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

  • ID: 4833460
  • Drug Pipelines
  • September 2019
  • Region: Global
  • 145 pages
  • DelveInsight
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FEATURED COMPANIES

‘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 the publisher, 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 the publisher, 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. The publisher 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 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
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

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. Publisher Capabilities

17. Disclaimer

18. About the Publisher
Note: Product cover images may vary from those shown
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  • Advicenne: ADV7103
  • Revive Therapeutics: Bucillamine
  • Otsuka Pharmaceutical: Tolvaptan
  • Bausch Health: Cuprimine (d-penicillamine)
  • Mission Pharmacal/ Retrophin: Tiopronin (Thiola)
Note: Product cover images may vary from those shown
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