Urea Cycle Disorder Treatment Market to grow by USD 1.57 billion from 2024-2033

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 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 $1.57 billion in 2028 at a compound annual growth rate (CAGR) of 3.9%. 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, personalized nutritional therapies:.

Market Overview -
Urea cycle disorder treatment refers to managing rare genetic diseases where the body lacks the enzymes necessary to break down ammonia, causing it to build up and potentially be poisonous. The objective of urea cycle disorder treatment is to reduce blood ammonia levels.

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Surging Prevalence Of Urea Cycle Disorders Fuels Expansion Of Treatment Market
The rise in the prevalence of urea cycle disorders is expected to propel the growth of the urea cycle disorders treatment market going forward. Urea cycle disorder refers to metabolic disorders that are inborn and caused by flaws in one of the enzymes or transporter molecules necessary for the liver to remove ammonia from the blood. The rise in the prevalence of urea cycle disorders is driven by genetic disorders. When ammonia accumulation is identified as a urea cycle disorder (UCD), a customized treatment strategy incorporating dietary control, medication, and monitoring can be implemented to manage and lessen its negative consequences. For instance, in April 2023, according to StatPearls Publishing LLC, a US-based medical education library, the prevalence of urea cycle disorders caused by ornithine transcarbamylase deficiency was 1 in 140,000 people. Furthermore, in January 2021, according to a report shared by the National Library of Medicine, a US-based medical library run by the federal government, there were 113 new cases of urea cycle disorders for every 35,000 live births in the United States. Therefore, the rise in the prevalence of urea cycle disorders is driving the growth of the urea cycle disorders treatment market.

Competitive Landscape -
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

Innovative Product Launches Drive Advancements In Urea Cycle Disorder Treatment Market
Product innovation has emerged as a key trend gaining popularity in the urea cycle disorder treatment market. Major companies operating in the urea cycle disorder treatment market are concentrating on creating innovative products to strengthen their position in the market. For instance, in September 2022, Medunik USA, a US-based pharmaceutical company, launched Pheburane oral pellets, a unique and flavor-masking formulation of sodium phenylbutyrate (NaPB) for the long-term management of certain urea cycle disorders. It is a prescription drug used in conjunction with a particular diet to treat urea cycle disorders (UCDs) in adults and children that involve carbamyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinate synthetase (AS) deficiency. It is measured using a reusable, calibrated dosing spoon included in each package; unlike other forms of NaPB, it does not need to be mixed or taken orally. Pheburane can be consumed together with a snack or meal, or it can be sprinkled on a spoonful of apple sauce or carrot puree and taken with water, fruit juice, or protein-free infant formula.

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Key Segments -

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

Key highlights covered in the report -
1. Detailed market size forecast and historical data analysis
2. Key drivers influencing market growth
3. Identification of upcoming trends and potential opportunities in the market
4. Analysis of major players strategies, to understand competitive dynamics and market positioning
5. Evaluation of regional dynamics

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