DelveInsight’s “Urea Cycle Disorders Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Urea Cycle Disorders market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Urea Cycle Disorders market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Urea Cycle Disorders: An Overview
Urea cycle disorders (UCDs) are a congenital error of nitrogen detoxification/arginine synthesis due to defects in the urea cycle enzymes such as carbamoylphosphate synthetase 1 (CPS1), ornithine transcarbamylase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL) and arginase 1 (ARG1), leading to respective deficiencies (abbreviated CPS1D, OTCD, ASSD, ASLD, and ARG1D).
All the urea cycle disorders are inherited in an autosomal recessive pattern, except ornithine transcarbamoylase (OTC) deficiency, which is inherited as an X-linked trait; however, female carriers of the OTC gene can become symptomatic.
Urea Cycle Disorders Market Key Facts
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According to Van Leung-Pineda (2019), the prevalence of UCDs in the US is approximately 1 in 35,000, with two-thirds having symptoms in the neonatal period.
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According to Mageed G (2015), the prevalence of UCDs in EU-5 was 1.89 per 100,000 in the 0-4-year-old age group, 1.63 per 100,000 in the 10-14-year-old age group, and 1.57 per 100,000 in the 15-17-year-old age group.
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According to NORD, OTC deficiency affects males more often than females, and most males are symptomatic in nature. In males, symptoms typically begin during the first few days of life.
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According to Kido et al. (2021), the estimated prevalence of UCDs is 1 per 50,000 live births in Japan.
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According to reports published by the CDC (Centers for Disease Control and Prevention), the anticipated incidence of UCD is 1 in 8500 births. The research findings suggest that many of the cases of UCD persist undiagnosed. In some of cases, the newborn with UCD dies without a conclusive diagnosis.
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Current research focuses on gene therapies and enzyme replacement therapy to address the existing issues. Pharmaceutical companies such as Acer Therapeutics, Aeglea BioTherapeutics, Synlogic, UltragenyxPharmaceutical, etc., are actively working toward developing potential therapies to fulfill the unmet medical needs of the currently used therapeutics.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Urea Cycle Disorders market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Urea Cycle Disorders market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Urea Cycle Disorders Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Urea Cycle Disorders Epidemiology Segmented by –
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Total Incident Cases of Urea Cycle Disorders in the 7MM [2019–2032]
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Onset-specific cases of Urea Cycle Disorders in the 7MMD [2019–2032]
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Total Incident Cases of Urea Cycle Disorders by types in the 7MM [2019–2032]
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Diagnosed and treatable Cases of Urea Cycle Disorders by types in the 7MM [2019–2032]
Urea Cycle Disorders Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the rate of uptake of the potential drugs recently launched in the Urea Cycle Disorders market or expected to be launched during the study period. The analysis covers the Urea Cycle Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Urea Cycle Disorders pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Urea Cycle Disorders Therapeutics Analysis
Several major pharma and biotech companies are developing therapies for Urea Cycle Disorders. Currently, Acer Therapeutics is leading the therapeutics market with its Urea Cycle Disorders drug candidates in the most advanced stage of clinical development.
The Leading Companies in the Urea Cycle Disorders Therapeutics Market Include:
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Acer Therapeutics
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Dimension Therapeutics
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Callitas Therapeutics
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Poseida Therapeutics
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Promethera Biosciences
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Arcturus Therapeutics
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Kaleido Biosciences
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Akaza Biopharma
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Evox Therapeutics
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Dipharma SA
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Sana Biotechnology
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Aeglea BioTherapeutics
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ERYTECH
And Many Others
Urea Cycle Disorders Drugs Covered in the Report Include:
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ACER-001: Acer Therapeutics
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Avalotcagene ontaparvovec: Dimension Therapeutics
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AEB1102: Aeglea BioTherapeutics
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DTX301: Ultragenyx Pharmaceutical
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Urea Cycle Disorders Competitive Intelligence Analysis
4. Urea Cycle Disorders Market Overview at a Glance
5. Urea Cycle Disorders Disease Background and Overview
6. Urea Cycle Disorders Patient Journey
7. Urea Cycle Disorders Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Urea Cycle Disorders Treatment Algorithm, Current Treatment, and Medical Practices
9. Urea Cycle Disorders Unmet Needs
10. Key Endpoints of Urea Cycle Disorders Treatment
11. Urea Cycle Disorders Marketed Products
12. Urea Cycle Disorders Emerging Drugs and Latest Therapeutic Advances
13. Urea Cycle Disorders Seven Major Market Analysis
14. Attribute Analysis
15. Urea Cycle Disorders Market Outlook (In US, EU5, and Japan)
16. Urea Cycle Disorders Access and Reimbursement Overview
17. KOL Views on the Urea Cycle Disorders Market
18. Urea Cycle Disorders Market Drivers
19. Urea Cycle Disorders Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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