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Urea Cycle Disorder (UCD) Market: Industry Analysis & Outlook (2019-2023)

Urea Cycle Disorder (UCD) Market: Industry Analysis & Outlook (2019-2023)

Urea cycle disorders (UCDs) are a group of orphan inherited defects of six enzymes and two transporters that constitute the urea cycle in the periportal liver cells. The age of onset symptoms vary between the different types of UCDs. UCDs often occurs during the period of infancy due to triggered postnatal catabolism. The diagnosis is made by analysis of the urine and blood for abnormal metabolites (substance produced by metabolism), and high ammonia levels. Frequent blood tests are done to monitor ammonia levels. The treatment of urea cycle disorders consists of dietary management to limit ammonia production. A liver transplant can be an effective treatment for urea cycle disorder for the proper functioning of urea cycle.

The urea cycle disorder treatment market in the U.S. and Europe is expected to grow rapidly in the next few years. Several pharmaceutical companies are under the development process of their late stage pipeline drugs for urea cycle disorders. Factors such as, increasing healthcare expenditures in the U.S, rise in the U.S. pharmaceutical R&D expenditures and collaboration between pharmaceutical companies and research institutes have driven the growth of the U.S. urea cycle disorder treatment market. Further, upcoming launches of anti-UCD drugs would contribute to the expansion of the urea cycle disorder treatment market in the U.S. as well as in Europe.

The U.S. urea cycle disorder treatment market is dominated by certain key players including Horizon Pharma Inc., Ultragenyx Pharmaceuticals Inc., Aeglea Bio Therapeutics and Synlogix. Among all the key players, Horizon Pharma ruled the U.S. UCD treatment market owing to the strong performance of its product. Horizon’ Ravicti is the market leader due its strong curative impact on the patients with UCD. The remaining key players have their products under the development process of pipeline drugs for UCDs. There would be a generic competition between the upcoming anti-UCD drugs in the coming years.


1. Overview
1.1 Urea Cycle
Table 1: Urea Cycle
1.2 Process of Urea Cycle
1.3 Urea Cycle Disorder (UCD)
1.4 Types of UCDs and Their Management
Table 2: Types of UCDs and Their Management
1.5 Diagnosis and Treatment of UCD
2. The U.S. Market Analysis
2.1 The U.S. Total UCD Population Forecast
Table 3: The U.S Total UCD Population Forecast (2019-2023)
2.2 The U.S. Existing and New UCD Population Forecast
Table 4: The U.S Existing and New UCD Population Forecast (2019-2023)
2.3 The U.S. Diagnosed UCD Population Forecast
Table 5: The U.S Diagnosed UCD Population Forecast (2019-2023)
2.4 The U.S. UCD Drug Market
2.4.1 The U.S. Ravicti Revenue
Table 6: The U.S Ravicti Revenue (2014-2018)
2.4.2 The U.S. Ravicti Revenue Forecast
Table 7: The U.S Ravicti Revenue Forecast (2019-2023)
2.4.3 The U.S KB195 Revenue Forecast
Table 8: The U.S KB195 Revenue Forecast (2022-2026)
2.4.4 The U.S. UCD Population Treated with KB195 Forecast
Table 9: The U.S. UCD Population Treated with KB195 Forecast (2022-2026)
3. Europe Market Analysis
3.1 Europe UCD Population Forecast
Table 10: Europe UCD Population Forecast (2019-2023)
3.2 Europe Existing and New UCD Population Forecast
Table 11: Europe Existing and New UCD Population Forecast (2019-2022)
3.3 Europe Diagnosed UCD Population Forecast
Table 12: Europe Diagnosed UCD Population Forecast (2019-2023)
3.4 Europe UCD Drug Market
3.4.1 Europe KB195 Revenue Forecast
Table 13: Europe KB195 Revenue Forecast (2023-2027)
3.4.2 Europe UCD Population Treated with KB195 Forecast
Table 14: Europe UCD Population Treated with KB195 Forecast (2023-2027)
4. Market Dynamics
4.1 Growth Drivers
4.1.1 Increasing Healthcare Expenditure in the U.S.
Table 15: The U.S. Healthcare Expenditure per Capita (2014-2018)
4.1.2 Rise in Pharmaceutical R&D Expenditures in the U.S.
Table 16: Pharmaceutical R&D Expenditures in the U.S. (2014-2018)
4.1.3 Collaboration of Pharmaceutical Companies with Research Institutes
4.2 Key Trends and Development
4.2.1 Shifts in the Newborn Population in the U.S. and Europe
Table 17: The U.S and Europe Newborn Population Forecast (2019-2023)
4.2.2 Upcoming Launches of Anti-UCD Drugs
Table 18: Upcoming Launches of Anti-UCD Drugs
4.2.3 Development of Orphan Drugs for Urea Cycle Disorder
Table 19: Regulations and Policies for Orphan Drugs
4.3 Challenges
4.3.1 Less Definitive Treatment Options
4.3.2 High Healthcare and Medical Costs
4.3.3 Stringent Medical Regulations
5. Competitive Landscape
5.1 Key Players – Drugs Pipeline in the Urea Cycle Disorder Space
Table 20: Key Players – Product Pipeline in the Urea Cycle Disorder Space
5.2 Key Players – Revenue Comparison
Table 21: Key Players – Revenue Comparison (2018)
5.3 Key Players – Market Capital Comparison
Table 22: Key Players – Market Capital Comparison
6. Company Profiles
6.1 Horizon Pharma Plc.
6.1.1 Business Overview
Table 23: Horizon Pharma Plc. Revenue by Segment (2018)
6.1.2 Financial Overview
Table 24: Horizon Pharma Plc. Revenue and Net Loss (2014-2018)
6.1.3 Business Strategies
Table 25: Horizon Pharma Plc. R&D Expenditure (2016-2018)
Table 26: Horizon Pharma Plc. Advertising Expenditure (2016-2018)
6.2 Ultragenyx Pharmaceuticals Inc.
6.2.1 Business Overview
Table 27: Ultragenyx Pharmaceuticals Revenue by Segment (2018)
6.2.2 Financial Overview
Table 28: Ultragenyx Pharmaceuticals Revenue and Net Loss (2014-2018)
6.2.3 Business Strategies
Table 29: Ultragenyx Pharmaceuticals R&D Expenditure (2016-2018)
6.3 Aeglea Bio Therapeutics
6.3.1 Business Overview
Table 30: Aeglea Bio Therapeutics Drugs Pipeline Overview (2018)
6.3.2 Financial Overview
Table 31: Aeglea Bio Therapeutics Revenue and Net Loss (2014-2018)
6.3.3 Business Strategies
Table 32: Aeglea Bio Therapeutics R&D Expenditure (2016-2018)
6.4 Synlogix
6.4.1 Business Overview
Table 33: Synlogix Drugs Pipeline Overview (2018)
6.4.2 Financial Overview
Table 34: Synlogix Revenue and Net Loss (2014-2018)
6.4.3 Business Strategies
Table 35: Synlogix R&D Expenditure (2016-2018)

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