The urea cycle disorders treatment market size has grown steadily in recent years. It will grow from $1.29 billion in 2023 to $1.35 billion in 2024 at a compound annual growth rate (CAGR) of 4.5%. The expansion observed in the historical period can be attributed to increased awareness and education, advancements in newborn screening practices, the emergence of patient support networks, and the establishment of standard of care guidelines.
The urea cycle disorders treatment market size is expected to see steady growth in the next few years. It will grow to $1.57 billion in 2028 at a compound annual growth rate (CAGR) of 3.9%. The anticipated growth in the forecast period can be attributed to advancements in enzyme replacement therapy, the expansion of newborn screening programs, heightened collaboration in research efforts, the broadening of therapeutic options, and the increasing utilization of telemedicine and remote care. Noteworthy trends expected during the forecast period include the implementation of advanced gene editing techniques, initiatives focused on awareness and advocacy, the exploration of regenerative medicine approaches, the conduct of long-term safety and efficacy studies, and the development of personalized nutritional therapies.
The increasing prevalence of urea cycle disorders is anticipated to drive the growth of the market for urea cycle disorders treatment. Urea cycle disorders are metabolic inborn disorders caused by defects in enzymes or transporter molecules crucial for the liver to eliminate ammonia from the blood. The rise in the prevalence of urea cycle disorders is primarily attributed to genetic disorders. Conditions identified as urea cycle disorders (UCD) involve the accumulation of ammonia, leading to customized treatment strategies that encompass dietary control, medication, and monitoring to manage and mitigate the adverse effects. As of April 2023, the prevalence of urea cycle disorders resulting from ornithine transcarbamylase deficiency was reported as 1 in 140,000 people according to StatPearls Publishing LLC. Additionally, in January 2021, the National Library of Medicine reported 113 new cases of urea cycle disorders per 35,000 live births in the United States. Therefore, the surge in the prevalence of urea cycle disorders is a significant driver of the urea cycle disorders treatment market's growth.
The increasing number of chronic kidney disorder cases is expected to contribute to the expansion of the urea cycle disorders treatment market. Chronic kidney disease (CKD), characterized by the gradual loss of kidney function over time, utilizes urea cycle disorder treatment to manage metabolic waste, especially elevated urea levels due to impaired kidney function. As of November 2023, an estimated 7.2 million individuals are believed to have chronic kidney disease (CKD) stages 1–5, with approximately 3.5 million people in the UK experiencing the later stages of CKD (stages 3-5), where symptoms become progressively challenging to control. Hence, the growing number of chronic kidney disorder cases is a driving force behind the urea cycle disorders treatment market's growth.
A product innovation has emerged as a notable trend, with major companies focusing on creating innovative products to strengthen their market positions. For example, in September 2022, Medunik USA introduced Pheburane oral pellets, a unique and flavor-masking formulation of sodium phenylbutyrate (NaPB) for the long-term management of certain urea cycle disorders. Pheburane, a prescription drug used in conjunction with a specific diet, offers a convenient administration method without the need for mixing. It can be consumed alone or sprinkled on food, enhancing patient compliance.
Major players in the urea cycle disorder treatment market are adopting a strategic partnership approach to advance therapies and improve disease management. In October 2021, Boehringer Ingelheim collaborated with Thoeris GmbH, an early-stage biopharmaceutical company in Europe, to explore novel therapies for patients with urea cycle disorders (UCDs). UCDs, rare diseases resulting from genetic liver dysfunctions leading to elevated ammonia levels, lack sufficient treatments. Boehringer Ingelheim's expertise in drug discovery and development aims to advance potential medications into clinical trials.
In November 2023, Zevra Therapeutics Inc. acquired Acer Therapeutics Inc. for $91 million, providing Zevra access to rare disease assets, including Edsivo and Olpruva. Edsivo is undergoing Phase III trials for vascular Ehlers-Danlos syndrome, while Olpruva has received FDA approval for certain urea cycle diseases. This acquisition enhances Zevra's market presence and diversifies its revenue stream in the rare disease therapeutics sector. Acer Therapeutics Inc. is a pharmaceutical company offering treatments for urea cycle disorders.
Major companies operating in the urea cycle disorders treatment market report are F. Hoffmann-La Roche AG, AbbVie Inc., Novartis AG, Sanofi S.A., AstraZeneca Plc., Alexion Pharmaceuticals Inc., Abbott Laboratories Inc., GlaxoSmithKline PLC, Takeda Pharmaceutical Co. Ltd., Danone S.A., Merck KGaA, Bausch Health Companies Inc., Grifols S.A., Horizon Therapeutics PLC, Leadiant Biosciences Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Travere Therapeutics Inc., Arcturus Therapeutics Holdings Inc., Selecta Biosciences Inc., Nutricia North America, Acer Therapeutics Inc., Aeglea BioTherapeutics Inc., Synlogic Inc., Lucane Pharma SA, Recordati Rare Diseases Inc., Orpharma Pty Ltd., Mead Johnson & Company LLC.
North America was the largest region in the urea cycle disorders treatment market in 2023. The regions covered in the urea cycle disorders treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the urea cycle disorders treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The main types of treatment for urea cycle disorders include amino acid supplements, sodium phenylbutyrate, glycerol phenylbutyrate, sodium benzoate, and others. Amino acid supplements contain one or more of the nine essential amino acids that the body requires but cannot produce independently. Specific types of enzyme deficiencies are addressed, such as OTC (ornithine transcarbamylase), AS (argininosuccinate synthetase) for citrullinemia, AG (arginase), AL (argininosuccinate lyase), and CPS1 (carbamoyl phosphate synthase). These treatments can be administered orally and through injections, and they are distributed through various channels, including hospital pharmacies, retail pharmacies, and online pharmacies.
The urea cycle disorders treatment market research report is one of a series of new reports that provides urea cycle disorders treatment market statistics, including urea cycle disorders treatment industry global market size, regional shares, competitors with a urea cycle disorders treatment market share, detailed urea cycle disorders treatment market segments, market trends and opportunities, and any further data you may need to thrive in the urea cycle disorders treatment industry. This urea cycle disorders treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The urea cycle disorders treatment market includes revenues earned by entities by hemodialysis, dietary management, and genetic counselling. The market value includes the value of related goods sold by the service provider or included within the service offering. The urea cycle disorders treatment market also includes sales of arginine, sodium phenylacetate, sodium benzoate, and carglumic acid. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The urea cycle disorders treatment market size is expected to see steady growth in the next few years. It will grow to $1.57 billion in 2028 at a compound annual growth rate (CAGR) of 3.9%. The anticipated growth in the forecast period can be attributed to advancements in enzyme replacement therapy, the expansion of newborn screening programs, heightened collaboration in research efforts, the broadening of therapeutic options, and the increasing utilization of telemedicine and remote care. Noteworthy trends expected during the forecast period include the implementation of advanced gene editing techniques, initiatives focused on awareness and advocacy, the exploration of regenerative medicine approaches, the conduct of long-term safety and efficacy studies, and the development of personalized nutritional therapies.
The increasing prevalence of urea cycle disorders is anticipated to drive the growth of the market for urea cycle disorders treatment. Urea cycle disorders are metabolic inborn disorders caused by defects in enzymes or transporter molecules crucial for the liver to eliminate ammonia from the blood. The rise in the prevalence of urea cycle disorders is primarily attributed to genetic disorders. Conditions identified as urea cycle disorders (UCD) involve the accumulation of ammonia, leading to customized treatment strategies that encompass dietary control, medication, and monitoring to manage and mitigate the adverse effects. As of April 2023, the prevalence of urea cycle disorders resulting from ornithine transcarbamylase deficiency was reported as 1 in 140,000 people according to StatPearls Publishing LLC. Additionally, in January 2021, the National Library of Medicine reported 113 new cases of urea cycle disorders per 35,000 live births in the United States. Therefore, the surge in the prevalence of urea cycle disorders is a significant driver of the urea cycle disorders treatment market's growth.
The increasing number of chronic kidney disorder cases is expected to contribute to the expansion of the urea cycle disorders treatment market. Chronic kidney disease (CKD), characterized by the gradual loss of kidney function over time, utilizes urea cycle disorder treatment to manage metabolic waste, especially elevated urea levels due to impaired kidney function. As of November 2023, an estimated 7.2 million individuals are believed to have chronic kidney disease (CKD) stages 1–5, with approximately 3.5 million people in the UK experiencing the later stages of CKD (stages 3-5), where symptoms become progressively challenging to control. Hence, the growing number of chronic kidney disorder cases is a driving force behind the urea cycle disorders treatment market's growth.
A product innovation has emerged as a notable trend, with major companies focusing on creating innovative products to strengthen their market positions. For example, in September 2022, Medunik USA introduced Pheburane oral pellets, a unique and flavor-masking formulation of sodium phenylbutyrate (NaPB) for the long-term management of certain urea cycle disorders. Pheburane, a prescription drug used in conjunction with a specific diet, offers a convenient administration method without the need for mixing. It can be consumed alone or sprinkled on food, enhancing patient compliance.
Major players in the urea cycle disorder treatment market are adopting a strategic partnership approach to advance therapies and improve disease management. In October 2021, Boehringer Ingelheim collaborated with Thoeris GmbH, an early-stage biopharmaceutical company in Europe, to explore novel therapies for patients with urea cycle disorders (UCDs). UCDs, rare diseases resulting from genetic liver dysfunctions leading to elevated ammonia levels, lack sufficient treatments. Boehringer Ingelheim's expertise in drug discovery and development aims to advance potential medications into clinical trials.
In November 2023, Zevra Therapeutics Inc. acquired Acer Therapeutics Inc. for $91 million, providing Zevra access to rare disease assets, including Edsivo and Olpruva. Edsivo is undergoing Phase III trials for vascular Ehlers-Danlos syndrome, while Olpruva has received FDA approval for certain urea cycle diseases. This acquisition enhances Zevra's market presence and diversifies its revenue stream in the rare disease therapeutics sector. Acer Therapeutics Inc. is a pharmaceutical company offering treatments for urea cycle disorders.
Major companies operating in the urea cycle disorders treatment market report are F. Hoffmann-La Roche AG, AbbVie Inc., Novartis AG, Sanofi S.A., AstraZeneca Plc., Alexion Pharmaceuticals Inc., Abbott Laboratories Inc., GlaxoSmithKline PLC, Takeda Pharmaceutical Co. Ltd., Danone S.A., Merck KGaA, Bausch Health Companies Inc., Grifols S.A., Horizon Therapeutics PLC, Leadiant Biosciences Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Travere Therapeutics Inc., Arcturus Therapeutics Holdings Inc., Selecta Biosciences Inc., Nutricia North America, Acer Therapeutics Inc., Aeglea BioTherapeutics Inc., Synlogic Inc., Lucane Pharma SA, Recordati Rare Diseases Inc., Orpharma Pty Ltd., Mead Johnson & Company LLC.
North America was the largest region in the urea cycle disorders treatment market in 2023. The regions covered in the urea cycle disorders treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the urea cycle disorders treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The main types of treatment for urea cycle disorders include amino acid supplements, sodium phenylbutyrate, glycerol phenylbutyrate, sodium benzoate, and others. Amino acid supplements contain one or more of the nine essential amino acids that the body requires but cannot produce independently. Specific types of enzyme deficiencies are addressed, such as OTC (ornithine transcarbamylase), AS (argininosuccinate synthetase) for citrullinemia, AG (arginase), AL (argininosuccinate lyase), and CPS1 (carbamoyl phosphate synthase). These treatments can be administered orally and through injections, and they are distributed through various channels, including hospital pharmacies, retail pharmacies, and online pharmacies.
The urea cycle disorders treatment market research report is one of a series of new reports that provides urea cycle disorders treatment market statistics, including urea cycle disorders treatment industry global market size, regional shares, competitors with a urea cycle disorders treatment market share, detailed urea cycle disorders treatment market segments, market trends and opportunities, and any further data you may need to thrive in the urea cycle disorders treatment industry. This urea cycle disorders treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The urea cycle disorders treatment market includes revenues earned by entities by hemodialysis, dietary management, and genetic counselling. The market value includes the value of related goods sold by the service provider or included within the service offering. The urea cycle disorders treatment market also includes sales of arginine, sodium phenylacetate, sodium benzoate, and carglumic acid. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Urea Cycle Disorders Treatment Market Characteristics3. Urea Cycle Disorders Treatment Market Trends and Strategies32. Global Urea Cycle Disorders Treatment Market Competitive Benchmarking33. Global Urea Cycle Disorders Treatment Market Competitive Dashboard34. Key Mergers and Acquisitions in the Urea Cycle Disorders Treatment Market
4. Urea Cycle Disorders Treatment Market - Macro Economic Scenario
5. Global Urea Cycle Disorders Treatment Market Size and Growth
6. Urea Cycle Disorders Treatment Market Segmentation
7. Urea Cycle Disorders Treatment Market Regional and Country Analysis
8. Asia-Pacific Urea Cycle Disorders Treatment Market
9. China Urea Cycle Disorders Treatment Market
10. India Urea Cycle Disorders Treatment Market
11. Japan Urea Cycle Disorders Treatment Market
12. Australia Urea Cycle Disorders Treatment Market
13. Indonesia Urea Cycle Disorders Treatment Market
14. South Korea Urea Cycle Disorders Treatment Market
15. Western Europe Urea Cycle Disorders Treatment Market
16. UK Urea Cycle Disorders Treatment Market
17. Germany Urea Cycle Disorders Treatment Market
18. France Urea Cycle Disorders Treatment Market
19. Italy Urea Cycle Disorders Treatment Market
20. Spain Urea Cycle Disorders Treatment Market
21. Eastern Europe Urea Cycle Disorders Treatment Market
22. Russia Urea Cycle Disorders Treatment Market
23. North America Urea Cycle Disorders Treatment Market
24. USA Urea Cycle Disorders Treatment Market
25. Canada Urea Cycle Disorders Treatment Market
26. South America Urea Cycle Disorders Treatment Market
27. Brazil Urea Cycle Disorders Treatment Market
28. Middle East Urea Cycle Disorders Treatment Market
29. Africa Urea Cycle Disorders Treatment Market
30. Urea Cycle Disorders Treatment Market Competitive Landscape and Company Profiles
31. Urea Cycle Disorders Treatment Market Other Major and Innovative Companies
35. Urea Cycle Disorders Treatment Market Future Outlook and Potential Analysis
36. Appendix
Executive Summary
Urea Cycle Disorders Treatment Global Market Report 2024 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on urea cycle disorders treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
Reasons to Purchase
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- All data from the report will also be delivered in an excel dashboard format.
Where is the largest and fastest growing market for urea cycle disorders treatment? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? This report answers all these questions and many more.
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include:
- The impact of sanctions, supply chain disruptions, and altered demand for goods and services due to the Russian Ukraine war, impacting various macro-economic factors and parameters in the Eastern European region and its subsequent effect on global markets.
- The impact of higher inflation in many countries and the resulting spike in interest rates.
- The continued but declining impact of COVID-19 on supply chains and consumption patterns.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Report Scope
Markets Covered:1) By Treatment: Amino Acid Supplements; Sodium Phenylbutyrate; Glycerol Phenylbutyrate; Sodium Benzoate; Other Treatments
2) By Enzyme Deficiency: Ornithine Transcarbamylase (OTC); Argininosuccinate Synthetase (AS); Arginase (AG); Argininosuccinate Lyase (AL); Carbamoyl Phosphate Synthase (CPS1)
3) By Route of Administration: Oral; Injectables
4) By Distribution Channel: Hospital Pharmacies; Retail Pharmacies; Online Pharmacies
Key Companies Mentioned: F. Hoffmann-La Roche AG; AbbVie Inc.; Novartis AG; Sanofi S.A.; AstraZeneca Plc.
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes
Delivery Format: PDF, Word and Excel Data Dashboard
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- F. Hoffmann-La Roche AG
- AbbVie Inc.
- Novartis AG
- Sanofi S.A.
- AstraZeneca Plc.
- Alexion Pharmaceuticals Inc.
- Abbott Laboratories Inc.
- GlaxoSmithKline Plc
- Takeda Pharmaceutical Co. Ltd.
- Danone S.A.
- Merck KGaA
- Bausch Health Companies Inc.
- Grifols S.A.
- Horizon Therapeutics Plc
- Leadiant Biosciences Inc.
- BioMarin Pharmaceutical Inc.
- Ultragenyx Pharmaceutical Inc.
- Travere Therapeutics Inc.
- Arcturus Therapeutics Holdings Inc.
- Selecta Biosciences Inc.
- Nutricia North America
- Acer Therapeutics Inc.
- Aeglea BioTherapeutics Inc
- Synlogic Inc.
- Lucane Pharma SA
- Recordati Rare Diseases Inc.
- Orpharma Pty Ltd.
- Mead Johnson & Company LLC
Methodology
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Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 175 |
| Published | February 2024 |
| Forecast Period | 2024 - 2028 |
| Estimated Market Value ( USD | $ 1.35 Billion |
| Forecasted Market Value ( USD | $ 1.57 Billion |
| Compound Annual Growth Rate | 3.9% |
| Regions Covered | Global |
