What Has Driven the Growth of the Urea Cycle Disorders Treatment Market in Recent Years?
The urea cycle disorders treatment market has seen considerable growth due to a variety of factors.
•The market size for treatment of urea cycle disorders has experienced a consistent expansion in recent times. It is projected to rise from $1.33 billion in 2024 to approximately $1.39 billion in 2025, demonstrating a compound annual growth rate (CAGR) of 4.0%.
Factors contributing to the growth observed in the historic period include increased awareness and education, enhancements in newborn screening procedures, the development of patient support networks, and the establishment of standard care guidelines.
What is the Market Size Forecast for the Urea Cycle Disorders Treatment Market?
The urea cycle disorders treatment market is expected to maintain its strong growth trajectory in upcoming years.
• The market size for treatment of urea cycle disorders is set to experience consistent growth in the upcoming years, expanding to a value of $1.65 billion by 2029 with a compound annual growth rate (CAGR) of 4.5%.
The projected growth in the coming period is fueled by improvements in enzyme replacement therapy, wider newborn screening programs, escalated research cooperation, proliferation of therapeutic solutions, telemedicine, and remote healthcare. Some of the significant trends during this forecast period are progressive gene editing techniques, initiatives for awareness and advocacy, methods in regenerative medicine, long-term safety and effectiveness studies, and tailored nutritional therapies.
What Are The Core Factors Supporting Growth In The Urea Cycle Disorders Treatment Market?
The increase in instances of urea cycle disorders is projected to boost the expansion of the urea cycle disorders treatment market. Urea cycle disorder is a form of metabolic disorder that is congenital and occurs due to defects in one of the enzymes or transport molecules essential for the liver to eliminate ammonia from the bloodstream. This rise is attributable to genetic disorders. If a urea cycle disorder (UCD) is identified as causing ammonia build-up, a personalised treatment approach, including dietary management, medication, and routine checks, may be adopted to mitigate its detrimental effects. For example, StatPearls Publishing LLC, a US medical education library, reported in April 2023 that the prevalence of urea cycle disorders caused by ornithine transcarbamylase deficiency was 1 in 140,000 individuals. Moreover, according to German digital medical education platform Lecturio, in September 2022, Urea cycle disorders (UCDs) happen in roughly 1 in every 35,000 live births worldwide and 1 in 8,200 live births in the US. This leads to an annual incidence of around 113 new cases in the U.S. and approximately 149 in Europe. As such, the increasing incidence of urea cycle disorders is a key factor in driving the growth of the urea cycle disorders treatment market.
What Are The Major Segments Within The Global 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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Which Trends Are Expected To Transform The Urea Cycle Disorders Treatment Market?
The urea cycle disorder treatment market is seeing an increased focus on product innovation as a primary trend. A majority of the major players in this market are striving to develop novel products to solidify their market position. For example, Medunik USA, a pharmaceutical company based in the US, in September 2022, launched an innovative flavor-enhancing formulation known as Pheburane oral pellets. This sodium phenylbutyrate (NaPB) formulation is used for the long-term treatment of certain urea cycle disorders. Suited for both adults and children, it is specifically used to treat urea cycle disorders characterized by a deficiency in carbamyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinate synthetase (AS). Its dosage is measured by a reusable, calibrated dosing spoon included in the package. Unlike its counterparts, Pheburane does not require oral consumption or mixing. It can be eaten with a meal or a snack, sprinkled over apple sauce or carrot puree, or combined with water, fruit juice, or protein-free infant formula.
Who Are the Key Players in the Urea Cycle Disorders Treatment Market?
Major companies operating in the urea cycle disorders treatment market 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
• Mead Johnson & Company
• LLC
• Horizon Therapeutics Plc
• Nestlé S.A.
• Danone S.A.
• Biophone
• Relief Therapeutics Holding AG
• TheraTriage
• Genomatica
• Protalix BioTherapeutics
• Erytech Pharma
• 4D Pharma
• Synlogic
• Amicus Therapeutics
• Sobi
• Astellas Pharma Inc.
• Chiesi Farmaceutici S.p.A.
• Medpace Holdings
• Inc.
• Celerion
• and PTC Therapeutics
What Is The Most Dominant Region In The Urea Cycle Disorders Treatment Market?
North America was the largest region in the urea cycle disorders treatment market in 2024. 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.
