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

September 2019 | 145 pages | ID: C2D3A5DA45BEN
DelveInsight

Abstracts

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DelveInsight’s ‘Cystinuria- Market Insights, Epidemiology, and Market Forecast-2028’ report deliver an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Cystinuria in the United States, EU5 (Germany, Spain, Italy, France and the United Kingdom), and Japan. The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, the current and forecasted market size of Cystinuria from 2017 to 2028 segmented by seven major markets.

Cystinuria is an autosomal recessive disorder of renal tubular and intestinal transport of dibasic amino acids, which results in increased urinary excretion of cystine, and dibasic amino acids, such as ornithine, lysine and arginine (COLA).

The Rare Kidney Stone Consortium has developed a registry and opportunities to lead further efforts in dealing with Cystinuria with current Cystinuria research focused on methods of monitoring disease activity, novel drug therapies and genotype-phenotype studies. Moreover, the launch of third-generation thiol group i.e., Bucillamine will drive the major market after 2021.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2017–2028

Cystinuria - Disease Understanding and Treatment Algorithm

Cystinuria is an autosomal recessive disorder that may include either the presence of cystine stones (as a diagnosis) or increased urinary excretion of dibasic amino acids; along with mutations on both alleles of one of the two genes (SLC3A1; SLC7A9).

The clinical features are related to stone formation and might include nausea, flank pain, hematuria, recurrent urinary tract infections, and rarely acute or chronic renal failure. A variety of other symptoms and occurrences include nausea, emesis, anorexia, and failure to thrive. It can be classified based on phenotypic features (Type 1 and Non-type 1 (type II, type III) and genotypic features (Type A, Type B, Type AB).

Cystinuria Epidemiology

The Cystinuria epidemiology division provides insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders (KOL).

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalence of Cystinuria in 7MM, Gender-Specific Prevalence of Cystinuria in 7MM, Age-Specific Prevalence of Cystinuria, Diagnosed Prevalence of Cystinuria and Type-Specific Diagnosed Prevalence of Cystinuria), scenario of Cystinuria in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017–2028.

According to DelveInsight, the total prevalent cases of Cystinuria (HE) in the 7MM was found to be 85,876 in 2017. The prevalence of Cystinuria in the 7MM is expected to increase during the study period of 2017-2028.

Cystinuria Drug Chapters

This segment of the Cystinuria report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

The current market of the Cystinuria is centered with the Alkalization agents (potassium citrate, acetazolamide), Thiol-binding agents, such as Cuprimine and Tiopronin, which are approved in the US by the FDA. Along with this, Captopril is used for the patients who are intolerant to Cuprimine and Tiopronin. Moreover, surgery (Extracorporeal shock wave lithotripsy, percutaneous nephrolithotomy (PCNL), Ureteroscopic manipulation) is also done in several Cystinuria cases.

Alkalization is an initial treatment approach which helps cystine to dissolve more readily in the urine and may also prevent the formation of stones. Drugs that may be prescribed to make the urine more alkaline include potassium citrate and acetazolamide. This treatment is accompanied by dietary salt restriction. Another treatment approach that can be used to manage Cystinuria include thiol-binding agents (Sulfhydroxyl compounds), such as Cuprimine (d-penicillamine) and Tiopronin (THIOLA). Both these drugs are approved by the FDA for the treatment for cystinuria. They can reduce the formation of cystine crystals by reacting with cystine and generating more soluble mixed disulfide compounds (drug-cysteine complex) and both drugs seem to have the capability of reducing stone recurrences. Recently FDA has approved the improved formulation of Tiopronin, i.e., the enteric-coated formulation of tiopronin (THIOLA EC), which is recommended to be administered with or without food, which overcomes the problem of original formulation Tiopronin.

Detailed chapters of current therapies such as Cuprimine (d-penicillamine) (Bausch Health), Tiopronin (Mission Pharmacal/Retrophin), and emerging therapies, such as ADV7103 (Advicenne), Bucillamine (Revive Therapeutics), Tolvaptan (Otsuka Pharmaceutical) and others have also been provided in the report.

Cystinuria Market Outlook

The Cystinuria market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

According to DelveInsight, the market of Cystinuria in 7MM was found to be approximately USD 625.22 Million in 2017.

Cystinuria market size in the United States accounted for 38% of the total market in 2017. The EU5 led the market in 2017, followed by the US and Japan respectively. Due to the high prevalence of this disease in the region, the Cystinuria treatment market in the EU5 is expected to dominate the market during the forecast period (2019-2028).

Cystinuria Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.

Currently, the market of Cystinuria depend on off-label therapies such as the Alkalization agents (potassium citrate, acetazolamide) approved thiol drugs such as Cuprimine and Tiopronin developed and marketed by Bausch Health and Mission Pharmacal/Retrophin respectively. DelveInsight believes that the current market of Cystinuria will increase with the launch of new therapies during the forecast period. Therapies such as ADV7103 (Advicenne) as Alkalization formulation and Bucillamine (Revive Therapeutics) as Thiol-binding agent, are expected to enter the market by 2028.

Cystinuria Report Insights

• Patient Population of Cystinuria
• Phenotypic Classification
• Genotypic Classification
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Cystinuria Report Key Strengths

• 10 Year Forecast
• 7MM Coverage
• Epidemiology Segmentation
• Drugs Uptake
• Highly Analyzed Market
• Key Cross Competition

Cystinuria 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 Cystinuria market
• Organize sales and marketing efforts by identifying the best opportunities for Cystinuria market
• To understand the future market competition in the Cystinuria market

Contents

1. KEY INSIGHTS

2. CYSTINURIA: MARKET OVERVIEW AT A GLANCE

2.1. Market Share (%) Distribution of Cystinuria in 2017
2.2. Market Share (%) Distribution of Cystinuria in 2028

3. DISEASE OVERVIEW: CYSTINURIA

3.1. Introduction
3.2. Classification
3.3. Clinical features
3.4. Genetics
3.5. Pathophysiology
3.6. Diagnosis

4. EPIDEMIOLOGY AND PATIENT POPULATION

4.1. Key Findings
4.2. 7MM Total Prevalent Patient Population of Cystinuria
4.3. Country Wise-Epidemiology of Cystinuria
4.4. United States
  4.4.1. Assumptions and Rationale
  4.4.2. Prevalent Cases of Cystinuria in the United States
  4.4.3. Gender-Specific Cases of Cystinuria in the United States
  4.4.4. Age-Specific Cases of Cystinuria in the United States
  4.4.5. Diagnosed Cases of Cystinuria in the United States
  4.4.6. Type-Specific Cases of Cystinuria in the United States
4.5. EU5
  4.5.1. Assumptions and Rationale
4.6. Germany
  4.6.1. Prevalent Cases of Cystinuria in Germany
  4.6.2. Gender-Specific Cases of Cystinuria in Germany
  4.6.3. Age-Specific Cases of Cystinuria in Germany
  4.6.4. Diagnosed Cases of Cystinuria in Germany
  4.6.5. Type-Specific Diagnosed Cases of Cystinuria in Germany
4.7. France
  4.7.1. Prevalent Cases of Cystinuria in France
  4.7.2. Gender-Specific Cases of Cystinuria in France
  4.7.3. Age-Specific Cases of Cystinuria in France
  4.7.4. Diagnosed Cases of Cystinuria in France
  4.7.5. Type-Specific Cases of Cystinuria in France
4.8. Italy
  4.8.1. Prevalent Cases of Cystinuria in Italy
  4.8.2. Gender-Specific Cases of Cystinuria in Italy
  4.8.3. Age-Specific Cases of Cystinuria in Italy
  4.8.4. Diagnosed Cases of Cystinuria in Italy
  4.8.5. Type-Specific Cases of Cystinuria in Italy
4.9. Spain
  4.9.1. Prevalent Cases of Cystinuria in Spain
  4.9.2. Gender-Specific Cases of Cystinuria in Spain
  4.9.3. Age-Specific Cases of Cystinuria in Spain
  4.9.4. Diagnosed Cases of Cystinuria in Spain
  4.9.5. Type-Specific Cases of Cystinuria in Spain
4.10. United Kingdom
  4.10.1. Prevalent Cases of Cystinuria in United Kingdom
  4.10.2. Gender-Specific Cases of Cystinuria in the United Kingdom
  4.10.3. Age-Specific Cases of Cystinuria in the United Kingdom
  4.10.4. Diagnosed Cases of Cystinuria in the United Kingdom
  4.10.5. Type-Specific Diagnosed Cases of Cystinuria in the United Kingdom
4.11. Japan
  4.11.1. Assumptions and Rationale
  4.11.2. Prevalent Cases of Cystinuria in Japan
  4.11.3. Gender-Specific Cases of Cystinuria in Japan
  4.11.4. Age-Specific Cases of Cystinuria in Japan
  4.11.5. Diagnosed Cases of Cystinuria in Japan
  4.11.6. Type-Specific Diagnosed Cases of Cystinuria in Japan

5. CURRENT TREATMENT PRACTICES

5.1. The European Association of Urology (EAU) Guidelines

6. UNMET NEEDS

7. MARKETED DRUGS

7.1. Cuprimine (d-penicillamine): Bausch Health
  7.1.1. Product Description
  7.1.2. Regulatory Milestones
  7.1.3. Other Development Activities
  7.1.4. Product Profile
7.2. Tiopronin (Thiola): Mission Pharmacal/ Retrophin
  7.2.1. Drug Description
  7.2.2. Regulatory Milestones
  7.2.3. Other Developmental Activities
  7.2.4. Safety and Efficacy
  7.2.5. Product Profile

8. EMERGING THERAPIES

8.1. Key Cross Competition
8.2. ADV7103: Advicenne
  8.2.1. Drug Description
  8.2.2. Other Developmental Activities
  8.2.3. Clinical Development
  8.2.4. Product Profile
8.3. Bucillamine: Revive Therapeutics
  8.3.1. Drug Description
  8.3.2. Other Developmental Activities
  8.3.3. Clinical Development
  8.3.4. Safety and Efficacy
  8.3.5. Product Profile
8.4. Tolvaptan: Otsuka Pharmaceutical
  8.4.1. Drug Description
  8.4.2. Clinical Development
  8.4.3. Safety and Efficacy
  8.4.4. Product Profile

9. CYSTINURIA: 7 MAJOR MARKET ANALYSIS

9.1. Key Findings
9.2. Market Size of Cystinuria in 7MM

10. MARKET OUTLOOK BY COUNTRY

11. THE UNITED STATES: MARKET OUTLOOK

11.1. United States Market Size
  11.1.1. Total Market Size of Cystinuria
  11.1.2. Market Size of Cystinuria by Therapies

12. EU-5 COUNTRIES: MARKET OUTLOOK

12.1. Germany
  12.1.1. Total Market Size of Cystinuria
  12.1.2. Market Size of Cystinuria by Therapies
12.2. France
  12.2.1. Total Market Size of Cystinuria
  12.2.2. Market Size of Cystinuria by Therapies
12.3. Italy
  12.3.1. Total Market Size of Cystinuria
  12.3.2. Market Size of Cystinuria by Therapies
12.4. Spain
  12.4.1. Total Market Size of Cystinuria
  12.4.2. Market Size of Cystinuria by Therapies
12.5. United Kingdom
  12.5.1. Total Market Size of Cystinuria
  12.5.2. Market Size of Cystinuria by Therapies
12.6. Japan: Market Outlook
  12.6.1. Total Market Size of Cystinuria
  12.6.2. Market Size of Cystinuria by Therapies

13. MARKET DRIVERS

14. MARKET BARRIERS

15. APPENDIX

15.1. Report Methodology

16. DELVEINSIGHT CAPABILITIES

17. DISCLAIMER

18. ABOUT DELVEINSIGHT

List Of Tables

LIST OF TABLES

Table 1: Phenotypic classification of cystinuria based on parental (heterozygote) findings
Table 2: Urinary cystine concentration
Table 3: Genotypic classification of Cystinuria
Table 4: Most common Cystinuria-associated mutations in rBAT*, SLC3A1, b0,+AT‡, and SLC7A9
Table 5: Prevalent Patient Population of Cystinuria in 7MM (2017–2028)
Table 6: Prevalent Cases of Cystinuria in the United States (2017–2028)
Table 7: Gender-Specific Cases of Cystinuria in the United States (2017–2028)
Table 8: Age-Specific Cases of Cystinuria in the United States (2017–2028)
Table 9: Diagnosed Cases of Cystinuria in the United States (2017–2028)
Table 10: Type-Specific Cases of Cystinuria in the United States (2017–2028)
Table 11: Prevalent Cases of Cystinuria in Germany (2017–2028)
Table 12: Gender-Specific Cases of Cystinuria in Germany (2017–2028)
Table 13: Age-Specific Cases of Cystinuria in Germany (2017–2028)
Table 14: Diagnosed Cases of Cystinuria in Germany (2017–2028)
Table 15: Type-Specific Cases of Cystinuria in Germany (2017–2028)
Table 16: Prevalent Cases of Cystinuria in France (2017–2028)
Table 17: Gender-Specific Cases of Cystinuria in France (2017–2028)
Table 18: Age-Specific Cases of Cystinuria in France (2017–2028)
Table 19: Diagnosed Cases of Cystinuria in France (2017–2028)
Table 20: Type-Specific Cases of Cystinuria in France (2017–2028)
Table 21: Prevalent Cases of Cystinuria in Italy (2017–2028)
Table 22: Gender-Specific Cases of Cystinuria in Italy (2017–2028)
Table 23: Age-Specific Cases of Cystinuria in Italy (2017–2028)
Table 24: Diagnosed Cases of Cystinuria in Italy (2017–2028)
Table 25: Type-Specific Cases of Cystinuria in Italy (2017–2028)
Table 26: Prevalent Cases of Cystinuria in Spain (2017–2028)
Table 27: Gender-Specific Cases of Cystinuria in Spain (2017–2028)
Table 28: Age-Specific Cases of Cystinuria in Spain (2017–2028)
Table 29: Diagnosed Cases of Cystinuria in Spain (2017–2028)
Table 30: Type-Specific Cases of Cystinuria in Spain (2017–2028)
Table 31: Prevalent Cases of Cystinuria in the United Kingdom (2017–2028)
Table 32: Gender-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 33: Age-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 34: Diagnosed Cases of Cystinuria in the United Kingdom (2017–2028)
Table 35: Type-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Table 36: Prevalent Cases of Cystinuria in Japan (2017–2028)
Table 37: Gender-Specific Cases of Cystinuria in Japan (2017–2028)
Table 38: Age-Specific Cases of Cystinuria in Japan (2017–2028)
Table 39: Diagnosed Cases of Cystinuria in Japan (2017–2028)
Table 40: Type-Specific Cases of Cystinuria in Japan (2017–2028)
Table 41: Methods to prevent recurrent cystine lithiasis*
Table 42: Dosing of cystine-binding thiol-containing medications (information derived from Micromedex, Lexicomp)
Table 43: Recommended surgical treatment of cystine stones as defined by stone size
Table 44: Grades of Recommendations for the treatment of cystine stones
Table 45: Recommendations for the treatment of cystine stones
Table 46: Key cross competition
Table 47: ADV7103, Clinical Trial Description, 2019
Table 48: Bucillamine, Clinical Trial Description, 2019
Table 49: Tolvaptan, Clinical Trial Description, 2019
Table 50: 7 Major Market Size of Cystinuria in USD Million (2017–2028)
Table 51: United States Market Size of Cystinuria in USD Million (2017–2028)
Table 52: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 53: Germany Market Size of Cystinuria in USD Million (2017–2028)
Table 54: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 55: France Market Size of Cystinuria in USD Million (2017–2028)
Table 56: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 57: Italy, Market Size of Cystinuria in USD Million (2017–2028)
Table 58: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 59: Spain, Market Size of Cystinuria in USD Million (2017–2028)
Table 60: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 61: UK Market Size of Cystinuria in USD Million (2017–2028)
Table 62: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Table 63: Japan, Market Size of Cystinuria in USD Million (2017–2028)
Table 64: Market Size of Cystinuria by Therapies in USD Million (2017–2028)

List Of Figures

LIST OF FIGURES

Figure 1: Cellular localization and function of rBAT (SLC3A1) and b0,+AT (SLC7A9)
Figure 2: Genomic structure and putative function of the encoded protein regions of the Cystinuria genes
Figure 3: Trafficking of cystine and dibasic amino acids in epithelial cells of the renal proximal tubule or small intestine
Figure 4: Diagnostic algorithm of Cystinuria
Figure 5: Prevalent Patient Population of Cystinuria in 7MM (2017–2028)
Figure 6: Prevalent Cases of Cystinuria in the United States (2017–2028)
Figure 7: Gender-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 8: Age-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 9: Diagnosed Cases of Cystinuria in the United States (2017–2028)
Figure 10: Type-Specific Cases of Cystinuria in the United States (2017–2028)
Figure 11: Prevalent Cases of Cystinuria Germany (2017–2028)
Figure 12: Gender-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 13: Age-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 14: Diagnosed Cases of Cystinuria in Germany (2017–2028)
Figure 15: Type-Specific Cases of Cystinuria in Germany (2017–2028)
Figure 16: Prevalent Cases of Cystinuria in France (2017–2028)
Figure 17: Gender-Specific Cases of Cystinuria in France (2017–2028)
Figure 18: Age-Specific Cases of Cystinuria in France (2017–2028)
Figure 19: Diagnosed Cases of Cystinuria in France (2017–2028)
Figure 20: Type-Specific Cases of Cystinuria in France (2017–2028)
Figure 21: Prevalent Cases of Cystinuria in Italy (2017–2028)
Figure 22: Gender-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 23: Age-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 24: Diagnosed Cases of Cystinuria in Italy (2017–2028)
Figure 25: Type-Specific Cases of Cystinuria in Italy (2017–2028)
Figure 26: Prevalent Cases of Cystinuria in Spain (2017–2028)
Figure 27: Gender-Specific Cases of Cystinuria in Spain (2017–2028)
Figure 28: Age-Specific Cases of Cystinuria in Spain (2017–2028)
Figure 29: Diagnosed Cases of Cystinuria in Spain (2017–2028)
Figure 30: Type-Specific of Cystinuria in Spain (2017–2028)
Figure 31: Prevalent Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 32: Gender-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 33: Age-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 34: Diagnosed Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 35: Type-Specific Cases of Cystinuria in the United Kingdom (2017–2028)
Figure 36: Prevalent Cases of Cystinuria in Japan (2017–2028)
Figure 37: Gender-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 38: Age-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 39: Diagnosed Cases of Cystinuria in Japan (2017–2028)
Figure 40: Type-Specific Cases of Cystinuria in Japan (2017–2028)
Figure 41: Treatment Algorithm of Cystinuria
Figure 42: Recommended algorithm of Cystinuria (EAU)
Figure 43: Unmet Needs
Figure 44: 7 Major Market Size of Cystinuria in USD Million (2017–2028)
Figure 45: Market Size of Cystinuria in the United States, USD Millions (2017–2028)
Figure 46: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 47: Market Size of Cystinuria in the Germany, USD Millions (2017–2028)
Figure 48: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 49: Market Size of Cystinuria in the France, USD Millions (2017–2028)
Figure 50: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 51: Market Size of Cystinuria in the Italy, USD Millions (2017–2028)
Figure 52: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 53: Market Size of Cystinuria in the Spain, USD Millions (2017–2028)
Figure 54: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 55: Market Size of Cystinuria in the UK, USD Millions (2017–2028)
Figure 56: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 57: Market Size of Cystinuria in the Japan, USD Millions (2017–2028)
Figure 58: Market Size of Cystinuria by Therapies in USD Million (2017–2028)
Figure 59: Market Drivers
Figure 60: Market Barriers