Some of the key facts of the report
1. The total Cystinosis prevalent population in seven major markets was 2,956 in 2017.
2. Among the European countries, the United Kingdom had the highest prevalent population of Cystinosis with 554 cases, and Italy had the lowest Cystinosis prevalent population, with 105 cases in 2017.
3. The total diagnosed Cystinosis prevalent cases in the 7MM was observed to be 1,308 in 2017.
4. In 2017, there was a total of 630 diagnosed prevalent cases of Cystinosis in the United States.
5. Japan had 24 diagnosed prevalent cases of Cystinosis in 2017.
Key benefits of the report
1. Cystinosis market report covers a descriptive overview and comprehensive insight of the Cystinosis epidemiology and Cystinosis market in the 7 MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
2. Cystinosis market report provides insights on the current and emerging therapies.
3. Cystinosis market report provides a global historical and forecasted market covering drug outreach in 7 MM.
4. Cystinosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Cystinosis market.
“Cystinosis market size in seven major markets (7MM) is estimated to be USD 130.46 million in 2017.”
The United States accounts for the highest Cystinosis market size, in comparison to the other major markets, i.e., EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan. Among the EU5 countries, the UK had the highest market size of Cystinosis, with approximately USD 14.37 million in 2017, while Italy had the smallest market size of Cystinosis.
Cysteamine reacts with lysosomal cysteine to form cysteamine-cysteine mixed disulfide, which exits the lysosome via the lysine cationic transport system and consequently lowers the intracellular cysteine concentrations. The two oral therapies (cysteamine bitartrate) that have been given a green signal in the US and Europe are, Procysbi and Cystagon. Cystagon is an immediate-release form of cysteamine. It was first approved in 1994 in the United States.
Procysbi (cysteamine bitartrate) is a delayed-release capsule indicated for Nephropathic Cystinosis treatment. The FDA and EMA approved Procysbi for the nephropathic Cystinosis treatment in adults and children in 2013.
In 2015, Procysbi got extended approval to treat children 2–6 years of age with nephropathic Cystinosis and later in 2017, the US FDA added one more expansion to cover children aged one year and older living with nephropathic Cystinosis.
There is a lack of standardized guidelines and recommendations for detecting ocular Cystinosis, patient care, and follow-up assessments. In considering the care and follow-up of cystinosis patients, specific challenges need to be addressed.
The launch of the emerging therapies is expected to significantly impact the Cystinosis treatment scenario in the upcoming years:-
1. AVR-RD-04 (CTNS-RD-04)
And many others
The key players in Cystinosis market are:
2. Eloxx Pharmaceuticals
And many others
Table of contents
1. Key Insights
2. Executive Summary of Cystinosis
3. Cystinosis Market Overview at a Glance
4. Cystinosis Disease Background and Overview
5. Cystinosis Epidemiology and Patient Population
6. Country Wise Cystinosis Epidemiology
6.1. United States Cystinosis Epidemiology
6.2. EU5 Epidemiology
6.2.5. United Kingdom
6.3. Japan Epidemiology
7. Cystinosis Treatment and Management
8. Unmet Needs
9. Cystinosis Marketed Drugs
9.1. Procysbi (cysteamine bitartrate): Horizon Pharma
9.2. Cystadrops (cysteamine hydrochloride): Recordati S.p.A.
9.3. Cystaran (cysteamine hydrochloride): Leadiant Biosciences
9.4. Cystagon (cysteamine bitartrate): Mylan Pharmaceuticals
10. Cystinosis Emerging Drugs
10.1. AVR-RD-04 (CTNS-RD-04): Avrobio
10.2. ELX-02: Eloxx Pharmaceuticals
10.3. A0003: Mylan
11. Cystinosis 7 Major Market Analysis
12. Cystinosis Market Outlook 7MM
12.1. United States Cystinosis Market Size
12.2.5. United Kingdom
13. Market Drivers
14. Market Barriers
15. SWOT Analysis
16. Access and Reimbursement
17. KOL Insights
19. DelveInsight Capabilities
21. About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.