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

  • ID: 4911965
  • Drug Pipelines
  • January 2020
  • Region: Global
  • 149 pages
  • DelveInsight
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FEATURED COMPANIES

  • Avrobio
  • Eloxx Pharmaceuticals
  • Leadiant Biosciences
  • Mylan EPD
  • Recordati S.p.A.
  • MORE
The ‘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 this research, 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 this research, 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 this research, 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 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.
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Avrobio
  • Eloxx Pharmaceuticals
  • Leadiant Biosciences
  • Mylan EPD
  • Recordati S.p.A.
  • MORE
1. Key Insights

2. Cystinosis Market Overview at a Glance
2.1. Market Share (%) Distribution of Cystinosis in 2017
2.2. Market Share (%) Distribution of Cystinosis in 2028

3. Cystinosis: Disease Background and Overview
3.1. Introduction
3.2. Classification of Cystinosis
3.2.1. Nephropathic cystinosis
3.2.2. Intermediate cystinosis/Late-Onset/Juvenile cystinosis
3.2.3. Non-nephropathic or ocular cystinosis
3.3. Cause of Cystinosis
3.4. Pathophysiology of Cystinosis
3.4.1. Cellular events in cystinosis related to cystine accumulation
3.4.2. Cellular events in cystinosis likely unrelated to cystine accumulation
3.5. Clinical description and complications
3.6. Genetic Basis of Cystinosis
3.7. Functional Characteristics of CTNS Mutations: Genotype-Phenotype Correlations
3.8. Diagnostics and Biomarker Follow-up
3.8.1. Differential Diagnosis and Biomarker Follow-up

4. Epidemiology and Patient Population
4.1. Key Findings
4.2. 7MM Total Prevalent Patient Population of Cystinosis
4.3. 7MM Total Diagnosed Prevalent Patient Population of Cystinosis

5. Country Wise-Epidemiology of Cystinosis
5.1. United States Epidemiology
5.1.1. Assumptions and Rationale
5.1.2. Total Prevalent Cases of Cystinosis in the United States
5.1.3. Total Diagnosed Prevalent Cases of Cystinosis in the United States
5.1.4. Type-Specific Diagnosed Prevalence of Cystinosis in the United States
5.2. EU5 Epidemiology
5.2.1. Germany
5.2.1.1. Assumptions and Rationale
5.2.1.2. Total Prevalent Cases of Cystinosis in Germany
5.2.1.3. Total Diagnosed Prevalent Cases of Cystinosis in Germany
5.2.1.4. Type-Specific Diagnosed Prevalence of Cystinosis in Germany
5.2.2. France
5.2.2.1. Assumptions and Rationale
5.2.2.2. Total Prevalent Cases of Cystinosis in France
5.2.2.3. Total Diagnosed Prevalent Cases of Cystinosis in France
5.2.2.4. Type-Specific Diagnosed Prevalence of Cystinosis in France
5.2.3. Italy
5.2.3.1. Assumptions and Rationale
5.2.3.2. Total Prevalent Cases of Cystinosis in Italy
5.2.3.3. Total Diagnosed Prevalent Cases of Cystinosis in Italy
5.2.3.4. Type-Specific Diagnosed Prevalence of Cystinosis in Italy
5.2.4. Spain
5.2.4.1. Assumptions and Rationale
5.2.4.2. Total Prevalent Cases of Cystinosis in Spain
5.2.4.3. Total Diagnosed Prevalent Cases of Cystinosis in Spain
5.2.4.4. Type-Specific Diagnosed Prevalence of Cystinosis in Spain
5.2.5. United Kingdom
5.2.5.1. Assumptions and Rationale
5.2.5.2. Total Prevalent Cases of Cystinosis in the United Kingdom
5.2.5.3. Total Diagnosed Prevalent Cases of Cystinosis in the United Kingdom
5.2.5.4. Type-Specific Diagnosed Prevalence of Cystinosis in the United Kingdom
5.3. Japan Epidemiology
5.3.1. Assumptions and Rationale
5.3.2. Total Prevalent Cases of Cystinosis in Japan
5.3.3. Total Diagnosed Prevalent Cases of Cystinosis in Japan
5.3.4. Type-Specific Diagnosed Prevalence of Cystinosis in Japan

6. Treatment and Management of Cystinosis
6.1. Symptomatic treatment
6.2. Cystine-depleting therapy
6.3. Therapeutic monitoring and Prognosis
6.4. Treatment Guidelines for Cystinosis
6.4.1. The European Reference Network for Rare Kidney Diseases

7. Unmet Needs

8. Marketed Drugs
8.1. Procysbi (cysteamine bitartrate): Horizon Pharma
8.1.1. Drug Description
8.1.2. Regulatory Milestones
8.1.3. Other Development Activities
8.1.4. Advantages and Disadvantages
8.1.5. Clinical Development
8.1.6. Clinical Trials Information
8.1.7. Safety and Efficacy
8.1.8. Product Profile
8.2. Cystadrops (cysteamine hydrochloride): Recordati S.p.A.
8.2.1. Drug Description
8.2.2. Regulatory Milestones
8.2.3. Other Development Activities
8.2.4. Advantages and Disadvantages
8.2.5. Clinical Development
8.2.6. Clinical Trials Information
8.2.7. Safety and Efficacy
8.2.8. Product Profile
8.3. Cystaran (cysteamine hydrochloride): Leadiant Biosciences
8.3.1. Drug Description
8.3.2. Regulatory Milestones
8.3.3. Other Development Activities
8.3.4. Advantages and Disadvantages
8.3.5. Safety and Efficacy
8.3.6. Product Profile
8.4. Cystagon (cysteamine bitartrate): Mylan Pharmaceuticals
8.4.1. Drug Description
8.4.2. Regulatory Milestones
8.4.3. Other Development Activities
8.4.4. Clinical Trial Information
8.4.5. Advantages and Disadvantages
8.4.6. Safety and Efficacy
8.4.7. Product Profile

9. Emerging Drugs
9.1. AVR-RD-04 (CTNS-RD-04): Avrobio
9.1.1. Product Description
9.1.2. Other Developmental Activities
9.1.3. Clinical Development
9.1.4. Clinical trial information
9.1.5. Safety and Efficacy
9.1.6. Product Profile
9.2. ELX-02: Eloxx Pharmaceuticals
9.2.1. Product Description
9.2.2. Other Developmental Activities
9.2.3. Clinical Development
9.2.4. Clinical trial information
9.2.5. Safety and Efficacy
9.2.6. Product Profile
9.3. A0003: Mylan
9.3.1. Product Description
9.3.2. Other Developmental Activities
9.3.3. Clinical Development
9.3.4. Product Profile

10. Cystinosis: 7 Major Market Analysis
10.1.1. Key Findings
10.1.2. Market Size of Cystinosis in the 7MM
10.1.3. Market Size of Cystinosis by Therapies in the 7MM

11. Market Outlook: 7MM
11.1. United States Market Size
11.1.1. The total Market size of Cystinosis
11.1.2. Cystinosis Market by Current Treatment and Emerging Therapies
11.2. EU5
11.2.1. Germany
11.2.1.1. Total Market size of Cystinosis
11.2.1.2. Cystinosis Market by Current Treatment and Emerging Therapies
11.2.2. France
11.2.2.1. Total Market size of Cystinosis
11.2.2.2. Cystinosis Market by Current Treatment & Emerging Therapies
11.2.3. Italy
11.2.3.1. Total Market size of Cystinosis
11.2.3.2. Cystinosis Market by Current Treatment & Emerging Therapies
11.2.4. Spain
11.2.4.1. Total Market size of Cystinosis
11.2.4.2. Cystinosis Market by Current Treatment and Emerging Therapies
11.2.5. United Kingdom
11.2.5.1. Total Market size of Cystinosis
11.2.5.2. Cystinosis Market by Current Treatment and Emerging Therapies
11.3. Japan
11.3.1. Total Market size of Cystinosis
11.3.2. Cystinosis Market by Current Treatment & Emerging Therapies

12. Market Drivers

13. Market Barriers

14. SWOT Analysis

15. KOL Insights

16. Appendix
16.1. Bibliography
16.2. Report Methodology

17. Researcher Capabilities

18. Disclaimer

19. About the Publisher

List of Tables:
Table 1 CTNS mutations and their corresponding phenotype, cystine transport activity, and location
Table 2 Total Prevalent Patient Population of Cystinosis in the 7MM (2017-2028)
Table 3 Total Diagnosed Prevalent Patient Population of Cystinosis in the 7MM (2017-2028)
Table 4 Total Prevalent Cases of Cystinosis in the United States (2017-2028)
Table 5 Total Diagnosed Prevalent Cases of Cystinosis in the United States (2017-2028)
Table 6 Type-Specific Diagnosed Prevalence of Cystinosis in the United States (2017-2028)
Table 7 Total Prevalent Cases of Cystinosis in Germany (2017-2028)
Table 8 Total Diagnosed Prevalent Cases of Cystinosis in Germany (2017-2028)
Table 9 Type-Specific Diagnosed Prevalence of Cystinosis in Germany (2017-2028)
Table 10 Total Prevalent Cases of Cystinosis in France (2017-2028)
Table 11 Total Diagnosed Prevalent Cases of Cystinosis in France (2017-2028)
Table 12 Type-Specific Diagnosed Prevalence of Cystinosis in France (2017-2028)
Table 13 Total Prevalent Cases of Cystinosis in Italy (2017-2028)
Table 14 Total Diagnosed Prevalent Cases of Cystinosis in Italy (2017-2028)
Table 15 Type-Specific Diagnosed Prevalence of Cystinosis in Italy (2017-2028)
Table 16 Total Prevalent Cases of Cystinosis in Spain (2017-2028)
Table 17 Total Diagnosed Prevalent Cases of Cystinosis in Spain (2017-2028)
Table 18 Type-Specific Diagnosed Prevalence of Cystinosis in Spain (2017-2028)
Table 19 Total Prevalent Cases of Cystinosis in the United Kingdom (2017-2028)
Table 20 Total Diagnosed Prevalent Cases of Cystinosis in the United Kingdom (2017-2028)
Table 21 Type-Specific Diagnosed Prevalence of Cystinosis in the UK (2017-2028)
Table 22 Total Prevalent Cases of Cystinosis in Japan (2017-2028)
Table 23 Total Diagnosed Prevalent Cases of Cystinosis in Japan (2017-2028)
Table 24 Type-Specific Diagnosed Prevalence of Cystinosis in Japan (2017-2028)
Table 25 Procysbi, Clinical Trial Description, 2019
Table 26 Cystadrops, Clinical Trial Description, 2019
Table 27 Market size of Cystinosis by Region in USD Million (2017–2028)
Table 28 7MM Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 29 United States Market Size of Cystinosis in USD Million (2017-2028)
Table 30 The US Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 31 Germany Market Size of Cystinosis in USD Million (2017-2028)
Table 32 Germany Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 33 France Market Size of Cystinosis in USD Million (2017-2028)
Table 34 France Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 35 Italy Market Size of Cystinosis in USD Million (2017-2028)
Table 36 Italy Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 37 Spain Market Size of Cystinosis in USD Million (2017-2028)
Table 38 Spain Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 39 United Kingdom Market Size of Cystinosis in USD Million (2017-2028)
Table 40 The UK Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Table 41 Japan Market Size of Cystinosis in USD Million (2017-2028)
Table 42 Japan Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)

List of Figures:
Figure 1 Chances of a carrier couple having a child with cystinosis
Figure 2 Classification of Cystinosis
Figure 3 Symptoms of different types of Cystinosis
Figure 4 Schematic representation of the CTNS gene and all reported mutations in cystinosis
Figure 5 Simplified scheme of the current knowledge on the pathogenesis of cystinosis
Figure 6 Pathophysiology of cystinosis
Figure 7 Structure of cystinosin with the indication of CTNS mutations having an effect on LAMP2A localization, interaction with V-ATPase-Regulator-Rag complex and glycosylation status.
Figure 8 Total Prevalent Patient Population of Cystinosis in the 7MM (2017-2028)
Figure 9 Total Diagnosed Prevalent Patient Population of Cystinosis in the 7MM (2017-2028)
Figure 10 Total Prevalent Cases of Cystinosis in the United States (2017-2028)
Figure 11 Total Diagnosed Prevalent Cases of Cystinosis in the United States (2017-2028)
Figure 12 Type-Specific Diagnosed Prevalence of Cystinosis in the United States (2017-2028)
Figure 13 Total Prevalent Cases of Cystinosis in Germany (2017-2028)
Figure 14 Total Diagnosed Prevalent Cases of Cystinosis in Germany (2017-2028)
Figure 15 Type-Specific Diagnosed Prevalence of Cystinosis in Germany (2017-2028)
Figure 16 Total Prevalent Cases of Cystinosis in France (2017-2028)
Figure 17 Total Diagnosed Prevalent Cases of Cystinosis in France (2017-2028)
Figure 18 Type-Specific Diagnosed Prevalence of Cystinosis in France (2017-2028)
Figure 19 Total Prevalent Cases of Cystinosis in Italy (2017-2028)
Figure 20 Total Diagnosed Prevalent Cases of Cystinosis in Italy (2017-2028)
Figure 21 Type-Specific Diagnosed Prevalence of Cystinosis in Italy (2017-2028)
Figure 22 Total Prevalent Cases of Cystinosis in Spain (2017-2028)
Figure 23 Total Diagnosed Prevalent Cases of Cystinosis in Spain (2017-2028)
Figure 24 Type-Specific Diagnosed Prevalence of Cystinosis in Spain (2017-2028)
Figure 25 Total Prevalent Cases of Cystinosis in the United Kingdom (2017-2028)
Figure 26 Total Diagnosed Prevalent Cases of Cystinosis in the United Kingdom (2017-2028)
Figure 27 Type-Specific Diagnosed Prevalence of Cystinosis in the United Kingdom (2017-2028)
Figure 28 Total Prevalent Cases of Cystinosis in Japan (2017-2028)
Figure 29 Total Diagnosed Prevalent Cases of Cystinosis in Japan (2017-2028)
Figure 30 Type-Specific Diagnosed Prevalence of Cystinosis in Japan (2017-2028)
Figure 31 Unmet Needs of Cystinosis
Figure 32 Market size of Cystinosis by Region in USD Million (2017–2028)
Figure 33 Market Size of Cystinosis by Therapies in the 7MM, in USD Million (2017–2028)
Figure 34 United States Market Size of Cystinosis in USD Million (2017-2028)
Figure 35 The US Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 36 Germany Market Size of Cystinosis in USD Million (2017-2028)
Figure 37 Germany Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 38 France Market Size of Cystinosis in USD Million (2017-2028)
Figure 39 France Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 40 Italy Market Size of Cystinosis in USD Million (2017-2028)
Figure 41 Italy Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 42 Spain Market Size of Cystinosis in USD Million (2017-2028)
Figure 43 Spain Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 44 United Kingdom Market Size of Cystinosis in USD Million (2017-2028)
Figure 45 The UK Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 46 Japan Market Size of Cystinosis in USD Million (2017-2028)
Figure 47 Japan Market Size of Cystinosis by Current Treatment and Emerging Therapies in USD Million (2017-2028)
Figure 48 Market Drivers
Figure 49 Market Barriers
Figure 50 SWOT analysis
Note: Product cover images may vary from those shown
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  • Horizon Pharma/Raptor Pharmaceuticals
  • Mylan Pharmaceuticals/Recordati
  • Leadiant Biosciences
  • Recordati S.p.A.
  • Avrobio
  • Eloxx Pharmaceuticals
  • Mylan EPD
Note: Product cover images may vary from those shown
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