Urea Cycle Disorders Treatment Global Market Report 2024

The urea cycle disorders treatment market size has grown steadily in recent years. It will grow from $1.29 billion in 2023 to $1.35 billion in 2024 at a compound annual growth rate (CAGR) of 4.5%. The expansion observed in the historical period can be attributed to increased awareness and education, advancements in newborn screening practices, the emergence of patient support networks, and the establishment of 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 $1.57 billion in 2028 at a compound annual growth rate (CAGR) of 3.9%. The anticipated growth in the forecast period can be attributed to advancements in enzyme replacement therapy, the expansion of newborn screening programs, heightened collaboration in research efforts, the broadening of therapeutic options, and the increasing utilization of telemedicine and remote care. Noteworthy trends expected during the forecast period include the implementation of advanced gene editing techniques, initiatives focused on awareness and advocacy, the exploration of regenerative medicine approaches, the conduct of long-term safety and efficacy studies, and the development of personalized nutritional therapies.

The increasing prevalence of urea cycle disorders is anticipated to drive the growth of the market for urea cycle disorders treatment. Urea cycle disorders are metabolic inborn disorders caused by defects in enzymes or transporter molecules crucial for the liver to eliminate ammonia from the blood. The rise in the prevalence of urea cycle disorders is primarily attributed to genetic disorders. Conditions identified as urea cycle disorders (UCD) involve the accumulation of ammonia, leading to customized treatment strategies that encompass dietary control, medication, and monitoring to manage and mitigate the adverse effects. As of April 2023, the prevalence of urea cycle disorders resulting from ornithine transcarbamylase deficiency was reported as 1 in 140,000 people according to StatPearls Publishing LLC. Additionally, in January 2021, the National Library of Medicine reported 113 new cases of urea cycle disorders per 35,000 live births in the United States. Therefore, the surge in the prevalence of urea cycle disorders is a significant driver of the urea cycle disorders treatment market's growth.

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 players in the urea cycle disorder treatment market are adopting a strategic partnership approach to advance therapies and improve disease management. In October 2021, Boehringer Ingelheim collaborated with Thoeris GmbH, an early-stage biopharmaceutical company in Europe, to explore novel therapies for patients with urea cycle disorders (UCDs). UCDs, rare diseases resulting from genetic liver dysfunctions leading to elevated ammonia levels, lack sufficient treatments. Boehringer Ingelheim's expertise in drug discovery and development aims to advance potential medications into clinical trials.

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 report are F. Hoffmann-La Roche AG, AbbVie Inc., Novartis AG, Sanofi S.A., AstraZeneca Plc., Alexion Pharmaceuticals Inc., Abbott Laboratories Inc., GlaxoSmithKline PLC, Takeda Pharmaceutical Co. Ltd., Danone S.A., Merck KGaA, Bausch Health Companies Inc., Grifols S.A., Horizon Therapeutics PLC, Leadiant Biosciences Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Travere Therapeutics Inc., Arcturus Therapeutics Holdings Inc., Selecta Biosciences Inc., Nutricia North America, Acer Therapeutics Inc., Aeglea BioTherapeutics Inc., Synlogic Inc., Lucane Pharma SA, Recordati Rare Diseases Inc., Orpharma Pty Ltd., Mead Johnson & Company LLC.

North America was the largest region in the urea cycle disorders treatment market in 2023. 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.

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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