Cystinuria Market Report and Forecast 2025-2034
Description
The cystinuria market size was valued at USD 907.19 Million in 2024, driven by increasing investment initiatives for research and development activities. The market size is anticipated to grow at a CAGR of 14.80% during the forecast period of 2025-2034 to achieve a value of USD 3606.76 Million by 2034.
Cystinuria Market Overview
Cystinuria, a rare kidney stone disease results in the formation of cystine stones in the kidneys, bladder, and ureters. This condition is inherited from both parents through a gene defect. Symptoms of cystinuria may include blood in the urine, severe pain on one side of the back or side, nausea, vomiting, and pain in the pelvis, groin, or abdomen. Cystinuria is typically asymptomatic when no stones are present; however, symptoms will recur each time stones form in the kidneys, which is common.
The cystinuria market demand is driven by the rising incidence of the genetic condition. The market is further experiencing growth driven by increased regulatory attention and greater awareness of available treatments for this condition. This growth is further supported by expanded healthcare infrastructure investments by the government and private organizations coupled with the rise in inherited disorder prevalence. The collaborations among key players and the growing disposable income of individuals are also collectively contributing to the cystinuria market growth.
However, the lack of knowledge among healthcare professionals and people owing to the rarity of the disease and limited reimbursement in developing economies is a major factor expected to restrain the market growth. However, this lack of knowledge may help the government to develop new programs and launch initiatives to spread awareness about cystinuria. Additionally, the excessive costs associated with research and development, coupled with the scarcity of treatment options, are again expected to hinder the market growth. Stringent drug approval regulations further impede the pace of market growth. Overcoming these challenges will be crucial for sustained and robust development in the cystinuria market to relieve the patients suffering from the condition.
Promising Developments
The market is poised for significant growth driven by some interesting and promising developments in treatments and substantial investments in research for cystinuria. For instance, in January 2024, a new treatment, Advicenne's potassium bicarbonate + potassium citrate in prolonged release (PR) form is progressing through Phase III clinical development, showing potential as a treatment for cystinuria. This advancement exhibits a significant step toward addressing the therapeutic needs of those affected by this rare kidney stone disease, contributing to the rising cystinuria market share.
Increasing Research Grants The market is driven by significant contributions through research grants. Determined researchers and their dedicated efforts backed by grants are highlighting the importance of exploring innovative solutions for this condition. For instance, the generous support from the Kiriwina Investment Company, amounting to USD 120,000 over five years, has enabled the establishment of a groundbreaking mouse model of cystinuria. Dr Malcolm Starkey received two grants to support his group’s research on cystinuria. This model, created through advanced CRISPR/Cas9 gene editing, exhibits the most common mutation causing the disease, providing a potentially strong platform for developing targeted therapies and propelling the cystinuria market demand.
These advancements reflect a growing interest and commitment within the medical community and among investors to dig deeper into understanding cystinuria and innovating treatments. The introduction of novel therapies in clinical development and substantial research funding is expected to drive the cystinuria market forward. As these initiatives progress, they promise to improve patient outcomes and potentially transform the standard of care for individuals battling cystinuria.
Clinical Trials
In December 2023, a clinical trial with Ethical Committee approval was conducted by Viola D’Ambrosio to characterize bone mineral density (BMD) in cystinuria. The study included thirty-nine patients and 40% were women, adult cystinuric patients followed at 3 specialized outpatient clinics in Italy (Rome, Naples, and Verona). The successful completion of the trial showcased a high prevalence of low BMD in cystinuric patients, despite having normal (or moderately impaired) kidney function and being relatively young.
The insights from this clinical trial highlighted the crucial gap of unmet needs in cystinuria. Such results are expected to gain traction among pharmaceutical companies to recognize the impacts of cystinuria on BMD in women along with kidney association. This may result in additional research and development of drugs and treatments to address this issue and boost the cystinuria market during the forecast period.
Cystinuria Market Segmentations
“Cystinuria Market Report and Forecast 2025-2034” offers a detailed analysis of the market based on the following segments:
Market Breakup by Disease Type
The United States is dominating the region market for cystinuria and is likely to lead the global market during the forecast period of 2025-2034. The region is dominating due to the increased prevalence of cystinuria and continued research and development activities to fill a critical gap of unmet medical needs in the market. The combined efforts of researchers and companies to create and establish an appropriate yet effective treatment for patients suffering from this rare disease are poised to foster cystinuria market growth in the forecast period. For instance, In February 2023, Ethicon, a Johnson & Johnson MedTech company, announced the first patient successfully receiving robotic-assisted removal of kidney stones using the MONARCH™ Platform for Urology.
As part of a clinical study, UCI Health, the clinical enterprise of the University of California, Irvine (UCI), used the MONARCH Platform for Urology to complete the first robotically assisted electromagnetic (EM)-guided percutaneous access and mini-percutaneous nephrolithotomy (PCNL) procedure. The clinical study involves a collaboration with co-investigator, Dr. Mihir Desai from the University of Southern California (USC).
Cystinuria Market: Competitor Landscape
The key features of the market report include patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:
*Please Note:* The report will take 5 business days to complete, after order confirmation.
Cystinuria Market Overview
Cystinuria, a rare kidney stone disease results in the formation of cystine stones in the kidneys, bladder, and ureters. This condition is inherited from both parents through a gene defect. Symptoms of cystinuria may include blood in the urine, severe pain on one side of the back or side, nausea, vomiting, and pain in the pelvis, groin, or abdomen. Cystinuria is typically asymptomatic when no stones are present; however, symptoms will recur each time stones form in the kidneys, which is common.
The cystinuria market demand is driven by the rising incidence of the genetic condition. The market is further experiencing growth driven by increased regulatory attention and greater awareness of available treatments for this condition. This growth is further supported by expanded healthcare infrastructure investments by the government and private organizations coupled with the rise in inherited disorder prevalence. The collaborations among key players and the growing disposable income of individuals are also collectively contributing to the cystinuria market growth.
However, the lack of knowledge among healthcare professionals and people owing to the rarity of the disease and limited reimbursement in developing economies is a major factor expected to restrain the market growth. However, this lack of knowledge may help the government to develop new programs and launch initiatives to spread awareness about cystinuria. Additionally, the excessive costs associated with research and development, coupled with the scarcity of treatment options, are again expected to hinder the market growth. Stringent drug approval regulations further impede the pace of market growth. Overcoming these challenges will be crucial for sustained and robust development in the cystinuria market to relieve the patients suffering from the condition.
Promising Developments
The market is poised for significant growth driven by some interesting and promising developments in treatments and substantial investments in research for cystinuria. For instance, in January 2024, a new treatment, Advicenne's potassium bicarbonate + potassium citrate in prolonged release (PR) form is progressing through Phase III clinical development, showing potential as a treatment for cystinuria. This advancement exhibits a significant step toward addressing the therapeutic needs of those affected by this rare kidney stone disease, contributing to the rising cystinuria market share.
Increasing Research Grants The market is driven by significant contributions through research grants. Determined researchers and their dedicated efforts backed by grants are highlighting the importance of exploring innovative solutions for this condition. For instance, the generous support from the Kiriwina Investment Company, amounting to USD 120,000 over five years, has enabled the establishment of a groundbreaking mouse model of cystinuria. Dr Malcolm Starkey received two grants to support his group’s research on cystinuria. This model, created through advanced CRISPR/Cas9 gene editing, exhibits the most common mutation causing the disease, providing a potentially strong platform for developing targeted therapies and propelling the cystinuria market demand.
These advancements reflect a growing interest and commitment within the medical community and among investors to dig deeper into understanding cystinuria and innovating treatments. The introduction of novel therapies in clinical development and substantial research funding is expected to drive the cystinuria market forward. As these initiatives progress, they promise to improve patient outcomes and potentially transform the standard of care for individuals battling cystinuria.
Clinical Trials
In December 2023, a clinical trial with Ethical Committee approval was conducted by Viola D’Ambrosio to characterize bone mineral density (BMD) in cystinuria. The study included thirty-nine patients and 40% were women, adult cystinuric patients followed at 3 specialized outpatient clinics in Italy (Rome, Naples, and Verona). The successful completion of the trial showcased a high prevalence of low BMD in cystinuric patients, despite having normal (or moderately impaired) kidney function and being relatively young.
The insights from this clinical trial highlighted the crucial gap of unmet needs in cystinuria. Such results are expected to gain traction among pharmaceutical companies to recognize the impacts of cystinuria on BMD in women along with kidney association. This may result in additional research and development of drugs and treatments to address this issue and boost the cystinuria market during the forecast period.
Cystinuria Market Segmentations
“Cystinuria Market Report and Forecast 2025-2034” offers a detailed analysis of the market based on the following segments:
Market Breakup by Disease Type
- Type I cystinuria
- Type II cystinuria
- Type III cystinuria
- Tiopronin
- Penicillamine
- Others
- Solutions
- Tablet
- Injection
- Others
- Oral
- Intravenous
- Topical
- Others
- Hospital Pharmacy
- Online Pharmacy
- Retail Pharmacy
- United States
- United Kingdom
- Germany
- France
- Italy
- Spain
- Japan
- China
The United States is dominating the region market for cystinuria and is likely to lead the global market during the forecast period of 2025-2034. The region is dominating due to the increased prevalence of cystinuria and continued research and development activities to fill a critical gap of unmet medical needs in the market. The combined efforts of researchers and companies to create and establish an appropriate yet effective treatment for patients suffering from this rare disease are poised to foster cystinuria market growth in the forecast period. For instance, In February 2023, Ethicon, a Johnson & Johnson MedTech company, announced the first patient successfully receiving robotic-assisted removal of kidney stones using the MONARCH™ Platform for Urology.
As part of a clinical study, UCI Health, the clinical enterprise of the University of California, Irvine (UCI), used the MONARCH Platform for Urology to complete the first robotically assisted electromagnetic (EM)-guided percutaneous access and mini-percutaneous nephrolithotomy (PCNL) procedure. The clinical study involves a collaboration with co-investigator, Dr. Mihir Desai from the University of Southern California (USC).
Cystinuria Market: Competitor Landscape
The key features of the market report include patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:
- Retrophin
- Recordati Rare Diseases
- Mission Therapeutics
- Enterome Bioscience
- Horizon Therapeutics
- Arcturus Therapeutics
- Codexis
- Mito pharmaceuticals
- Abbott
- Bausch Health Companies
- Allena Pharmaceuticals
- Mylan N.V. (now part of Viatris)
- Chiesi Farmaceutici S.p.A.
*Please Note:* The report will take 5 business days to complete, after order confirmation.
Table of Contents
350 Pages
- 1 Preface
- 1.1 Objectives of the Study
- 1.2 Key Assumptions
- 1.3 Report Coverage – Key Segmentation and Scope
- 1.4 Research Methodology
- 2 Executive Summary
- 3 Cystinuria Overview
- 3.1 Guidelines and Stages
- 3.2 Pathophysiology
- 3.3 Screening and Diagnosis
- 3.4 Pathway
- 4 Patient Profile
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Success Rate
- 5 Cystinuria Epidemiology Analysis- 8 Major Markets
- 5.1 8MM Epidemiology Scenario Overview (2018-2034)
- 5.2 United States Cystinuria Epidemiology Forecast (2018-2034)
- 5.3 EU-4 and United Kingdom Cystinuria Epidemiology Forecast (2018-2034)
- 5.3.1 Germany Cystinuria Epidemiology Forecast (2018-2034)
- 5.3.2 France Cystinuria Epidemiology Forecast (2018-2034)
- 5.3.3 Italy Cystinuria Epidemiology Forecast (2018-2034)
- 5.3.4 Spain Cystinuria Epidemiology Forecast (2018-2034)
- 5.3.5 United Kingdom Cystinuria Epidemiology Forecast (2018-2034)
- 5.4 Japan Cystinuria Epidemiology Forecast (2018-2034)
- 5.5 China Cystinuria Epidemiology Forecast (2018-2034)
- 6 Cystinuria Market Overview – 8 Major Markets
- 6.1 Cystinuria Market Historical Value (2018-2024)
- 6.2 Cystinuria Market Forecast Value (2025-2034)
- 7 Cystinuria Market Landscape – 8 Major Markets
- 7.1 Cystinuria Market: Developers Landscape
- 7.1.1 Analysis by Year of Establishment
- 7.1.2 Analysis by Company Size
- 7.1.3 Analysis by Region
- 7.2 Cystinuria Market: Product Landscape
- 7.2.1 Analysis by Drug Class
- 7.2.2 Analysis by Route of Administration
- 8 Cystinuria Challenges and Unmet Needs
- 8.1 Pathway Challenges
- 8.2 Compliance and Drop-Out Analysis
- 8.3 Awareness and Prevention Gaps
- 9 Cost of Treatment
- 10 Cystinuria Market Dynamics
- 10.1 Market Drivers and Constraints
- 10.2 SWOT Analysis
- 10.3 Porter’s Five Forces Model
- 10.4 Key Demand Indicators
- 10.5 Key Price Indicators
- 10.6 Industry Events, Initiatives, and Trends
- 10.7 Value Chain Analysis
- 11 Cystinuria Market Segmentation – 8 Major Markets
- 11.1 Cystinuria Market by Disease Type
- 11.1.1 Market Overview
- 11.1.2 Type I Cystinuria
- 11.1.3 Type II Cystinuria
- 11.1.4 Type III Cystinuria
- 11.2 Cystinuria Market by Drug Type
- 11.2.1 Market Overview
- 11.2.2 Tiopronin
- 11.2.3 Penicillamine
- 11.2.4 Others
- 11.3 Cystinuria Market by Dosage Form
- 11.3.1 Market Overview
- 11.3.2 Solutions
- 11.3.3 Tablet
- 11.3.4 Injection
- 11.3.5 Others
- 11.4 Cystinuria Market by Route of Administration
- 11.4.1 Market Overview
- 11.4.2 Oral
- 11.4.3 Intravenous
- 11.4.4 Topical
- 11.4.5 Others
- 11.5 Cystinuria Market by Distribution Channel
- 11.5.1 Market Overview
- 11.5.2 Hospital Pharmacy
- 11.5.3 Online Pharmacy
- 11.5.4 Retail Pharmacy
- 11.6 Cystinuria Market by Region
- 11.6.1 Market Overview
- 11.6.2 United States
- 11.6.3 EU-4 and the United Kingdom
- 11.6.3.1 Germany
- 11.6.3.2 France
- 11.6.3.3 Italy
- 11.6.3.4 Spain
- 11.6.3.5 United Kingdom
- 11.6.4 Japan
- 11.6.5 China
- 12 United States Cystinuria Market
- 12.1 United States Cystinuria Market Historical Size (2018-2024)
- 12.2 United States Cystinuria Market Forecast Size (2025-2034)
- 12.3 United States Cystinuria Market by Disease Type
- 12.3.1 Market Overview
- 12.3.2 Type I Cystinuria
- 12.3.3 Type II Cystinuria
- 12.3.4 Type III Cystinuria
- 12.4 United States Cystinuria Market by Drug Type
- 12.4.1 Market Overview
- 12.4.2 Tiopronin
- 12.4.3 Penicillamine
- 12.4.4 Others
- 13 EU-4 and United Kingdom Cystinuria Market
- 13.1 EU-4 and United Kingdom Cystinuria Market Historical Value (2018-2024)
- 13.2 EU-4 and United Kingdom Cystinuria Market Forecast Value (2025-2034)
- 13.3 EU-4 and United Kingdom Cystinuria Market by Disease Type
- 13.3.1 Market Overview
- 13.3.2 Type I Cystinuria
- 13.3.3 Type II Cystinuria
- 13.3.4 Type III Cystinuria
- 13.4 EU-4 and United Kingdom Cystinuria Market by Drug Type
- 13.4.1 Market Overview
- 13.4.2 Tiopronin
- 13.4.3 Penicillamine
- 13.4.4 Others
- 14 Japan Cystinuria Market
- 14.1 Japan Cystinuria Market Historical Size (2018-2024)
- 14.2 Japan Cystinuria Market Forecast Size (2025-2034)
- 14.3 Japan Cystinuria Market by Disease Type
- 14.3.1 Market Overview
- 14.3.2 Type I Cystinuria
- 14.3.3 Type II Cystinuria
- 14.3.4 Type III Cystinuria
- 14.4 Japan Cystinuria Market by Drug Type
- 14.4.1 Market Overview
- 14.4.2 Tiopronin
- 14.4.3 Penicillamine
- 14.4.4 Others
- 15 China Cystinuria Market
- 15.1 China Cystinuria Market Historical Size (2018-2024)
- 15.2 China Cystinuria Market Forecast Size (2025-2034)
- 15.3 China Cystinuria Market by Disease Type
- 15.3.1 Market Overview
- 15.3.2 Type I Cystinuria
- 15.3.3 Type II Cystinuria
- 15.3.4 Type III Cystinuria
- 15.4 China Cystinuria Market by Drug Type
- 15.4.1 Market Overview
- 15.4.2 Tiopronin
- 15.4.3 Penicillamine
- 15.4.4 Others
- 16 Regulatory Framework
- 16.1 Regulatory Overview
- 16.1.1 US FDA
- 16.1.2 EU EMA
- 16.1.3 Japan PMDA
- 16.1.4 Others
- 17 Patent Analysis
- 17.1 Analysis by Type of Patent
- 17.2 Analysis by Publication year
- 17.3 Analysis by Issuing Authority
- 17.4 Analysis by Patent Age
- 17.5 Analysis by CPC Analysis
- 17.6 Analysis by Patent Valuation
- 17.7 Analysis by Key Players
- 18 Grants Analysis
- 18.1 Analysis by Year
- 18.2 Analysis by Amount Awarded
- 18.3 Analysis by Issuing Authority
- 18.4 Analysis by Grant Application
- 18.5 Analysis by Funding Institute
- 18.6 Analysis by NIH Departments
- 18.7 Analysis by Recipient Organization
- 19 Clinical Trials Analysis
- 19.1 Analysis by Trial Registration Year
- 19.2 Analysis by Trial Status
- 19.3 Analysis by Trial Phase
- 19.4 Analysis by Therapeutic Area
- 19.5 Analysis by Geography
- 20 Funding and Investment Analysis
- 20.1 Analysis by Funding Instances
- 20.2 Analysis by Type of Funding
- 20.3 Analysis by Funding Amount
- 20.4 Analysis by Leading Players
- 20.5 Analysis by Leading Investors
- 20.6 Analysis by Geography
- 21 Partnership and Collaborations Analysis
- 21.1 Analysis by Partnership Instances
- 21.2 Analysis by Type of Partnership
- 21.3 Analysis by Leading Players
- 21.4 Analysis by Geography
- 22 Supplier Landscape
- 22.1 Retrophin
- 22.1.1 Financial Analysis
- 22.1.2 Product Portfolio
- 22.1.3 Demographic Reach and Achievements
- 22.1.4 Mergers and Acquisitions
- 22.1.5 Certifications
- 22.2 Recordati Rare Diseases Inc.
- 22.2.1 Financial Analysis
- 22.2.2 Product Portfolio
- 22.2.3 Demographic Reach and Achievements
- 22.2.4 Mergers and Acquisitions
- 22.2.5 Certifications
- 22.3 Mission Therapeutics
- 22.3.1 Financial Analysis
- 22.3.2 Product Portfolio
- 22.3.3 Demographic Reach and Achievements
- 22.3.4 Mergers and Acquisitions
- 22.3.5 Certifications
- 22.4 Enterome Bioscience
- 22.4.1 Financial Analysis
- 22.4.2 Product Portfolio
- 22.4.3 Demographic Reach and Achievements
- 22.4.4 Mergers and Acquisitions
- 22.4.5 Certifications
- 22.5 Horizon Therapeutics
- 22.5.1 Financial Analysis
- 22.5.2 Product Portfolio
- 22.5.3 Demographic Reach and Achievements
- 22.5.4 Mergers and Acquisitions
- 22.5.5 Certifications
- 22.6 Arcturus Therapeutics
- 22.6.1 Financial Analysis
- 22.6.2 Product Portfolio
- 22.6.3 Demographic Reach and Achievements
- 22.6.4 Mergers and Acquisitions
- 22.6.5 Certifications
- 22.7 Codexis
- 22.7.1 Financial Analysis
- 22.7.2 Product Portfolio
- 22.7.3 Demographic Reach and Achievements
- 22.7.4 Mergers and Acquisitions
- 22.7.5 Certifications
- 22.8 Mito Pharmaceuticals
- 22.8.1 Financial Analysis
- 22.8.2 Product Portfolio
- 22.8.3 Demographic Reach and Achievements
- 22.8.4 Mergers and Acquisitions
- 22.8.5 Certifications
- 22.9 Abbott
- 22.9.1 Financial Analysis
- 22.9.2 Product Portfolio
- 22.9.3 Demographic Reach and Achievements
- 22.9.4 Mergers and Acquisitions
- 22.9.5 Certifications
- 22.10 Bausch Health Companies
- 22.10.1 Financial Analysis
- 22.10.2 Product Portfolio
- 22.10.3 Demographic Reach and Achievements
- 22.10.4 Mergers and Acquisitions
- 22.10.5 Certifications
- 22.11 Allena Pharmaceuticals
- 22.11.1 Financial Analysis
- 22.11.2 Product Portfolio
- 22.11.3 Demographic Reach and Achievements
- 22.11.4 Mergers and Acquisitions
- 22.11.5 Certifications
- 22.12 Mylan N.V. (now part of Viatris)
- 22.12.1 Financial Analysis
- 22.12.2 Product Portfolio
- 22.12.3 Demographic Reach and Achievements
- 22.12.4 Mergers and Acquisitions
- 22.12.5 Certifications
- 22.13 Chiesi Farmaceutici S.p.A.
- 22.13.1 Financial Analysis
- 22.13.2 Product Portfolio
- 22.13.3 Demographic Reach and Achievements
- 22.13.4 Mergers and Acquisitions
- 22.13.5 Certifications
- 23 Cystinuria Market - Distribution Model (Additional Insight)
- 23.1 Overview
- 23.2 Potential Distributors
- 23.3 Key Parameters for Distribution Partner Assessment
- 24 Key Opinion Leaders (KOL) Insights (Additional Insight)
- 25 Company Competitiveness Analysis (Additional Insight)
- 25.1 Very Small Companies
- 25.2 Small Companies
- 25.3 Mid-Sized Companies
- 25.4 Large Companies
- 25.5 Very Large Companies
- 26 Payment Methods (Additional Insight)
- 26.1 Government Funded
- 26.2 Private Insurance
- 26.3 Out-of-Pocket
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