U.S. Urea Cycle Disorders Treatment Market

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

Market Dynamics

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:

• This report provides in-depth analysis of the U.S. urea cycle disorders market, and provides market size (US$ Million) and compound annual growth rate (CAGR%) for the forecast period (2023–2030), considering 2022 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends,  regional outlook, and competitive strategies adopted by key players
• It profiles key players in the U.S. urea cycle disorders market based on the following parameters – company highlights, products portfolio, key highlights, financial performance, and strategies Key companies covered as a part of this study 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
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The U.S. urea cycle disorders market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the U.S. urea cycle disorders market

Detailed Segmentation:

• U.S. Urea Cycle Disorders Treatment Market, By Treatment Type:
  • Amino Acid Supplements
  • Sodium Phenylbutyrate
  • Glycerol Phenylbutyrate
  • Sodium Benzoate
  • Others
• U.S. Urea Cycle Disorders Treatment Market, By Enzyme Deficiency Type :
  • OTC – Ornithine Transcarbamylase
  • AS – Argininosuccinate Synthetase (citrullinemia)
  • AG – Arginase
  • AL – Argininosuccinate Lyase
  • CPS1 – Carbamoyl Phosphate Synthase
  • NAGS – N-acetylglutamate Synthase
• U.S. Urea Cycle Disorders Treatment Market, By Route of Administration:
  • Oral
  • Injectable
• U.S. Urea Cycle Disorders Treatment Market, By Distribution channel:
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
• Company Profiles
  • Horizon Therapeutics Plc.*
    • Company Highlights
    • Products Portfolio
    • Key Highlights
    • Financial Performance
    • Strategies
  • Bausch Health Companies Inc.
  • Recordati Rare Diseases Inc.
  • Nestlé S.A.
  • Danone S.A.
  • Lucane Pharma SA
  • Acer Therapeutics Inc.
  • Ultragenyx Pharmaceutical
  • Aeglea Biotherapeutics, Inc
  • Arcturus Therapeutics Holdings Inc.
  • Orpharma Pty Ltd.
  • Selecta Biosciences, Inc.
  • Abbott
  • Mead Johnson & Company, LLC

“*” marked represents similar segmentation in other categories in the respective section.