U.S. Urea Cycle Disorders Treatment market is estimated to be valued at US$ 510.0 million in 2023 and is expected to exhibit a CAGR of 3.56% during the forecast period (2023-2030). Increasing product approvals for urea cycle disorders and increasing number of pipeline products is expected to boost the growth of the market over the forecast period. Moreover, increasing adoption of inorganic growth strategies by key players for Urea Cycle Disorders Treatment by regulatory bodies is expected to propel the growth of the U.S. Urea Cycle Disorders Treatment market over the forecast period
Analysts’ Views on U.S. Urea Cycle Disorders Treatment market:
Manufacturers are focusing on increasing the number of pipeline products for urea cycle disorders and the development of novel treatments for urea cycle disorder, which is expected to propel the market growth of the U.S. urea cycle disorder treatment market over the forecast period.
Figure 1. U.S. Urea Cycle Disorders Treatment Market Share (%), by Treatment Type, 2023
U.S. Urea Cycle Disorders Treatment Market - Driver
Increasing product approvals for urea cycle disorders
Increasing product approvals for urea cycle disorders treatment by regulatory bodies is expected to drive market growth over the forecast period. For instance, in December 2022, Acer Therapeutics Inc., a pharmaceutical company, and its collaboration partner, Relief Therapeutics Holding AG, a commercial-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) had approved OLPRUVA (sodium phenylbutyrate) for oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
Figure 2. U.S. Urea Cycle Disorders Treatment Market Share (%), by Enzyme Deficiency Type, 2023
U.S. Urea Cycle Disorders Treatment Market– Impact of Coronavirus (COVID-19) Pandemic
Supply chain and manufacturing activities in the U.S. have been disrupted due to lockdowns implemented by governments in the past year, and also facing problems with regards to the transportation of drug products. The coronavirus or COVID-19 outbreak that started in Wuhan, China has spread across continents, affecting various industries globally. The supply of key materials has been severely disrupted due to forced quarantine, and a lack of labor and raw materials. As the link between regional warehouses is not smooth, the transportation of raw materials between regions cannot be carried out successfully. This shortage of raw materials and components has affected the supply chain of the U.S. urea cycle disorders treatment market. Due to the severe shortage of medical resources at the front line, only patients diagnosed with serious conditions can be hospitalized. Unfortunately, the pathogenic mechanism of the virus has not been identified completely, therefore, there is no specific drug and treatment except for symptomatic and supportive treatments. Respiratory support devices such as life-support machines, atomizers, oxygen generators, and monitors are primary clinical treatment medical devices. Thus, from diagnosis to cure, the need for instruments for measuring temperature, nucleic acid diagnostic kits, antiviral medical products, and life-support machines has increased consistently. Furthermore, the COVID-19 pandemic has impacted the global as well as the U.S. economy and in turn, the urea cycle disorders treatment market.
|Base Year:||2022||Market Size in 2023:||US$ 510.0 Mn|
|Historical Data for:||2017 to 2021||Forecast Period:||2023 to 2030|
|Forecast Period 2023 to 2030 CAGR:||3.56%||2030 Value Projection:||US$ 651.5 Mn|
Horizon Therapeutics Plc, Bausch Health Companies Inc, Recordati Rare Diseases Inc., Nestlé S.A., Danone S.A., Lucane Pharma SA, Acer Therapeutics Inc., Ultragenyx Pharmaceutical Inc., Aeglea Biotherapeutics, Inc., Arcturus Therapeutics Holdings Inc., Orpharma Pty Ltd., Selecta Biosciences, Inc., Abbott, and Mead Johnson & Company, LLC
|Restraints & Challenges:||
U.S. Urea Cycle Disorders Treatment Market Segmentation:
U.S. Urea Cycle Disorders Treatment market is segmented into Treatment Type, Enzyme Deficiency Type, Route of Administration, and Distribution Channel
Based on Treatment Type, the market is segmented into Amino Acid Supplements, Sodium Phenylbutyrate, Glycerol Phenylbutyrate, Sodium Benzoate, and Others. Due to key players focusing on strategies such as product acquisition to strengthen their product portfolio for the treatment of UCDs, the Glycerol Phenylbutyrate segment accounted for a significant share of 62.8% in the U.S. market in 2023.
In terms of Enzyme Deficiency Type, the U.S. Urea Cycle Disorders Treatment market has been segmented into OTC – Ornithine Transcarbamylase, AS – Argininosuccinate Synthetase (citrullinemia), AG – Arginase, AL – Argininosuccinate Lyase, CPS1 – Carbamoyl Phosphate Synthase, and NAGS – N-acetylglutamate Synthase. Due to increasing incidence of the N-acetylglutamate synthase deficiency in the U.S., N-acetylglutamate Synthase (NAGS) segment is expected to dominate the market over the forecast period.
On the basis of Route of Administration, the market has been divided into Oral and Injectable. Due to the Increasing approvals of oral suspension by regulatory bodies, the Oral segment is expected to dominate the market over the forecast period.
On the basis of Distribution Channel, the market has been divided into Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies. Due to major players are focusing on running special awareness programs for the treatment of urea cycle disorders through healthcare facilities, the Hospital pharmacies segment expected to dominate the market over the forecast period.
U.S. Urea Cycle Disorders Treatment Market: Key Developments
Increasing collaboration for urea cycle disorders treatment by market players is expected to propel the growth of the U.S. Urea Cycle Disorders Treatment market over the forecast period. For instance, in January 2021, Recordati Rare Diseases Inc., a biopharmaceutical company, received the U.S. Food and Drug Administration (FDA) approval for CARBAGLU for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA) in pediatric. CARBAGLU was previously approved by U.S. Food Drug and Administration in the year 2021 for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency.
U.S. Urea Cycle Disorders Treatment Market: Restraint
High Cost Of Urea Cycle Disorders Treatment
Drugs such as RAVICTI is a nitrogen-binding agents approved for the treatment of chronic management of urea cycle disorder (UCD) in adults and children. RAVICTI is developed by Horizon Therapeutics plc. a global biotechnology company, and it costs around US$ 793,632 for a year of treatment of a patient suffering from urea cycle disorder. RAVICTI is one of the most expensive drugs available in the U.S. for the treatment of UCD.
U.S. Urea Cycle Disorders Treatment Market - Key Players
Major players operating in the U.S. Urea Cycle Disorders Treatment market include Horizon Therapeutics Plc, Bausch Health Companies Inc, Recordati Rare Diseases Inc., Nestlé S.A., Danone S.A., Lucane Pharma SA, Acer Therapeutics Inc., Ultragenyx Pharmaceutical Inc., Aeglea Biotherapeutics, Inc., Arcturus Therapeutics Holdings Inc., Orpharma Pty Ltd., Selecta Biosciences, Inc., Abbott and Mead Johnson & Company, LLC
Definition: Urea cycle disorders (UCDs) are a group of orphan inherited defects of six enzymes and two transporters that constitute the urea cycle in the periportal liver cells that affect how the body removes the waste that is made from the breaking down of protein. The symptoms of urea cycle disorders vary in severity and result from the excessive accumulation of ammonia in the blood and body tissues called hyperammonemia. Common symptoms include lack of appetite, vomiting, drowsiness, seizures, and/or coma. In severe cases, if the urea cycle disorder the liver may be abnormally enlarged, which is called hepatomegaly
Urea cycle disorders are congenital diseases that are caused by a dysfunctional urea cycle. Urea cycle disorders are the result of a deficiency of one of the six enzymes that catalyze the various biochemical reactions in the urea cycle which converts ammonia to urea for removal via urine. Urea cycle disorders belong to inborn errors of metabolism that are associated with fatal brain damage and death in newborns. The diagnosis of urea cycle disorder is done by the analysis of urine and blood for abnormal metabolites such as substances produced by metabolism and high ammonia levels. Frequent blood tests are done to monitor ammonia levels. The treatment of urea cycle disorders consists of dietary management to limit ammonia production. A liver transplant can be an effective treatment for urea cycle disorder for the proper functioning of the urea cycle
Increasing product approvals by regulatory bodies which is expected to drive the market growth over the forecast period. For instance, In October 2020, Aeglea Biotherapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to ACN00177 for the treatment of Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine, which leads to a wide range of life-altering complications and reduced life expectancy.
Increasing product launches by key players for ornithine transcarbamylase (OTC) deficiency treatment is expected to drive market growth over the forecast period. For instance, in September 2022, Medunik USA, a pharmaceutical company, announced the launch of Pheburane oral pellets, a unique taste-masked formulation of sodium phenylbutyrate (NaPB). Pheburane is a prescription medicine, used along with a specific diet, for the long-term management of adults and children with urea cycle disorders (UCDs).
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.
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