Urea Cycle Disorders Treatment Market Report 2025

The urea cycle disorders treatment market size has grown steadily in recent years. It will grow from $1.33 billion in 2024 to $1.39 billion in 2025 at a compound annual growth rate (CAGR) of 4%. The growth in the historic period can be attributed to awareness and education, improvements in newborn screening, emergence of patient support networks, 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 $1.65 billion in 2029 at a compound annual growth rate (CAGR) of 4.5%. The growth in the forecast period can be attributed to advancements in enzyme replacement therapy, expanded newborn screening programs, increased collaboration in research, expansion of therapeutic options, telemedicine and remote care. Major trends in the forecast period include advanced gene editing techniques, awareness and advocacy initiatives, regenerative medicine approaches, long-term safety and efficacy studies, and personalized nutritional therapies.

The increasing prevalence of urea cycle disorders is projected to drive the growth of the urea cycle disorders treatment market in the future. Urea cycle disorders refer to metabolic conditions that are inherited and result from defects in one of the enzymes or transporter molecules essential for the liver's removal of ammonia from the bloodstream. The rise in these disorders is primarily attributed to genetic factors. When ammonia accumulation is identified as a urea cycle disorder (UCD), a tailored treatment approach involving dietary management, medication, and monitoring can be established to mitigate its adverse effects. For example, in April 2023, data from StatPearls Publishing LLC, a US-based medical education resource, indicated that the prevalence of urea cycle disorders due to ornithine transcarbamylase deficiency is 1 in 140,000 individuals. Additionally, in September 2022, according to Lecturio, a Germany-based digital medical education platform, Urea cycle disorders (UCDs) occur in about 1 in every 35,000 live births globally and 1 in 8,200 live births in the United States, resulting in an annual incidence of roughly 113 new cases in the U.S. and around 149 in Europe. Therefore, the rising prevalence of urea cycle disorders is fueling the growth of the urea cycle disorders treatment market.

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 companies in the urea cycle disorder treatment sector are embracing a strategic partnership approach to enhance innovative therapies and improve disease management. Strategic partnerships involve companies utilizing each other's strengths and resources to achieve mutual advantages and success. For example, in June 2024, Orsini Specialty Pharmacy, a US-based provider of personalized care and medications, and Zevra Therapeutics Inc., a US-based company focused on rare disease therapeutics, announced that Orsini will serve as the pharmacy partner for OLPRUVA (sodium phenylbutyrate) oral suspension. OLPRUVA is a prescription medication used alongside specific therapies, including dietary modifications, for the long-term management of urea cycle disorders (UCDs) in particular adult and pediatric patients with deficiencies in carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

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 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.

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. 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.

Urea cycle disorder treatment involves managing rare genetic diseases in which the body lacks the necessary enzymes to break down ammonia, leading to its accumulation and potential toxicity. The primary goal of urea cycle disorder treatment is to reduce blood ammonia levels.

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.

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