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.
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
1.2 Process of Urea Cycle
1.3 Urea Cycle Disorder (UCD)
1.4 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
2.2 The U.S. Existing and New UCD Population Forecast
2.3 The U.S. Diagnosed UCD Population Forecast
2.4 The U.S. UCD Drug Market
2.4.1 The U.S. Ravicti Revenue
2.4.2 The U.S. Ravicti Revenue Forecast
2.4.3 The U.S KB195 Revenue Forecast
2.4.4 The U.S. UCD Population Treated with KB195 Forecast
3. EUROPE MARKET ANALYSIS
3.1 Europe UCD Population Forecast
3.2 Europe Existing and New UCD Population Forecast
3.3 Europe Diagnosed UCD Population Forecast
3.4 Europe UCD Drug Market
3.4.1 Europe KB195 Revenue Forecast
3.4.2 Europe UCD Population Treated with KB195 Forecast
4. MARKET DYNAMICS
4.1 Growth Drivers
4.1.1 Increasing Healthcare Expenditure in the U.S.
4.1.2 Rise in Pharmaceutical R&D Expenditures in the U.S.
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
4.2.2 Upcoming Launches of Anti-UCD Drugs
4.2.3 Development of Orphan Drugs for Urea Cycle Disorder
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
5.2 Key Players – Revenue Comparison
5.3 Key Players – Market Capital Comparison
6. COMPANY PROFILES
6.1 Horizon Pharma Plc.
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Ultragenyx Pharmaceuticals Inc.
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Aeglea Bio Therapeutics
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Synlogix
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
1.1 Urea Cycle
1.2 Process of Urea Cycle
1.3 Urea Cycle Disorder (UCD)
1.4 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
2.2 The U.S. Existing and New UCD Population Forecast
2.3 The U.S. Diagnosed UCD Population Forecast
2.4 The U.S. UCD Drug Market
2.4.1 The U.S. Ravicti Revenue
2.4.2 The U.S. Ravicti Revenue Forecast
2.4.3 The U.S KB195 Revenue Forecast
2.4.4 The U.S. UCD Population Treated with KB195 Forecast
3. EUROPE MARKET ANALYSIS
3.1 Europe UCD Population Forecast
3.2 Europe Existing and New UCD Population Forecast
3.3 Europe Diagnosed UCD Population Forecast
3.4 Europe UCD Drug Market
3.4.1 Europe KB195 Revenue Forecast
3.4.2 Europe UCD Population Treated with KB195 Forecast
4. MARKET DYNAMICS
4.1 Growth Drivers
4.1.1 Increasing Healthcare Expenditure in the U.S.
4.1.2 Rise in Pharmaceutical R&D Expenditures in the U.S.
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
4.2.2 Upcoming Launches of Anti-UCD Drugs
4.2.3 Development of Orphan Drugs for Urea Cycle Disorder
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
5.2 Key Players – Revenue Comparison
5.3 Key Players – Market Capital Comparison
6. COMPANY PROFILES
6.1 Horizon Pharma Plc.
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Ultragenyx Pharmaceuticals Inc.
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Aeglea Bio Therapeutics
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Synlogix
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
LIST OF FIGURES
Urea Cycle
The U.S Total UCD Population Forecast (2019-2023)
The U.S Existing and New UCD Population Forecast (2019-2023)
The U.S Diagnosed UCD Population Forecast (2019-2023)
The U.S Ravicti Revenue (2014-2018)
The U.S Ravicti Revenue Forecast (2019-2023)
The U.S KB195 Revenue Forecast (2022-2026)
The U.S. UCD Population Treated with KB195 Forecast (2022-2026)
Europe UCD Population Forecast (2019-2023)
Europe Existing and New UCD Population Forecast (2019-2022)
Europe Diagnosed UCD Population Forecast (2019-2023)
Europe KB195 Revenue Forecast (2023-2027)
Europe UCD Population Treated with KB195 Forecast (2023-2027)
The U.S. Healthcare Expenditure per Capita (2014-2018)
Pharmaceutical R&D Expenditures in the U.S. (2014-2018)
The U.S and Europe Newborn Population Forecast (2019-2023)
Key Players – Market Capital Comparison
Horizon Pharma Plc. Revenue by Segment (2018)
Horizon Pharma Plc. Revenue and Net Loss (2014-2018)
Horizon Pharma Plc. R&D Expenditure (2016-2018)
Horizon Pharma Plc. Advertising Expenditure (2016-2018)
Ultragenyx Pharmaceuticals Revenue by Segment (2018)
Ultragenyx Pharmaceuticals Revenue and Net Loss (2014-2018)
Ultragenyx Pharmaceuticals R&D Expenditure (2016-2018)
Aeglea Bio Therapeutics Revenue and Net Loss (2014-2018)
Aeglea Bio Therapeutics R&D Expenditure (2016-2018)
Synlogix Revenue and Net Loss (2014-2018)
Synlogix R&D Expenditure (2016-2018)
Urea Cycle
The U.S Total UCD Population Forecast (2019-2023)
The U.S Existing and New UCD Population Forecast (2019-2023)
The U.S Diagnosed UCD Population Forecast (2019-2023)
The U.S Ravicti Revenue (2014-2018)
The U.S Ravicti Revenue Forecast (2019-2023)
The U.S KB195 Revenue Forecast (2022-2026)
The U.S. UCD Population Treated with KB195 Forecast (2022-2026)
Europe UCD Population Forecast (2019-2023)
Europe Existing and New UCD Population Forecast (2019-2022)
Europe Diagnosed UCD Population Forecast (2019-2023)
Europe KB195 Revenue Forecast (2023-2027)
Europe UCD Population Treated with KB195 Forecast (2023-2027)
The U.S. Healthcare Expenditure per Capita (2014-2018)
Pharmaceutical R&D Expenditures in the U.S. (2014-2018)
The U.S and Europe Newborn Population Forecast (2019-2023)
Key Players – Market Capital Comparison
Horizon Pharma Plc. Revenue by Segment (2018)
Horizon Pharma Plc. Revenue and Net Loss (2014-2018)
Horizon Pharma Plc. R&D Expenditure (2016-2018)
Horizon Pharma Plc. Advertising Expenditure (2016-2018)
Ultragenyx Pharmaceuticals Revenue by Segment (2018)
Ultragenyx Pharmaceuticals Revenue and Net Loss (2014-2018)
Ultragenyx Pharmaceuticals R&D Expenditure (2016-2018)
Aeglea Bio Therapeutics Revenue and Net Loss (2014-2018)
Aeglea Bio Therapeutics R&D Expenditure (2016-2018)
Synlogix Revenue and Net Loss (2014-2018)
Synlogix R&D Expenditure (2016-2018)
LIST OF TABLES
Types of UCDs and Their Management
Upcoming Launches of Anti-UCD Drugs
Regulations and Policies for Orphan Drugs
Key Players – Product Pipeline in the Urea Cycle Disorder Space
Key Players – Revenue Comparison (2018)
Aeglea Bio Therapeutics Drugs Pipeline Overview (2018)
Synlogix Drugs Pipeline Overview (2018)
Types of UCDs and Their Management
Upcoming Launches of Anti-UCD Drugs
Regulations and Policies for Orphan Drugs
Key Players – Product Pipeline in the Urea Cycle Disorder Space
Key Players – Revenue Comparison (2018)
Aeglea Bio Therapeutics Drugs Pipeline Overview (2018)
Synlogix Drugs Pipeline Overview (2018)
