Urea Cycle Disorder Treatment Market Analysis North America, Europe, Asia, Rest of World (ROW) - US, China, Germany, UK, Canada - Size and Forecast 2024-2028

Urea Cycle Disorder Treatment Market Size 2024-2028

The urea cycle disorder treatment market size is estimated to grow by USD 215.4 million at a CAGR of 3.2% between 2023 and 2028. Urea cycle disorders (UCDs) are metabolic conditions that impair the normal processing of ammonia in the body, leading to a dangerous build-up of this toxic substance. The prevalence of UCDs is increasing due to advancements in newborn screening programs and improved diagnostic techniques. Furthermore, the aging population is at a higher risk of developing UCDs due to the natural decline in kidney function. In response to these trends, the biotechnology industry is focusing on strategic initiatives to develop novel treatments and therapies for UCDs. These include enzyme replacement therapies, gene therapies, and dietary interventions. By investing in research and development, companies can address the unmet medical needs of patients with UCDs and improve their quality of life. It also includes an in-depth analysis of drivers, trends, and challenges. 

What will be the size of the Market During the Forecast Period?

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Market Segmentation

Therapy Analysis

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 374.20 million in 2018.  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.

Regional Analysis

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North America is estimated to contribute 39% 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 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 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 market and technological advancements in the treatment of urea cycle disorder are helping in addressing the existing challenges and also contributing to regional NAGS N acetylglutamate Synthase 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 market. Hence, such factors will boost regional market growth during the forecast period.

Key Market Players 

Companies 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 companies, including:

Abbott Laboratories - The company offers urea cycle disorder drugs such as Cyclinex-1.

• 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.
• Ultragenyx Pharmaceutical Inc.

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.

Market Dynamics and Customer Landscape

Urea Cycle Disorders (UCDs) are rare metabolic conditions that affect the production of urea, leading to the accumulation of toxic ammonia in the blood. Treatment for UCDs primarily focuses on managing hyperammonemia and preventing further ammonia buildup. Pharmaceutical sector and biotech companies are investing heavily in R&D to develop effective therapies for UCDs. Ornithine Transcarbamylase Deficiency (OTCD) is the most common type of UCD. Treatment options include protein restriction, amino acid supplements like glycerol phenylbutyrate and sodium benzoate, and enzyme replacement therapies. OTC Ornithine Transcarbamylase is also available. Acer Therapeutics Inc. is a leading company in the UCD treatment market, focusing on developing and commercializing therapies for various UCDs. Pharmacies play a crucial role in the distribution of UCD treatments. These include hospital pharmacies, retail pharmacies, and online pharmacies. Statpearls, a medical database, provides comprehensive information on UCDs and their treatments. Other key players in the market include AS Argininosuccinate Synthetase, which develops oral and injectable treatments for UCDs. The EU member states have implemented various initiatives to ensure the availability and accessibility of UCD treatments.

Key Market Driver

One of the key factors driving the 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.

Significant Market Trends

The increasing growth potential in emerging economies is a key trend in the market. Developing countries like India and Wuhan, China have massive 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. 

Major Market Challenges

The low access to specialized care is challenging the 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 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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Key Market Customer Landscape

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.

Market Customer Landscape

Segment Overview

The market research report provides comprehensive data (region wise segment analysis), with forecasts and estimates in "USD Million" for the period 2024 to 2028, as well as historical data from 2018 to 2022 for the following segments.

• Therapy Outlook
  • Glycerol phenylbutyrate
  • Sodium phenylbutyrate
  • Amino acid supplements
  • Sodium benzoate
  • Others

• Route Of Administration Outlook
  • Oral
  • Injectables

• Geography Outlook
  • North America
    • The U.S.
    • Canada
  • Europe
    • The U.K.
    • Germany
    • France
    • Rest of Europe
  • Asia
    • China
    • India
  • Rest of World
    • Australia
    • Argentina
    • Brazil

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Market Analyst Overview

The market is rapidly expanding, driven by advancements in R&D spending and innovations from research institutes. Key players like Thoeris GmbH and Relief Therapeutics Holding AG are pushing the envelope with new technologies and power assist devices. Emerging products such as battery-operated electric wheelchairs are being developed to better meet the needs of disabled populations, including those affected by cerebral palsy and mobility impairment disorders. The electric wheelchair segment is growing, with a notable focus on pediatric segment solutions and bariatric wheelchairs for a diverse range of users. Homecare and nursing homes are increasingly adopting advanced mobility devices to enhance home care settings and support personal users. Innovations are also seen in sports wheelchairs, offering improved functionality for users with developmental disabilities and cardiovascular disease. Enhanced medical devices are crucial for rehabilitation centers and hospitals and clinics, ensuring greater independence and quality of life for individuals with various mobility needs.

Further, the market is seeing significant advancements, driven by ongoing R&D spending and collaboration between research institutes and pharmaceutical companies. Disorders like ornithine transcarbamylas (OTC) deficiency and N acetyl glutamate synthase (NAGS) deficiency are key focus areas. Treatments such as Carglumic acid, known commercially as CARBAGLU, and OLPRUVA (sodium phenylbutyrate) are critical for managing these inborn errors of metabolism. The National Library of Medicine and the UK Health Research Authority provide valuable resources and data supporting research into enzyme deficiency type and their impacts on hepatic function and hepatomegaly. These conditions are increasingly relevant due to the COVID-19 outbreak, which has highlighted the need for robust treatments in managing complex metabolic disorders. As research continues, the development of new therapies aims to address the metabolic challenges posed by these disorders, improving patient outcomes and quality of life.

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What are the Key Data Covered in this Market Forecasting Report?

• CAGR of the market during the forecast period
• Detailed information on factors that will drive the market forecast report of the market between 2024 and 2028
• Precise estimation of the market size and its contribution to the parent market
• Accurate predictions about upcoming market trends and analysis and changes in consumer behavior
• Growth of the market across Europe, North America, APAC, South America, and Middle East and Africa
• Thorough market growth analysis of the market’s competitive landscape and detailed information about companies
• Comprehensive market analysis and report on the factors that will challenge the market research and growth of market companies

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