The expected launch of potential therapies may increase
the Stargardt disease market size in the coming years, assisted by
an increase in the diagnosed prevalent population. Owing to the
positive outcomes of several products during the developmental
stage by key players such as Alkeus Pharmaceuticals, Kubota
Pharmaceuticals, Nanoscope Therapeutics, and others.
LAS VEGAS, April 24, 2024 /PRNewswire/ --
DelveInsight's Stargardt Disease Market
Insights report includes a comprehensive understanding of
current treatment practices, Stargardt disease 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) and the United Kingdom, and Japan].
Key Takeaways from the Stargardt Disease Market
Report
- According to DelveInsight's analysis, the market size for
Stargardt disease reached ~USD 28
million in 2023 across the 7MM.
- In 2023, the United States
accounted for the highest number of Stargardt disease cases, which
is 43% of the diagnosed-prevalent cases of Stargardt disease
in the 7MM.
- Leading Stargardt disease companies such as Kubota
Pharmaceuticals, Nanoscope Therapeutics, Alkeus Pharmaceuticals,
Belite Bio, Astellas Pharma, and others are developing novel
Stargardt disease drugs that can be available in the Stargardt
disease market in the coming years.
- The promising Stargardt disease therapies in the pipeline
include Emixustat, MCO-010, ALK-001 (Gildeuretinol), Tinlarebant
(LSB-008), IZERVAY (avacincaptad pegol), and
others.
- By 2034, MCO-010 (sonpiretigene isteparvovec) is
expected to garner the highest market share, followed by ALK-001
(gildeuretinol) in the 7MM.
- In January 2024, Alkeus
Pharmaceuticals announced positive interim data showing
gildeuretinol halted Stargardt disease progression for up to six
years. In its ongoing TEASE-3 clinical trial in early-stage
Stargardt disease, the first three teenage patients enrolled in
TEASE-3 and treated with oral gildeuretinol acetate remained
asymptomatic and free of disease progression for their treatment
duration ranging between two (one patient) and six years (two
patients).
Discover which therapies are expected to grab the major
Stargardt disease market share @ Stargardt Disease Market
Report
Stargardt Disease Overview
Stargardt disease, also called Stargardt's macular dystrophy or
juvenile macular degeneration, is the most common type of macular
degeneration seen in young individuals. Those affected by this
condition experience a decline in their vision, typically appearing
during their teenage years or early adulthood. This loss of vision
is linked to the deterioration of the retinal pigment epithelium
(RPE) and the gradual decrease in functional photoreceptors. In
nearly every instance, Stargardt disease leads to a permanent
reduction in vision that cannot be reversed.
The diagnostic assessment of Stargardt disease relies on factors
such as familial background, clarity of vision, examination of the
back of the eye, assessment of the visual field, use of fluorescein
angiography, observation of fundus autofluorescence (FAF),
electroretinography (ERG), and optical coherence tomography (OCT).
Currently, routine genetic testing is not commonly performed.
In the early stages of Stargardt disease, visual field tests
often appear normal. However, as the condition progresses, patients
may develop partial blind spots in the center of their vision,
which can eventually become complete blind spots. Typically, those
with Stargardt disease retain their peripheral vision, but in
severe cases where there is extensive retinal degeneration, a
narrowing of the visual field may occur. Another important
observation is the change in the preferred area of the retina used
for focusing vision.
Stargardt Disease Epidemiology Segmentation
In the US, out of all age groups, the highest onset age-specific
cases accounted for >20 years, followed by 20-39
years in 2020. In contrast, the least onset age-specific cases
were observed in =60 years age groups.
The Stargardt disease market report proffers
epidemiological analysis for the study period 2020–2034 in the 7MM
segmented into:
- Total Diagnosed Prevalent Cases of Stargardt Disease
- Type-specific Cases of Stargardt Disease
- Symptom-specific Cases of Stargardt Disease
- Treated Cases of Stargardt Disease
Stargardt Disease Treatment Market
The current Stargardt disease treatment choices involve methods
such as photoprotection and low-vision aids to assist in preventing
the advancement of the disease. Other options, such as slowing down
the visual cycle with medications, gene therapy, and various
treatments, are aimed at halting the buildup of lipofuscin and
offer hope for long-term improvement in vision. All-trans-retinal
that is not bound can cause damage from light exposure to the
particularly sensitive ABCA4, further hindering its function.
Considering that individuals with Stargardt disease already have
impaired ABCA4 function and heightened levels of trapped
all-trans-retinal in their photoreceptors, it is reasonable to
expect that these patients would be highly susceptible to the
effects of light exposure.
Vitamin A supplementation has been seen as a treatment choice
for specific retinal degenerative illnesses like Retinitis
Pigmentosa. Nonetheless, new information indicates that in cases of
ABCA4-mediated disease, the use of vitamin A supplements speeds up
the buildup of lipofuscin pigments in the RPE.
New methods for treating Stargardt Disease are centered on
tackling the root genetic issues and controlling symptoms to
decelerate the advancement of the disease. A strategy includes
employing innovative forms of vitamin A derivatives intended to
lessen harmful substances in the retina. The exploration of gene
therapy, which involves gene substitution and editing, is ongoing
to rectify the genetic mutations accountable for the condition.
Additionally, neurotrophic agents such as ciliary neurotrophic
factor (CNTF) are being used to protect photoreceptors and the
retinal pigment epithelium.
To know more about Stargardt disease treatment guidelines, visit
@ Stargardt Disease Management
Stargardt Disease Pipeline Therapies and Key
Companies
- Emixustat: Kubota Pharmaceuticals
- MCO-010: Nanoscope Therapeutics
- ALK-001 (Gildeuretinol): Alkeus Pharmaceuticals
- Tinlarebant (LSB-008): Belite Bio
- IZERVAY (avacincaptad pegol): Astellas Pharma
Learn more about the FDA-approved drugs for Stargardt disease @
Drugs for Stargardt Disease Treatment
Stargardt Disease Market Dynamics
The dynamics of the Stargardt disease market are expected to
change in the coming years. Stargardt disease, recognized by
regulatory agencies like the FDA as an orphan disease due to
its well-identified genetic basis primarily linked to mutations in
the ABCA4 gene, allows for targeted research and potential
therapeutic interventions, including the development of orphan
drugs and accelerated approval pathways, with ongoing
investigations into gene therapies, pharmacological interventions,
and emerging technologies providing opportunities for the creation
of novel and effective treatments, while the rapid advancements
in genetic technologies, such as CRISPR-Cas9, offer potential
breakthroughs in gene editing and correction of ABCA4
mutations.
As many potential therapies are being investigated for the
treatment of Stargardt disease, it is safe to predict that the
treatment space will significantly impact the Stargardt
disease 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 Stargardt disease market
in the 7MM.
However several factors may impede the growth of the Stargardt
disease market. Currently, there are no approved treatments
that effectively halt or reverse the progression of Stargardt
Disease, representing a significant unmet medical need, as
the absence of reliable biomarkers for disease progression and
treatment response complicates clinical trials and hinders the
development of personalized medicine, while insufficient
funding for rare diseases may impede research progress, slowing
down the development of potential therapies for Stargardt disease,
and the rarity of the disease and challenges in patient
recruitment may extend clinical trial timelines and limit
the available data for analysis.
Moreover, Stargardt disease treatment poses a
significant economic burden and disrupts patients' overall
well-being and QOL. Furthermore, the Stargardt disease market
growth may be offset by failures and discontinuation of emerging
therapies, unaffordable pricing, market access and
reimbursement issues, and a shortage of healthcare
specialists. In addition, the undiagnosed, unreported cases
and the unawareness about the disease may also impact the
Stargardt disease market growth.
Stargardt Disease
Market Report Metrics
|
Details
|
Study Period
|
2020–2034
|
Coverage
|
7MM [the United States,
the EU4 (Germany, France, Italy, and Spain) and the United Kingdom,
and Japan].
|
Stargardt Disease
Market CAGR
|
32 %
|
Stargardt
Disease Market Size in
2023
|
USD 28
Million
|
Key Stargardt Disease
Companies
|
Kubota Pharmaceuticals,
Nanoscope Therapeutics, Alkeus Pharmaceuticals, Belite Bio,
Astellas Pharma, and
others
|
Key Pipeline Stargardt Disease Therapies
|
Emixustat, MCO-010,
ALK-001 (Gildeuretinol), Tinlarebant (LSB-008), IZERVAY
(avacincaptad pegol), and
others
|
Scope of the Stargardt Disease Market
Report
- Therapeutic Assessment: Stargardt Disease current
marketed and emerging therapies
- Stargardt Disease Market Dynamics: Key
Market Forecast Assumptions of Emerging Stargardt Disease Drugs and
Market Outlook
- Competitive Intelligence Analysis: SWOT analysis
and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, Stargardt Disease
Market Access and Reimbursement
Discover more about Stargardt disease drugs in development
@ Stargardt Disease Clinical Trials
Table of Contents
1
|
Key Insights
|
2
|
Report
Introduction
|
3
|
Stargardt Disease
Market Overview at a Glance
|
3.1
|
Market Share (%)
Distribution of Therapies in 2025
|
3.2
|
Market Share (%)
Distribution of Therapies in 2034
|
4
|
Executive Summary of
Stargardt Disease
|
5
|
Key Events
|
6
|
Disease Background and
Overview
|
6.1
|
Introduction
|
6.2
|
Cause of Stargardt
Disease
|
6.3
|
Clinical Representation
of Stargardt Disease
|
6.4
|
Pathophysiology of
Stargardt Disease
|
6.5
|
Classification of
Stargardt Disease
|
6.6
|
Diagnosis
|
6.7
|
Treatment
|
7
|
Methodology
|
8
|
Epidemiology and
Patient Population
|
8.1
|
Key Findings
|
8.2
|
Assumptions and
Rationale
|
8.3
|
Diagnosed Prevalent
Cases of Stargardt Disease in the 7MM
|
8.4
|
The United
States
|
8.4.1
|
Diagnosed Prevalent
Cases of Stargardt Disease in the United States
|
8.4.2
|
Onset Age-specific
Cases of Stargardt Disease in the United States
|
8.4.3
|
Type-specific Cases of
Stargardt Disease in the United States
|
8.4.4
|
Symptom-specific Cases
of Symptomatic Stargardt Disease in the United States
|
8.4.5
|
Treated Cases of
Stargardt Disease in the United States
|
8.5
|
EU4 and the
UK
|
8.5.1
|
Diagnosed Prevalent
Cases of Stargardt Disease in EU4 and the UK
|
8.5.2
|
Onset Age-specific
Cases of Stargardt Disease in EU4 and the UK
|
8.5.3
|
Type-specific Cases of
Stargardt Disease in EU4 and the UK
|
8.5.4
|
Symptom-specific Cases
of Symptomatic Stargardt Disease in EU4 and the UK
|
8.5.5
|
Treated Cases of
Stargardt Disease in EU4 and the UK
|
8.6
|
Japan
|
8.6.1
|
Diagnosed Prevalent
Cases of Stargardt Disease in Japan
|
8.6.2
|
Onset Age-specific
Cases of Stargardt Disease in Japan
|
8.6.3
|
Type-specific Cases of
Stargardt Disease in Japan
|
8.6.4
|
Symptom-specific Cases
of Symptomatic Stargardt Disease in Japan
|
8.6.5
|
Treated Cases of
Stargardt Disease in Japan
|
9
|
Patient
Journey
|
10
|
Emerging
Therapies
|
10.1
|
Key Cross of Emerging
Therapies
|
10.2
|
Emixustat: Kubota
Pharmaceuticals
|
10.2.1
|
Product
Description
|
10.2.2
|
Other Developmental
Activities
|
10.2.3
|
Clinical
Development
|
10.2.3
|
Safety and
Efficacy
|
10.3
|
MCO-010: Nanoscope
Therapeutics
|
10.3.1
|
Product
Description
|
10.3.2
|
Other Developmental
Activities
|
10.3.3
|
Clinical
Development
|
10.3.1
|
Safety and
Efficacy
|
10.4
|
ALK-001
(Gildeuretinol): Alkeus Pharmaceuticals
|
10.4.1
|
Product
Description
|
10.4.2
|
Other Developmental
Activities
|
10.4.3
|
Clinical
Development
|
10.4.4
|
Safety and
Efficacy
|
10.5
|
Tinlarebant (LSB-008):
Belite Bio
|
10.5.1
|
Product
Description
|
10.5.2
|
Other Developmental
Activities
|
10.5.3
|
Clinical
Development
|
10.5.4
|
Safety and
Efficacy
|
10.6
|
IZERVAY (avacincaptad
pegol): Astellas Pharma
|
10.6.1
|
Product
Description
|
10.6.2
|
Other Developmental
Activities
|
10.6.3
|
Clinical
Development
|
11
|
Stargardt Disease -
Seven Major Market Analysis
|
11.1
|
Key Findings
|
11.2
|
Market
Outlook
|
11.3
|
Conjoint
Analysis
|
11.4
|
Key Market Forecast
Assumptions
|
11.5
|
Total Market Size of
Stargardt Disease in the 7MM
|
11.6
|
Market Size of
Stargardt Disease by Therapies in the 7MM
|
11.7
|
The United States
Market Size
|
11.7.1
|
Total Market Size of
Stargardt Disease in the United States
|
11.7.2
|
Market Size of
Stargardt Disease by Therapies in the United States
|
11.8
|
EU4 and the UK Market
Size
|
11.8.1
|
Total Market Size of
Stargardt Disease in EU4 and the UK
|
11.8.2
|
Market Size of
Stargardt Disease by Therapies in EU4 and the UK
|
11.9
|
Japan Market
Size
|
11.9.1
|
Total Market Size of
Stargardt Disease in Japan
|
11.9.2
|
Market Size of
Stargardt Disease by Therapies in Japan
|
12
|
KOL Views
|
13
|
Unmet Needs
|
12
|
SWOT
Analysis
|
13
|
Market Access and
Reimbursement
|
13.1
|
The United
States
|
13.1.1
|
Centre for Medicare
& Medicaid Services (CMS)
|
13.2
|
EU4 and the
UK
|
13.2.1
|
Germany
|
13.2.2
|
France
|
13.2.3
|
Italy
|
13.2.4
|
Spain
|
13.2.5
|
United
Kingdom
|
13.3
|
Japan
|
13.3.1
|
MHLW
|
13.4
|
Market Access and
Reimbursement in Stargardt Disease
|
14
|
Appendix
|
14.1
|
Acronyms and
Abbreviations
|
14.2
|
Report
Methodology
|
14.3
|
Bibliography
|
15
|
DelveInsight
Capabilities
|
16
|
Disclaimer
|
17
|
About
DelveInsight
|
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