Cystinosis Market

Market Overview:

The 7 major cystinosis markets reached a value of USD 198.8 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 324.9 Million by 2035, exhibiting a growth rate (CAGR) of 4.58% during 2025-2035.

The cystinosis market has been comprehensively analyzed in IMARC's new report titled "Cystinosis Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Cystinosis is a rare, multisystem hereditary illness characterized by the accumulation of cystine, an amino acid, in several human tissues and organs such as the kidneys, eyes, muscles, liver, pancreas, and brain. The kidneys and eyes are the two organs most damaged. Cystinosis is classified into three types: nephropathic cystinosis, intermediate cystinosis, and non-nephropathic (or ocular) cystinosis. The age of onset, symptoms, and severity of this ailment might vary widely from person to person. Some common indications of the disorder include excessive thirst, electrolyte imbalances, vomiting, dehydration, fever, growth delay, failure to thrive, scarred or cloudy corneas, light sensitivity, vision loss, difficulty swallowing, etc. Diagnosing cystinosis commonly involves a comprehensive evaluation of characteristic features, blood tests, and physical examination. The healthcare provider may also utilize urinalysis to determine the excess loss of minerals, nutrients, electrolytes and amino acids. Furthermore, tandem mass spectrometry technique might be performed to accurately measure cystine levels in white blood cells. In some cases, genetic testing may be recommended to confirm a diagnosis in patients.

The escalating prevalence of mutations in the CTNS gene, which can lead to a deficiency in the protein cystinosin, resulting in cystine buildup and crystal formation, is primarily driving the cystinosis market. In addition to this, the inflating utilization of cystine-depleting agents, like cysteamine, since they work by interacting with cystine within lysosomes and converting it into a form that can be excreted, thereby effectively lowering cystine levels, is also creating a positive outlook for the market. Moreover, the widespread adoption of renal replacement therapy on account of its several advantages, including restored normal kidney function, improved calcium-phosphate balance, and reduced risk of renal osteodystrophy, is further bolstering the market growth. Apart from this, the rising usage of Nissen fundoplication to help maintain nutrient absorption, improving overall growth and health, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of gene therapy that aims to replace the faulty CTNS gene with a functional copy, restoring the ability to transport cystine out of lysosomes, is expected to drive the cystinosis market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the cystinosis market in the United States, EU5 (Germany, Spain, Italy, France, and 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 cystinosis and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and 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 cystinosis market in any manner.

Drugs:

• Procysbi (cysteamine bitartrate) is used to treat nephropathic cystinosis in adults and children aged 2 years or older. Cysteamine is an aminothiol that transforms cystine into cysteine and cysteine-cysteamine mixed disulfides, minimizing corneal cystine crystals' formation. This medication participates in a thiol-disulfide exchange interaction with lysosomes, leading to cysteine departure from the lysosome in patients diagnosed with cystinosis.
• Cystaran (cysteamine ophthalmic solution) is a cystine-depleting drug used to treat corneal cystine crystal accumulation in cystinosis patients. Cysteamine, the active ingredient in Cystaran, is an aminothiol that depletes lysosomal cystine, inhibiting the formation of cystine crystals in physiological tissues. Within lysosomes, cysteamine reacts with cystine to create cysteine and cysteine-cysteamine mixed disulfide. These compounds pass through the lysosomal membrane and are removed from the cell.
• Cystadrops (cysteamine ophthalmic solution) 0.37% is a prescription medication used to treat corneal cystine crystal deposits in the eye in adults and children with cystinosis. It reduces cystine crystal accumulation in the cornea of cystinosis patients. The drug enters lysosomes, breaks cystine disulfide bonds, and converts cystine into cysteine and a cysteine-cysteamine mixed disulfide, which can exit the lysosome, preventing corneal damage and improving ocular health.
• NPI-001 is a glutathione mimic that neutralizes reactive oxygen species (ROS) and protects against oxidative damage induced by cystine buildup. It protects organs from degeneration by restoring cellular redox balance, which is especially important in the eyes and kidneys, where oxidative stress accelerates disease progression. NPI-001 aims to retain organ function while supplementing existing cystine-depleting therapy.

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

Competitive Landscape:

This report also provides a detailed analysis of the current cystinosis 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 cystinosis 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 cystinosis market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the cystinosis 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 cystinosis across the seven major markets?
• What is the number of prevalent cases (2019-2035) of cystinosis by age across the seven major markets?
• What is the number of prevalent cases (2019-2035) of cystinosis by gender across the seven major markets?
• How many patients are diagnosed (2019-2035) with cystinosis across the seven major markets?
• What is the size of the cystinosis 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 cystinosis?
• What will be the growth rate of patients across the seven major markets?

Cystinosis: 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 cystinosis 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 cystinosis market?
• What are the key regulatory events related to the cystinosis market?
• What is the structure of clinical trial landscape by status related to the cystinosis market?
• What is the structure of clinical trial landscape by phase related to the cystinosis market?
• What is the structure of clinical trial landscape by route of administration related to the cystinosis market?