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The urea cycle disorder (UCDs) treatment market size is estimated to grow by USD 235.5 million at a CAGR of 3.57% between 2022 and 2027.
Hyperammonemia is a metabolic sickness characterized by expanded levels of the nitrogen compound ammonia. Hence, disruption of the urea cycle will result in the accumulation of ammonia which is toxic to the central nervous system. Despite the condition being rare, there is a necessity to recognize effective treatments to control the impact of the disease. Additionally, R&D studies that focus on the genetic features of urea cycle disorders shall help vendors in product development, which will expand the scope of the market. Moreover, studies have displayed that patients with early-onset disease suffer high morbidity while those with late-onset disease suffer less severely and have one or more acute episodes. Hence, such studies will help in better comprehension of the various stages of the disease and effective treatments, that will have a positive impact on the market growth.
Technavio has segmented the market into Therapy, Route of Administration, and Geography.
It also includes an in-depth analysis of drivers, trends, and challenges.
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The glycerol phenylbutyrate segment is estimated to witness significant growth during the forecast period. RAVICTI or glycerol phenylbutyrate is an oral solution that is used for the long-term treatment of high ammonia concentration in the blood (hyperammonemia). This is primarily caused by urea cycle disorder (UCD). However, glycerol phenylbutyrate must only be chosen as a last resort after other options like a low-protein diet and supplements have been exhausted. Moreover, if glycerol phenylbutyrate is used for treatment, the patient must ensure it is along with a low-protein diet.
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The glycerol phenylbutyrate segment was the largest segment and was valued at USD 366.40 million in 2017. It has been observed that a low-protein diet and sometimes using supplements along with glycerol phenylbutyrate can help maintain ammonia levels in infants, children, and adults with urea cycle disorders. Moreover, it is an easy-to-swallow liquid medicine and is odorless, making it easy to administer. The presence of numerous vendors such as Biophone, Nuray, and MSN are also likely to contribute to the market growth of this segment during the forecast period.
The oral segment includes medications in the form of tablets, capsules, chewable or orally disintegrating tablets. It can also be taken in the form of drops, syrups, or solutions. The most common treatment options are glycerol phenylbutyrate or sodium phenylbutyrate which help in eliminating excess ammonia levels in the blood and help control hyperammonemia. Moreover, L-Arginine is used for catalyzing enzymes of the urea cycle along with antacids to help limit gastrointestinal side effects like heartburn and abdominal pain. This segment is the most preferred due to ease of availability and consumption in a variety of liquid and solid forms which will result in the market growth of this segment during the forecast period.
Injectables play a significant role in the treatment of UCDs. There is an increasing adoption of this segment for specific situations, especially during acute hyperammonemia or when precise drug delivery is necessary. Intravenous (IV) injections comprise the direct administration of medications or therapeutic solutions into the bloodstream through a vein in the body. The main advantage of this route is that it enables the rapid absorption and distribution of the drug throughout the body. Several medications including sodium benzoate or sodium phenylacetate are carried out intravenously to ensure the immediate reduction of ammonia levels, in in order to prevent neurological damage or organ failure. Hence, such factors are fuelling the growth of this segment which in turn will drive the market growth during the forecast period.
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North America is estimated to contribute 36% to the growth of the global market during the forecast period. Technavio’s analysts have elaborately explained the regional trends and drivers that will shape the market during the forecast period. The main drivers of the urea cycle disorder treatment market in North America include the increasing product launches and other strategic initiatives such as mergers and acquisitions by market vendors in the region. In the US, besides these factors, the increasing prevalence of urea cycle disorders compared to other regions will also be responsible for further growth of the urea cycle disorder treatment market during the forecast period. As per a study published in January 2021 titled "Management of Tardive Urea Cycle Disorders - A Remaining Challenge for Critical Care Physicians", there are 113 UCD patients in the US annually.
However, factors, like increased R&D into the Urea Cycle Disorder treatment market and technological advancements in the treatment of urea cycle disorder, are helping in addressing the existing challenges and also contributing to regional UCD treatment market growth. For instance, in October 2021, ACER-001 (sodium phenylbutyrate) by Acer Therapeutics Inc. was approved by the US Food and Drug Administration (FDA) for therapeutic purposes for urea cycle disorder patients, which shows further scope of the urea cycle disorder treatment market. Hence, such factors will boost regional market growth during the forecast period.
The COVID-19 outbreak has had a mixed impact on the market as COVID-19 drugs interfered with the urea cycle and resulted in certain enzyme deficiencies in certain enzymes while also causing closures of manufacturing plants. Shortages of skilled workers also resulted in reduced production of UCD treatment drugs. However, after large-scale vaccination drives, manufacturing units restarted operations, and hence the regional market is predicted to grow further during the forecast period.
Vendors are implementing various strategies, such as strategic alliances, partnerships, mergers and acquisitions, geographical expansion, and product/service launches, to enhance their presence in the market. The report also includes detailed analyses of the competitive landscape of the market and information about 19 market vendors, including:
Abbott Laboratories - The company offers urea cycle disorder drugs such as Cyclinex-1.
Qualitative and quantitative analysis of vendors has been conducted to help clients understand the wider business environment as well as the strengths and weaknesses of key market players. Data is qualitatively analyzed to categorize vendors as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize vendors as dominant, leading, strong, tentative, and weak.
There are multiple factors influencing market growth. Our researchers analyzed the data with 2022 as the base year, along with the key trends, and challenges.
One of the key factors driving the urea cycle disorder treatment market growth is the rising geriatric patients. There is an increasing growth of the global UCD treatment market fuelled by the growing focus on early screening and diagnosis of rare diseases such as UCDs. The rising focus on early detection is an essential element in enhancing the overall management and treatment of UCDs.
Moreover, early screening and diagnosis play a crucial role in determining UCDs at their primary stages, even before symptoms become too serious. Additionally, it has become possible to detect these rare genetic metabolic disorders more accurately and efficiently due to the advancements in medical technology and genetic testing. Hence, such factors are positively impacting the urea cycle disorder (UCDs) treatment market. Therefore, it is expected to drive the market growth during the forecast period.
The increasing growth potential in emerging economies is a key trend in the urea cycle disorder treatment market. Developing countries like India and China have massive Urea Cycle Disorder treatment market growth potential due to the rising healthcare expenditure, increasing number of hospitals, and increasing incidence of lifestyle diseases. Other factors that drive the UCD treatment market in such regions are the presence of several raw material suppliers, lower labor costs, and relative ease of acquiring raw materials.
Additionally, the increasing number of hospitals and clinics in emerging economies is also resulting in rising demand for advanced screening and diagnostic procedures to improve patient outcomes. The expanding healthcare requirements of the growing population to treat diverse medical conditions shall also propel the demand for medical instruments and devices, including those to treat UCD. Hence, such factors will boost the growth of the market during the forecast period.
The low access to specialized care is challenging the urea cycle disorder treatment market growth. The treatment of medical conditions such as UCD requires long-term, specialized care from experienced healthcare providers who are skilled in the treatment of rare disorders like UCD. Moreover, they require treatment in specialized centers and large hospitals and clinics, which are limited in developing countries or remote areas, which limits the growth of the UCD treatment market.
Hence, this often results in delayed diagnosis and suboptimal and infrequent treatment. As a consequence, patients with conditions like UCD have limited support and inefficient treatment, which hamper the growth of the urea cycle disorder treatment market. Hence factors like the reduced number of qualified and skilled healthcare providers and few specialized centers for treatments of UCD pose a hindrance to market growth during the forecast period.
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The market report includes the adoption lifecycle of the market, covering from the innovator’s stage to the laggard’s stage. It focuses on adoption rates in different regions based on penetration. Furthermore, the report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their growth strategies.
Global Urea Cycle Disorder Treatment Market Customer Landscape
The urea cycle disorder treatment market report forecasts market growth by revenue at global, regional & country levels and provides an analysis of the latest trends and growth opportunities from 2017 to 2027.
Urea Cycle Disorder Treatment Market Scope |
|
Report Coverage |
Details |
Page number |
153 |
Base year |
2022 |
Historic period |
2017-2021 |
Forecast period |
2023-2027 |
Growth momentum & CAGR |
Accelerate at a CAGR of 3.57% |
Market growth 2023-2027 |
USD 235.5 million |
Market structure |
Fragmented |
YoY growth 2022-2023(%) |
2.87 |
Regional analysis |
North America, Europe, Asia, and the Rest of the World (ROW) |
Performing market contribution |
North America at 36% |
Key countries |
US, Canada, Germany, UK, and China |
Competitive landscape |
Leading Vendors, Market Positioning of Vendors, Competitive Strategies, and Industry Risks |
Key companies profiled |
Abbott Laboratories, Acer Therapeutics Inc., Aeglea BioTherapeutics Inc., Arcturus Therapeutics Holdings Inc., Bausch Health Co. Inc., Boehringer Ingelheim International GmbH, CAMP4, Dipharma SA, Eurocept B.V., Horizon Therapeutics Plc, Immedica Pharma AB, Medunik USA, Nestle SA, Orpharma Pty Ltd., Reckitt Benckiser Group Plc, Recordati S.p.A, RELIEF THERAPEUTICS Holding SA, Swedish Orphan Biovitrum AB, Synlogic Inc., and Ultragenyx Pharmaceutical Inc. |
Market dynamics |
Parent market analysis, Market growth inducers and obstacles, Fast-growing and slow-growing segment analysis, COVID-19 impact and recovery analysis and future consumer dynamics, and Market condition analysis for the forecast period. |
Customization purview |
If our report has not included the data that you are looking for, you can reach out to our analysts and get segments customized. |
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1 Executive Summary
2 Market Landscape
3 Market Sizing
4 Historic Market Size
5 Five Forces Analysis
6 Market Segmentation by Therapy
7 Market Segmentation by Route of Administration
8 Customer Landscape
9 Geographic Landscape
10 Drivers, Challenges, and Trends
11 Vendor Landscape
12 Vendor Analysis
13 Appendix
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