Indication name: Stargardt Disease
Stargardt Disease – Market outlook,
Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030
(Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan
& China).
Stargardt disease is also called Stargardt macular
dystrophy, juvenile macular degeneration, or fundus flavimaculatus. The disease
causes progressive damage — or degeneration — of the macula, which is a small
area in the center of the retina that is responsible for sharp, straight-ahead
vision. In most cases, Stargardt macular degeneration is caused by mutations in
the ABCA4 gene. Less often, mutations in the ELOVL4 gene cause this condition.
The ABCA4 and ELOVL4 genes provide instructions for making proteins that are
found in light-sensing (photoreceptor) cells in the retina.
Classification-
Fishman STGD Classification
Stage 1:
Pigmentary changes in the macula or
pisciform ring of flecks within 1 DD on all sides of the fovea.
ERG and EOG are normal
Stage 2:
Pisciform flecks present beyond 1
DD of the fovea, often extending beyond the arcades and nasal to the optic disc
ERG and EOG normal, but cone/rod
response may be subnormal
Stage 3:
Diffusely resorbed flecks and
choriocapillaris atrophy of the macula
EOG subnormal
ERG subnormal cone or rod amplitudes
Central field defects +
midperipheral/peripheral field defect
Stage 4:
Diffusely absorbed flecks and
choriocapillaris/RPE atrophy throughout
the entire fundus
ERG with reduced rod and cone
amplitudes
Peripheral fields with moderate to
extensive restriction
Epidemiology- Stargardt macular
degeneration is the most common form of juvenile macular degeneration, the
signs and symptoms of which begin in childhood. A prospective epidemiologic
study (Cornish et. al 2017) undertaken under the auspices of the British
Ophthalmological Surveillance Unit (BOSU) suggests an annual incidence in the
United Kingdom of between 0.110 and 0.128 per 100 000 individuals. Thelansis
estimated prevalence of Stargardt macular degeneration is 1 in 8,000 to 10,000
individuals.
Competitive landscape of Stargardt
Disease includes country specific approved as well as pipeline therapies. Any
asset/ product specific designation or review and Accelerated Approval are
being tracked and supplemented with analyst commentary.
KOLs insights of Stargardt Disease
across 8 MM market from center of Excellence/ Public/ Private hospitals
participated in the study. Insights around current treatment landscape,
epidemiology, clinical characteristics, future treatment paradigm and Unmet
needs.
Stargardt Disease Market Forecast:
Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data
Inputs with sourcing, Market Event and Product Event, Country specific Forecast
Model, Market uptake and patient share uptake, Attribute Analysis, Analog
Analysis, Disease burden and pricing scenario, Summary and Insights.
S. No Asset Company Partner Phase
1
ACU-4429 Kubota
Pharmaceutical Holdings Co., Ltd.
N/A III
2
Zimura Iveric Bio Archemix IIb
3
ALK-001 Alkeus
Pharmaceuticals, Inc. N/A II
4
ASP7317 Astellas Pharma,
Inc. CHA Bio & Diostech II
5
LBS-008 Lin Bioscience,
Inc. N/A I
6
NSR-ABCA4 Biogen, Inc. N/A Preclinical
7
QRX-1011 ProQR Therapeutics
N.V. N/A Preclinical
8
REV-0100 reVision
Therapeutics, Inc. Cornell
University Preclinical
9
Stargardt Program Applied
Genetic Technologies Corporation
N/A Preclinical
10 STGD1 Minigene Program (IVERIC/UMass) Iveric Bio N/A Preclinical
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