Cystinosis - Market Insights, Epidemiology, and Market Forecast-2028

DelveInsight’s ‘Cystinosis - Market Insights, Epidemiology, and Market Forecast-2028’ report deliver an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of Cystinosis in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
Cystinosis is a rare genetic, metabolic, lysosomal storage disease caused by mutations in the CTNS gene on chromosome 17p13. The condition results in an abnormal accumulation of the amino acid cystine in various organs and tissues of the body, such as the kidneys, eyes, muscles, pancreas, and brain. There are three types of cystinosis, depending on when symptoms first appear (known as onset): Infantile (early-onset) cystinosis; later childhood or adolescent (late-onset) cystinosis; and adult cystinosis. About 95% of patients with cystinosis have the infantile/early-onset form, making it the most common variant of this disease, and most patients develop kidney problems. In many cases, the categorization is also done as, nephropathic cystinosis, intermediate cystinosis, and non-nephropathic (or ocular) cystinosis.
According to DelveInsight’s, it was observed that most of the Cystinosis cases are contributed by nephropathic cystinosis, and least is contributed by ocular type.
Geography Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2017–2028
Cystinosis–Disease Understanding
Cystinosis is not something that a person can catch from another individual. It is a genetic condition, which means a child is born with it. The age of onset, symptoms, and severity of cystinosis can vary greatly from one person to another. Nephropathic cystinosis presents in infancy and is the most common and severe form. Over a period of years, cystine damages various organs, including the kidneys, liver, muscles, white blood cells, eyes, and central nervous system. The cystine crystallizes in cells throughout the body, slowly destroying the organs. The earliest abnormalities are seen in the kidney.
Cystinosis Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalent Cases of Cystinosis in the 7MM, Total Diagnosed Prevalent Cases of Cystinosis in the 7MM, and Type-specific Diagnosed Prevalent Cases of Cystinosis in the 7MM) scenario of Cystinosis in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017–2028.
According to DelveInsight’s, the total prevalent population of Cystinosis in seven major markets is 2,956 in 2017. These cases are expected to increase with a significant CAGR during the study period (2017–2028).
The total diagnosed prevalent population of Cystinosis in seven major markets was 1,308 in 2017.
Cystinosis Drug Chapters
The mainstay of cystinosis treatment is the cystine-depleting aminothiol cysteamine or mercaptoethylamine. The two oral therapies (cysteamine bitartrate) that are approved in the US and Europe are, Procysbi and Cystagon. In 1994, Cystagon was introduced, and this is currently the most widely used preparation of cysteamine. Later in 1997, the drug got approval in Europe, and in 2014 got approved in Japan. Procysbi (cysteamine bitartrate) is a delayed-release capsule indicated for the treatment of nephropathic cystinosis. The drug was discovered and developed by Raptor Pharmaceutical (Raptor). In the year 2013, the FDA and EMA approved Procysbi for the treatment of nephropathic cystinosis in adults and children. Additionally, there are two therapies (Cystadrops and Cystaran) that are marketed for ocular type. Cystadrops is marketed in Europe and Cystaran is marketed in the US. Recently, in 2019, Recordati has filed NDA for Cystadrops in the US. This therapy already got approval in Europe in the year 2017, for the treatment of ocular cystinosis. With the expected launch of Cystadrops in the US, cystaran may face a tough competition.
Due to disease complexity, not many companies are working in this therapeutic area. The few companies that are focusing on this target are Avrobio, Eloxx Pharmaceuticals, and Mylan. AVR-RD-04 is a lentiviral-based gene therapy being developed by Avrobio for Cystinosis patients. Since it’s a gene therapy—a one-time treatment—it is the potential to provide a permanent cure for the disease. Hence it will be a major factor for attracting the patient pool. AVR-RD-04 will face a tough competition with ELX-02 and other approved therapies and generate a major market share. The present treatment option for cystinosis has cystine depleting therapies only, whereas, ELX-02 represents a different mechanism of action (Ribosomal protein modulators) and hence it is expected that it will give a tough competition to procysbi and cystagon.
Cystinosis Market Outlook
According to DelveInsight, the total market size of Cystinosis in seven major markets was USD 130.46 million in 2017. The market size of Cystinosis is expected to increase at a significant CAGR during the study period (2017–2028).
Among all the seven major markets, the United States accounts for the highest market size of Cystinosis. In 2017, the market size of Cystinosis in the US was found to be USD 86.52 million.
Cystinosis Drugs Uptake
The current market size of Cystinosis is mainly attributed by procysbi and cystagon that are marketed for the treatment of nephropathic cystinosis; cystaran and cystadrops that is marketed for ocular cystinosis. There are several other off-labeled therapies as well that help in the management of cystinosis that includes the use of hormonal therapy and renal cell transplantation. The market size is expected to increase during the forecast period (2019–2028), due to potential therapies AVR-RD-04, ELX-02 and A0003.
Cystinosis Report Insights
• Patient Population by total prevalent cases of Cystinosis
• Total Diagnosed prevalent cases of Cystinosis
• Type-specific prevalent cases of Cystinosis
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies
Cystinosis Report Key Strengths
• 10 Year Forecast
• 7MM Coverage
• Epidemiology Segmentation
• Drugs Uptake
• Highly Analyzed Market
• Key Cross Competition
Cystinosis Report Assessment
• Current Treatment Practices
• Unmet Needs
• Detailed Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
Key Benefits
• This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving the Cystinosis market
• Organize sales and marketing efforts by identifying the best opportunities for Cystinosis market
• To understand the future market competition in the Cystinosis market.