Stargardt Disease Market

Market Overview:

The 7 major Stargardt disease markets reached a value of USD 33.2 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 316 Million by 2035, exhibiting a growth rate (CAGR) of 22.74% during 2025-2035.

The Stargardt disease market has been comprehensively analyzed in IMARC's new report titled "Stargardt Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Stargardt disease refers to an inherited single-gene disorder that affects the retina, which is the part of the eye that detects light and sends visual information to the brain. The ailment is characterized by macular degeneration that begins in childhood, adolescence, or adulthood, causing progressive loss of vision. The primary symptom of the disease is a decrease in visual acuity, which cannot be corrected with glasses. This manifests as a diminished ability to see fine details while viewing or reading distant objects. Various other common indications associated with Stargardt disease include wavy vision, blurriness, blind spots, loss of depth perception, impaired color vision, sensitivity to glare, difficulty adapting to dim lighting, etc. The diagnosis of the condition is mainly made through clinical features, medical history, and an eye examination via slit lamp. The healthcare provider may also perform genetic testing to ensure the presence of genome abnormalities within the body. Numerous diagnostic tools, such as spectral-domain optical coherence tomography, microperimetry, electroretinography, etc., are utilized in patients to confirm a diagnosis and rule out other possible causes.

The increasing prevalence of genetic disorders caused by mutations in one of several genes involved in the processing of vitamin A in the retina is primarily driving the Stargardt disease market. In addition to this, the widespread adoption of intravitreal vasodilators, including dobesilate, that inhibit overexpression of fibroblast growth factor and improve visual acuity in patients is also bolstering the market growth. Furthermore, the inflating application of fluorescein angiography for diagnosing various ocular pathologies by assessing the anatomy and physiology of retinal and choroidal circulation is acting as another significant growth-inducing factor. Apart from this, the escalating demand for vision rehabilitation therapy to treat the ailment, since it can maximize visual functioning and improve the quality of life for patients, is further creating a positive outlook for the market. Moreover, the emerging popularity of gene replacement therapy, which works by replacing the incorrect gene variant with the correct version, thereby enhancing the body's ability to fight the condition, is expected to drive the Stargardt disease market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Stargardt disease market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Stargardt disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Stargardt disease market in any manner.

Recent Developments:

• In November 2024, Ocugen, Inc announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). 
• In June 2024, Belite Bio, Inc. reported that its lead pipeline, Tinlarebant, received Sakigake Designation by the Ministry of Health, Labour, and Welfare in Japan (MHLW) for the treatment of Stargardt disease.
• In May 2024, Ocugen, Inc. stated that dosing had been completed in the second cohort of its Phase 1/2 GARDian clinical study for OCU410ST (AAV-hRORA), a modifier gene therapy candidate being studied for Stargardt disease as a one-time treatment for life.
• In April 2024, SalioGen Therapeutics disclosed the nomination of SGT-1001, a development candidate for the management of Stargardt disease. SGT-1001 is being developed as a one-time, non-viral therapy to reduce or prevent the gradual loss of central vision in persons with Stargardt disease, regardless of mutation type.
• In January 2024, Ascidian Therapeutics stated that the US Food and Drug Administration (FDA) had approved its investigational new drug (IND) application and granted ACDN-01 Fast Track Designation. ACDN-01 is the first clinical-stage RNA exon editor and the only treatment that addresses the genetic etiology of Stargardt disease.

Key Highlights:

• Stargardt disease is the most prevalent inherited childhood and adult maculopathy.
• It accounts for up to 7% of all retinopathies, with an incidence of one in every 8000-10,000 persons.
• Stargardt Disease affects around 30,000 persons in the United States.
• It is a prevalent cause of central vision loss in those under the age of 50, with start usually between the ages of 10 and 20.

Drugs:

Tinlarebant is a novel oral medication that aims to minimize the buildup of vitamin A-based toxins (bisretinoids) that cause retinal degeneration in Stargardt disease. Tinlarebant reduces and maintains serum retinol binding protein 4 (RBP4), the only protein that transports retinol from the liver to the eye. Tinlarebant inhibits the synthesis of bisretinoids by regulating the quantity of retinol that enters the eye.

Emixustat hydrochloride (ACU-4429) is being developed to treat juvenile macular degeneration (Stargardt disease). It is taken orally in the form of a tablet and works by targeting RPE65. The medication candidate is a non-retinoid visual cycle modulator (VCM) being developed using VCM technology.

Izervay (Avacincaptad pegol) is a novel complement inhibitor used for the treatment of Stargardt disease. It is intended to act by blocking the C5 protein, which is a component of the complement system.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Stargardt disease market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Stargardt disease market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Stargardt disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Stargardt disease market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the Stargardt disease market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the Stargardt disease market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2019-2035) of Stargardt disease across the seven major markets?
• What is the number of prevalent cases (2019-2035) of Stargardt disease by age across the seven major markets?
• What is the number of prevalent cases (2019-2035) of Stargardt disease by gender across the seven major markets?
• How many patients are diagnosed (2019-2035) with Stargardt disease across the seven major markets?
• What is the size of the Stargardt disease patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of Stargardt disease?
• What will be the growth rate of patients across the seven major markets?
   

Stargardt Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Stargardt disease drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Stargardt disease market?
• What are the key regulatory events related to the Stargardt disease market?
• What is the structure of clinical trial landscape by status related to the Stargardt disease market?
• What is the structure of clinical trial landscape by phase related to the Stargardt disease market?
• What is the structure of clinical trial landscape by route of administration related to the Stargardt disease market?