Urea Cycle Disorders Market to Expand Significantly by 2032, States DelveInsight Report | Aeglea BioTherapeutics, Ultragenyx Pharmaceutical, Arcturus Therapeutics, Horizon Pharma

DelveInsight's "Urea Cycle Disorders Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Urea Cycle Disorders, historical and forecasted epidemiology as well as the Urea Cycle Disorders market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Urea Cycle Disorders Market Report:
• The Urea Cycle Disorders market size is anticipated to grow with a significant CAGR during the study period (2019-2032)
• In November 2024, Zevra Therapeutics is adjusting its commercial strategy for OLPRUVA to concentrate on patient segments with fewer access barriers. In collaboration with The National Urea Cycle Disorders Foundation, the company has launched a targeted patient education campaign emphasizing the importance of timely and accurate blood ammonia level testing, as elevated levels may indicate a UCD.
• In April 2024, iECURE has obtained approval for its Investigational New Drug (IND) application from the United States Food and Drug Administration (US FDA) for ECUR-506 in ornithine transcarbamylase (OTC) deficiency.
• In March 2024, CAMP4 Therapeutics Corp., a clinical-stage biotechnology firm utilizing regulatory RNA to restore healthy gene expression, announced today that the initial participant has been dosed in its Phase 1 clinical trial of CMP-CPS-001, a potential first-in-class treatment for urea cycle disorders (UCDs).
• In December 2023, The European Commission approved LOARGYS (pegzilarginase) for treating arginase 1 deficiency (ARG1-D) in adults, adolescents, and children aged 2 years and older. Shortly afterward, the company expressed plans to resubmit the Biologics License Application (BLA) in the US in 2024, seeking approval for LOARGYS in the treatment of ARG1-D.
• According to Centers for Disease Control and Prevention reports from 2022, it is estimated that urea cycle disorders (UCD) occur in approximately 1 in 8,500 births. Research indicates that a significant number of UCD cases remain undiagnosed, and tragically, some affected newborns pass away without receiving a definitive diagnosis.
• Stone et al. (2023) reported that approximately 66% of urea cycle disorders (UCDs) are caused by mutations in OTC, around 20% by ASS1 mutations, and approximately 10% by ASL mutations.
• Sawada et al. (2022) conducted a study estimating that urea cycle disorders (UCDs) have an incidence ranging from approximately 1 in every 8,000 to 44,000 births. Ornithine transcarbamylase deficiency (OTCD), the most prevalent UCD, is inherited as an X-linked trait.
• Key Urea Cycle Disorders Companies: Acer Therapeutics, Dimension Therapeutics, Callitas Therapeutics, Poseida Therapeutics, Promethera Biosciences, Arcturus Therapeutics, Kaleido Biosciences, Akaza Biopharma, Evox Therapeutics, Dipharma SA, Sana Biotechnology, Aeglea BioTherapeutics, Erytech, and others
• Key Urea Cycle Disorders Therapies: LUNAR-OTC (ARCT-810), ECUR-506, AEB1102 (pegzilarginase), DTX301, ARCT-810, HPN-100, RAVICTI, and others
• The Urea Cycle Disorders epidemiology based on gender analyzed that 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
• The Urea Cycle Disorders market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Urea Cycle Disorders pipeline products will significantly revolutionize the Urea Cycle Disorders market dynamics.

Urea Cycle Disorders Overview
Urea Cycle Disorders (UCDs) are a group of rare genetic conditions that affect the body's ability to remove ammonia, a toxic byproduct of protein metabolism, from the bloodstream. This process, known as the urea cycle, typically occurs in the liver. When the cycle is disrupted due to enzyme deficiencies, ammonia builds up, leading to potentially life-threatening symptoms such as vomiting, confusion, and lethargy. If untreated, UCDs can cause brain damage, coma, or death. Treatment often involves a combination of dietary management (low-protein diet), medications to help remove ammonia, and in some cases, liver transplantation

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Urea Cycle Disorders Epidemiology
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 trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Urea Cycle Disorders Epidemiology Segmentation:
The Urea Cycle Disorders market report proffers epidemiological analysis for the study period 2019-2032 in the 7MM segmented into:
• Total Incident Cases of Urea Cycle Disorders in the 7MM
• Onset-specific Cases of Urea Cycle Disorders in the 7MM
• Type-specific Cases of Urea Cycle Disorders in the 7MM
• Diagnosed and Treatable Cases of Urea Cycle Disorders in the 7MM

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Urea Cycle Disorders Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Urea Cycle Disorders market or expected to get launched during the study period. The analysis covers 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 analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Urea Cycle Disorders Therapies and Key Companies
• LUNAR-OTC (ARCT-810): Arcturus Therapeutics
• ECUR-506: iECURE
• AEB1102 (pegzilarginase): Aeglea BioTherapeutics
• DTX301: Ultragenyx Pharmaceutical
• ARCT-810: Arcturus Therapeutics
• HPN-100: Horizon Pharma
• RAVICTI: Horizon Therapeutics, LLC

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Urea Cycle Disorders Market Strengths
• Initial signs of translational promise in early phase clinical trials in late-onset OTCdeficient adult patients are paving the way for a wider development of gene therapy for younger patients and for all UCDs.
• LOARGYS (pegzilarginase), approved in Europe, is the first and only disease modifying treatment for ARG1-D.

Urea Cycle Disorders Market Opportunities
• UCDs emerging landscape consist of various gene therapies and mRNA therapies that have the potential to address the genetic deficiencies responsible for UCDs, potentially minimizing the need for nitro scavengers by intervening earlier in the urea cycle.
• A better understanding of UCDs genotypes may allow for more personalized treatment plans based on the specific genetic mutations involved.

Scope of the Urea Cycle Disorders Market Report
• Study Period: 2019-2032
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Urea Cycle Disorders Companies: Acer Therapeutics, Dimension Therapeutics, Callitas Therapeutics, Poseida Therapeutics, Promethera Biosciences, Arcturus Therapeutics, Kaleido Biosciences, Akaza Biopharma, Evox Therapeutics, Dipharma SA, Sana Biotechnology, Aeglea BioTherapeutics, Erytech, and others
• Key Urea Cycle Disorders Therapies: LUNAR-OTC (ARCT-810), ECUR-506, AEB1102 (pegzilarginase), DTX301, ARCT-810, HPN-100, RAVICTI, and others
• Urea Cycle Disorders Therapeutic Assessment: Urea Cycle Disorders current marketed and Urea Cycle Disorders emerging therapies
• Urea Cycle Disorders Market Dynamics: Urea Cycle Disorders market drivers and Urea Cycle Disorders market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Urea Cycle Disorders Unmet Needs, KOL's views, Analyst's views, Urea Cycle Disorders Market Access and Reimbursement

To know more about Urea Cycle Disorders companies working in the treatment market, visit @ Urea Cycle Disorders Clinical Trials and Therapeutic Assessment
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Table of Contents
1. Urea Cycle Disorders Market Report Introduction
2. Executive Summary for Urea Cycle Disorders
3. SWOT analysis of Urea Cycle Disorders
4. Urea Cycle Disorders Patient Share (%) Overview at a Glance
5. Urea Cycle Disorders Market Overview at a Glance
6. Urea Cycle Disorders Disease Background and Overview
7. Urea Cycle Disorders Epidemiology and Patient Population
8. Country-Specific Patient Population of Urea Cycle Disorders
9. Urea Cycle Disorders Current Treatment and Medical Practices
10. Urea Cycle Disorders Unmet Needs
11. Urea Cycle Disorders Emerging Therapies
12. Urea Cycle Disorders Market Outlook
13. Country-Wise Urea Cycle Disorders Market Analysis (2019-2032)
14. Urea Cycle Disorders Market Access and Reimbursement of Therapies
15. Urea Cycle Disorders Market Drivers
16. Urea Cycle Disorders Market Barriers
17. Urea Cycle Disorders Appendix
18. Urea Cycle Disorders Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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