experience only moderate symptom reduction, leaving substantial
needs unmet. Eloxx Pharmaceuticals' gene therapy is set to
launch at premium prices, potentially raising
accessibility and affordability concerns in certain nations.
Off-label treatments such as ACE inhibitors and ARBs pose a
significant threat to the Alport syndrome market's overall
growth.
Emerging therapies, with their high treatment costs,
could further limit market access, rendering drugs, specialized
care, and potential renal replacement unaffordable for many
families. Financial constraints might hinder patients'
critical therapy access, a problem compounded by various FDA trial
discontinuations and approval withdrawals. In 2022, Bardoxolone
Methyl faced FDA rejection, and its Japanese trial was halted,
while Sanofi discontinued Lademirsen's Phase II
trial. In addition, the undiagnosed, unreported cases and
the unawareness about the disease may also impact the Alport
syndrome market growth.
Alport Syndrome
Market Report Metrics
|
Details
|
Study Period
|
2020–2034
|
Coverage
|
7MM [the United States,
the EU4 (Germany, France, Italy, and Spain), the United Kingdom,
and Japan].
|
Alport Syndrome
Market CAGR
|
69 %
|
Alport Syndrome
Market Size in 2023
|
USD 20
Million
|
Key Alport Syndrome
Companies
|
Eloxx Pharmaceuticals,
Chinook Therapeutics/Novartis, Bayer, Calliditas Therapeutics,
Evotec, and others
|
Key Pipeline Alport Syndrome Therapies
|
ELX-02, Atrasentan,
Finerenone, Setanaxib, BAY3401016,
and others
|
Scope of the Alport Syndrome Market
Report
- Therapeutic Assessment: Alport Syndrome current
marketed and emerging therapies
- Alport Syndrome Market Dynamics: Key
Market Forecast Assumptions of Emerging Alport Syndrome Drugs and
Market Outlook
- Competitive Intelligence Analysis: SWOT analysis
and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, Alport Syndrome
Market Access and Reimbursement
Discover more about Alport syndrome drugs in development @
Alport Syndrome Clinical Trials
Table of Contents
1
|
Key Insights
|
2
|
Report
Introduction
|
3
|
Executive Summary of
Alport Syndrome
|
4
|
Epidemiology and Market
Forecast Methodology
|
5
|
Key Events
|
6
|
Alport Syndrome Market
Overview at a Glance
|
6.1
|
Market Size
Distribution by Therapies in 2023
|
6.2
|
Market Size
Distribution by Therapies in 2034
|
7
|
Disease Background and
Overview
|
7.1
|
Introduction
|
7.2
|
Types of Alport
Syndrome
|
7.3
|
Signs and
Symptoms
|
7.3.1
|
Renal
Manifestations
|
7.3.2
|
Hearing
Impairment
|
7.3.3
|
Ocular
Manifestations
|
7.3.4
|
Leiomyomatosis
|
7.4
|
Causes of Alport
Syndrome
|
7.5
|
Pathophysiology of
Alport Syndrome
|
7.6
|
Diagnosis of Alport
Syndrome
|
7.6.1
|
Clinical
Diagnosis
|
7.6.2
|
Genetic
Diagnosis
|
7.6.3
|
Other Tests
|
7.7
|
Prognosis
|
7.8
|
Differential
Diagnosis
|
7.9
|
Prognosis
|
8
|
Treatment of Alport
Syndrome
|
8.1
|
Treatment
Algorithm
|
9
|
Diagnostic and
Treatment Guidelines
|
9.1
|
Guidelines for Genetic
Testing and Management of Alport Syndrome
|
9.2
|
Clinical Practice
Recommendations for the Diagnosis and Management of Alport Syndrome
in Children, Adolescents, and Young Adults (2020)
|
9.3
|
Expert Guidelines for
the Management of Alport Syndrome and Thin Basement Membrane
Nephropathy: American Society of Nephrology (2013)
|
10
|
Epidemiology and
Patient Population
|
10.1
|
Key Findings
|
10.2
|
Assumptions and
Rationale
|
10.3
|
Total Prevalent Cases
of Alport Syndrome in the 7MM
|
10.4
|
Total Diagnosed
Prevalent Cases of Alport Syndrome in the 7MM
|
10.5
|
The United
States
|
10.5.1
|
Total Prevalent Cases
of Alport Syndrome in the United States
|
10.5.2
|
Total Diagnosed
Prevalent Cases of Alport Syndrome in the United States
|
10.5.3
|
Gender-specific Cases
of Alport Syndrome in the United States
|
10.5.4
|
Age-specific Cases of
Alport syndrome in the United States
|
10.5.5
|
Type-specific Cases of
Alport Syndrome in the United States
|
10.6
|
EU4 and the
UK
|
10.6.1
|
Total Prevalent Cases
of Alport Syndrome in EU4 and the UK
|
10.6.2
|
Total Diagnosed
Prevalent Cases of Alport Syndrome in EU4 and the UK
|
10.6.3
|
Gender-specific Cases
of Alport Syndrome in EU4 and the UK
|
10.6.4
|
Age-specific Cases of
Alport Syndrome in EU4 and the UK
|
10.6.5
|
Type-specific Cases of
Alport syndrome in EU4 and the UK
|
10.7
|
Japan
|
10.7.1
|
Total Prevalent Cases
of Alport Syndrome in Japan
|
10.7.2
|
Total Diagnosed
Prevalent Cases of Alport Syndrome in Japan
|
10.7.3
|
Gender-specific Cases
of Alport Syndrome in Japan
|
10.7.4
|
Age-specific Cases of
Alport Syndrome in Japan
|
10.7.5
|
Type-specific Cases of
Alport Syndrome in Japan
|
11
|
Patient
Journey
|
12
|
Emerging
Therapies
|
12.1
|
Key Cross
Competition
|
12.2
|
ELX-02: Eloxx
Pharmaceuticals
|
12.2.1
|
Product
Description
|
12.2.2
|
Other Developmental
Activity
|
12.2.3
|
Clinical
Development
|
12.2.4
|
Safety and
Efficacy
|
12.3
|
Atrasentan: Chinook
Therapeutics/Novartis
|
12.3.1
|
Product
Description
|
12.3.2
|
Other Developmental
Activity
|
12.3.3
|
Clinical
Development
|
12.3.4
|
Safety and
Efficacy
|
12.4
|
Finerenone:
Bayer
|
12.4.1
|
Product
Description
|
12.4.2
|
Clinical
Development
|
12.4.3
|
Safety and
Efficacy
|
12.5
|
Setanaxib: Calliditas
Therapeutics
|
12.5.1
|
Product
Description
|
12.5.2
|
Other Developmental
Activity
|
12.6
|
BAY3401016:
Evotec/Bayer
|
12.6.1
|
Product
Description
|
12.6.2
|
Other Developmental
Activity
|
13
|
Alport Syndrome: 7MM
Market Analysis
|
13.1
|
Key Findings
|
13.2
|
Market
Outlook
|
13.3
|
Conjoint
Analysis
|
13.4
|
Key Market Forecast
Assumptions
|
13.5
|
Total Market Size of
Alport Syndrome in the 7MM
|
13.6
|
United States Market
Size
|
13.6.1
|
Total Market Size of
Alport Syndrome in the United States
|
13.6.2
|
Market Size of Alport
Syndrome by Therapies in the United States
|
13.7
|
EU4 and the UK Market
Size
|
13.7.1
|
Total Market Size of
Alport Syndrome in EU4 and the UK
|
13.7.2
|
Market Size of Alport
Syndrome by Therapies in EU4 and the UK
|
13.8
|
Japan Market
Size
|
13.8.1
|
Total Market Size of
Alport Syndrome in Japan
|
13.8.2
|
Market Size of Alport
Syndrome by Therapies in Japan
|
14
|
Unmet Needs
|
15
|
SWOT
Analysis
|
16
|
KOL Views
|
17
|
Market Access and
Reimbursement
|
17.1
|
United
States
|
17.1.1
|
Centers for Medicare
and Medicaid Services (CMS)
|
17.2
|
EU4 and the
UK
|
17.2.1
|
Germany
|
17.2.2
|
France
|
17.2.3
|
Italy
|
17.2.4
|
Spain
|
17.2.5
|
United
Kingdom
|
17.3
|
Japan
|
17.3.1
|
MHLW
|
18
|
Appendix
|
18.1
|
Bibliography
|
18.2
|
Report
Methodology
|
19
|
DelveInsight
Capabilities
|
20
|
Disclaimer
|
21
|
About
DelveInsight
|
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