Urea Cycle Disorders Treatment Market Projected to Reach $1.62 Billion with 4.2% CAGR by 2029

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The Business Research Company’s Urea Cycle Disorders Treatment Global Market Report 2025 – Market Size, Trends, And Forecast 2025-2034

LONDON, GREATER LONDON, UNITED KINGDOM, December 9, 2025 /EINPresswire.com/ -- What Is The Expected Cagr For The Urea Cycle Disorders Treatment Market Through 2025?
In recent times, the market size for treatment of urea cycle disorders has experienced consistent growth. The market is projected to escalate from $1.33 billion in 2024 to $1.37 billion in 2025, exhibiting a compound annual growth rate (CAGR) of 2.9%. This historical growth trajectory is attributable to factors such as increased awareness and education, enhancements in newborn screening, the rise of patient support networks, and the establishment of standard care guidelines.

In the forthcoming years, there will likely be consistent expansion in the size of the urea cycle disorders treatment market. It is projected to develop until it reaches a value of $1.62 billion in 2029, growing at a compound annual growth rate (CAGR) of 4.2%. The predicted growth during this forecast period can be linked to progresses in enzyme replacement therapy, broader newborn screening programs, escalation in research collaborations, enhancement in therapeutic options, and the introduction of telemedicine and remote care. Significant trends during this forecast period encompass breakthroughs in gene editing techniques, initiatives for awareness and advocacy, strategies in regenerative medicine, in-depth safety and efficacy studies, as well as personalized nutritional therapies.

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What Are The Key Factors Driving Growth In The Urea Cycle Disorders Treatment Market?
The expanding occurrence of urea cycle disorders is anticipated to stimulate the expansion of the market for treatment of these conditions. Urea cycle disorders are congenital metabolic conditions triggered by defects in one or more enzymes or transporter molecules required for the liver to eliminate ammonia from the bloodstream. The surge in occurrences of these disorders is due to genetic abnormalities. Customized treatment strategies including dietary management, medication, and monitoring can be applied to control and mitigate the adverse effects when an ammonia build-up is detected as a urea cycle disorder (UCD). For example, StatPearls Publishing LLC, a US medical education library, reported in April 2023 that urea cycle disorders resulting from ornithine transcarbamylase deficiency affected 1 out of 140,000 people. In addition, in September 2022, Lecturio, a German digital medical education platform, indicated that worldwide, urea cycle disorders (UCDs) are found in roughly 1 in every 35,000 live births and in the United States, 1 in 8,200 live births, giving rise to an annual incidence of around 113 new cases in the U.S. and roughly 149 in Europe. Consequently, it is this growing prevalence of urea cycle disorders that is propelling the advancement of the treatment market for these disorders.

What Are The Top Players Operating In The Urea Cycle Disorders Treatment Market?
Major players in the Urea Cycle Disorders Treatment include:
• Bausch Health Companies Inc.
• Recordati Rare Diseases
• Eurocept Pharmaceuticals Holding (Lucane Pharma SA)
• Acer Therapeutics
• Ultragenyx Pharmaceutical
• Aeglea BioTherapeutics
• Arcturus Therapeutics, Inc.
• Orpharma Pty Ltd.
• Selecta Biosciences, Inc.
• Abbott Laboratories

What Are The Key Trends Shaping The Urea Cycle Disorders Treatment Industry?
The trend of product innovation is becoming increasingly notable in the market for urea cycle disorder treatment. Aiming to secure a strong market position, key companies in this industry are focusing on the development of innovative products. In a representative example from September 2022, Medunik USA, an American pharmaceutical corporation, introduced Pheburane oral pellets to the market. This novel, flavor-concealing NaPB formulation was designed for prolonged management of specific types of urea cycle disorders. This prescription medication, which is meant to be used with a specific diet, treats urea cycle disorders, namely those relating to deficiencies of CPS, OTC, or AS in both adults and children. Unlike other NaPB variants, there's no need for mixing or oral consumption as it comes with a reusable, calibrated dosing spoon for easy measuring. Pheburane can be eaten along with a meal or snack. Alternatively, it can be sprinkled on a spoonful of apple sauce or carrot puree and followed by drinking water, fruit juice, or protein-free infant formula.

Comprehensive Segment-Wise Insights Into The Urea Cycle Disorders Treatment Market
The urea cycle disorders treatment market covered in this report is segmented –

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

Subsegments:
1) By Amino Acid Supplements: Arginine Supplements, Citrulline Supplements, Other Essential Amino Acids
2) By Sodium Phenylbutyrate: Powder Form, Tablet Form
3) By Glycerol Phenylbutyrate: Oral Solutions, Other Forms
4) By Sodium Benzoate: Intravenous Solutions, Oral Formulations
5) By Other Treatments: Liver Transplantation, Dietary Modifications, Enzyme Replacement Therapy, Other Supportive Therapies

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Global Urea Cycle Disorders Treatment Market - Regional Insights
In 2024, North America led the market for urea cycle disorders treatment. The market report for urea cycle disorders treatment included areas such as Asia-Pacific, Western Europe, Eastern Europe, South America, the Middle East, and Africa.

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