Stargardt Disease Market to Grow Rapidly at a CAGR of 32% by 2034 | DelveInsight

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.

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

Related Reports

Stargardt Disease Pipeline

Stargardt Disease Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Stargardt disease companies, including IVERIC bio, Nanoscope Therapeutics, Alkeus Pharmaceuticals, Kubota Vision, Astellas Pharma, Stargazer Pharmaceuticals, Inc., Belite Bio, ProQR Therapeutics, ReVision Therapeutics, among others.

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Stargardt Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Stargardt disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

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SOURCE DelveInsight Business Research, LLP

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