What is Ornithine Transcarbamylase (OTC) Deficiency?
Ornithine transcarbamylase (OTC/OTCD) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme ornithine transcarbamylase (OTC) and is the only X-linked urea cycle disorder. The disorder that prevents the breakdown and excretion of ammonia; this allows ammonia to rise to toxic levels and affect the central nervous system.
What is the cause of OTC deficiency?
The underlying cause of OTC deficiency is a gene mutation on the X chromosome and over 100 mutations have been found to result in OTCD.
What are the symptoms of Ornithine Transcarbamylase (OTC) Deficiency?
OTC deficiency symptoms includes vomiting, refusal to eat, progressive lethargy, and coma. The severity and age of onset of OTC deficiency vary from person to person, even within the same family. Most commonly, symptoms of OTC deficiency begin in infancy (the early-onset form). An infant with OTC deficiency may be lacking energy (lethargic), be unwilling to eat, and have problems with breathing rate or body temperature.
How is Ornithine Transcarbamylase Deficiency diagnosed?
A diagnosis of OTC deficiency should be considered in any newborn that has an undiagnosed illness characterized by vomiting, progressive lethargy, and irritability. The study of blood plasma and urine is used to differentiate OTC deficiency from other urea cycle disorders. Individuals with OTC deficiency usually have both low levels of citrulline and high glutamine in the blood and high levels of orotic acid in the urine. In rare cases, OTC deficiency may be detected by surgical removal (biopsy) and microscopic examination of tissue samples from the liver, duodenum, and rectum where deficient enzyme activity may be seen. DNA genetic testing is also available to confirm the diagnosis.
How OTC Deficiency is treated?
OTC deficiency treatment includes hydration, arginine, and hemodialysis. Long-term therapy for OTC deficiency combines dietary restrictions and the stimulation of alternative methods of converting and excreting nitrogen from the body (alternative pathways therapy).
What are Ornithine Transcarbamylase Deficiency Epidemiology Insights?
- OTC deficiency is the most common of the urea cycle disorders (UCD). UCD occur in 1 of 8200 US live births, making these disorders more common in the US than globally.
- OTC deficiency occurs more commonly in neonates and early childhood than in adulthood.
- OTC deficiency affects males more often than females and is fully expressed in males only.
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What is Ornithine Transcarbamylase Deficiency Market Outlook?
According to DelveInsight, Ornithine Transcarbamylase Deficiency (OTC Deficiency) market in 7MM is expected to change in the study period 2018-2030.
The Ornithine Transcarbamylase Deficiency market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Ornithine Transcarbamylase Deficiency (OTC Deficiency) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a thorough detail of OTC Deficiency market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.
How many companies are developing drugs for the Ornithine Transcarbamylase Deficiency treatment?
The pipeline of Ornithine Transcarbamylase Deficiency possess very few potential key players, such as Arcturus Therapeutics, Ultragenyx and others. The dynamics of OCT deficiency market are anticipated to change in the coming years owing to the improvement in the research and development activities so that market will comprise efficient treatment options. The launch of emerging therapies is expected during the forecast period of 2020–2030.
Table of contents
1. Key Insights
2. Executive Summary of Ornithine Transcarbamylase Deficiency (OTC Deficiency)
3. Competitive Intelligence Analysis for Ornithine Transcarbamylase Deficiency (OTC Deficiency)
4. Ornithine Transcarbamylase Deficiency (OTC Deficiency): Market Overview at a Glance
4.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Share (%) Distribution in 2018
4.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Share (%) Distribution in 2030
5. Ornithine Transcarbamylase Deficiency (OTC Deficiency): Disease Background and Overview
5.1. Introduction
5.2. Sign and Symptoms
5.3. Pathophysiology
5.4. Risk Factors
5.5. Diagnosis
6. Patient Journey
7. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology and Patient Population
7.1. Epidemiology Key Findings
7.2. Assumptions and Rationale: 7MM
7.3. Epidemiology Scenario: 7MM
7.3.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in the 7MM (2018-2030)
7.4. United States Epidemiology
7.4.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in the United States (2018-2030)
7.5. EU-5 Country-wise Epidemiology
7.5.1. Germany Epidemiology
7.5.1.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in Germany (2018-2030)
7.5.2. France Epidemiology
7.5.2.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in France (2018-2030)
7.5.3. Italy Epidemiology
7.5.3.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in Italy (2018-2030)
7.5.4. Spain Epidemiology
7.5.4.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in Spain (2018-2030)
7.5.5. United Kingdom Epidemiology
7.5.5.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in the United Kingdom (2018-2030)
7.5.6. Japan Epidemiology
7.5.6.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Epidemiology Scenario in Japan (2018-2030)
8. Treatment Algorithm, Current Treatment, and Medical Practices
8.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Treatment and Management
8.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Treatment Algorithm
9. Unmet Needs
10. Key Endpoints of Ornithine Transcarbamylase Deficiency (OTC Deficiency) Treatment
11. Marketed Products
11.1. List of Marketed Products in the 7MM
11.2. Drug Name: Company Name
11.2.1. Product Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Pivotal Clinical Trials
11.2.5. Summary of Pivotal Clinical Trial
List to be continued in report
12. Emerging Therapies
12.1. Key Cross
12.2. Drug Name: Company Name
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Safety and Efficacy
12.2.5. Product Profile
List to be continued in report
13. Ornithine Transcarbamylase Deficiency (OTC Deficiency): Seven Major Market Analysis
13.1. Key Findings
13.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size in 7MM
13.3. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in the 7MM
14. Attribute analysis
15. 7MM: Market Outlook
15.1. United States: Market Size
15.1.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in the United States
15.1.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in the United States
15.2. EU-5 countries: Market Size and Outlook
15.3. Germany Market Size
15.3.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in Germany
15.3.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in Germany
15.4. France Market Size
15.4.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in France
15.4.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in France
15.5. Italy Market Size
15.5.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in Italy
15.5.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in Italy
15.6. Spain Market Size
15.6.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in Spain
15.6.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in Spain
15.7. United Kingdom Market Size
15.7.1. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in the United Kingdom
15.7.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in the United Kingdom
15.8. Japan Market Outlook
15.8.1. Japan Market Size
15.8.2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Total Market Size in Japan
15.8.3. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Size by Therapies in Japan
16. Access and Reimbursement Overview of Ornithine Transcarbamylase Deficiency (OTC Deficiency)
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
20.1. Bibliography
20.2. Report Methodology
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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