Urea Cycle Disorders Treatment Market Analysis By Treatment Type(Amino Acid Supplements, Sodium Phenylbutyrate, Glycerol Phenylbutyrate, Sodium Benzoate), By Enzyme Deficiency Type(Ornithine Transcarbamylas (OTC), Argininosuccinate Synthetase (citrullinemia) (AS), Arginase (AG), Argininosuccinate Lyase (AL), Carbamoyl Phosphate Synthase (CPS1), N-acetylglutamate Synthase (NAGS)), By Route of Administration(Oral, Injectable) & Forecast 2024-2035

  • 02/03/2023
  • 223
  • Healthcare & Life Sciences
The global urea cycle disorders treatment market size was valued at USD 1,250 million in 2021 and is projected to reach around USD 1,650 million in 2030 exhibiting a CAGR of 3.5% in the forecasted period. The increasing prevalence of urea cycle disorders and the growing number of pipeline products. Product approvals, launches, and strategic initiatives by important market players are anticipated to contribute to market growth. Furthermore, research and development studies underlining the genetic description of urea cycle disorders will benefit market players align their product development supporting to market growth. On the other hand, the high cost of therapeutics can be a restraint in the growth of this market.

The increasing prevalence of urea cycle disorders is anticipated to upsurge the demand for treatment preferences for urea cycle disorders (UCD), which is estimated to drive the growth of global urea cycle disorders treatment market throughout the forecasted period. As per the Urea Cycle Disorders Consortium (UCDC), a section of the Rare Diseases Clinical Research Network (RDCRN), united prevalence of urea cycle disorders is nearby 1 in 30,000 people in the U.S in 2017.

Recently, nations have faced an enormous threat of COVID-19. As per the World Health Organization (WHO), coronavirus is an infectious disease, and most people diseased with the COVID-19 virus will experience mild to moderate respiratory illness. The occurrence of the COVID-19 pandemic damaged the world economy and the healthcare system. The item “Longitudinal Metabolomics Reveals Ornithine Cycle Dysregulation Correlates with Inflammation and Coagulation in COVID-19 Severe Patients,” issued in December 2021, recognized that the ornithine cycle dysregulation is significantly interrelated with swelling and coagulation in severe patients, which may be a possible machinery of COVID-19 pathogenicity. Consequently, the COVID-19 medicines hindered the urea cycle, aiming to a lack of certain enzymes. Therefore, there is a growing demand for its therapeutics, contributing to market growth.

Though, the production of these drugs was hindered owing to the momentary lockdown in the manufacturing plants, dearth of skilled labour, and increasing cases of COVID-19 inside the manufacturing plants, which has led to outages, thus, hampering the market. Hence, the above factors indicate that COVID-19 considerably impacted the market.
Enzyme Deficiency Type Insights
Ornithine transcarbamylase segment is projected to account for the major market share through the forecasted period. Ornithine transcarbamylase deficiency is an extremely prevalent urea cycle disorder. It is an X-linked genetic disorder that inhibits the breakdown and excretion of ammonia. It allows ammonia to accumulate, escalating to toxic levels where it affects the central nervous system.
The factors such as product approvals, intensifying research and development, and calculated initiatives accepted by major market players are anticipated to back to the substantial growth of the market. For instance, in July 2021, Arcturus Therapeutics Holdings Inc. got approval from the UK Health Research Authority to start a Phase II clinical study for ARCT-810, a new mRNA-based therapeutic candidate for Ornithine Transcarbamylase (OTC) Deficiency. Moreover, in April 2020, the company recognized two clinical trials for its flagship asset ARCT-810. The Company’s Investigational New Drug (IND) application for a Phase Ib study in patients with OTC deficiency was certified to proceed by the United States Food and Drug Administration (FDA), and a further Clinical Trial Application (CTA) for a Phase I study in strong volunteers was accepted by the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). Consequently, with rising product approvals and research and development activities, the segment is anticipated to present effective therapeutics to boost the market’s growth.

Region Insights
Among regions, North America is anticipated to hold leading position in the global urea cycle disorders treatment market during the forecast period, owing to key companies focusing on product launches for the treatment of Urea cycle disorders. For instance, in January 2021, Recordati Rare Diseases Inc. received the U.S. Food and Drug Administration (FDA) sanction for CARBAGLU for handling the acute hyperammonemia.

The primary boosting factors for the growth of the North American urea cycle disorders treatment market are the growing prevalence of urea cycle disorders in the region and the snowballing number of pipeline products. Additionally, increasing product launches and other strategic initiatives such as mergers and acquisitions and partnerships are also anticipated to drive the market’s growth.

The United States within North America is estimated to hold a significant share of the market throughout the forecasted period. For example, as per the study published in January 2021, titled “Management of Late Onset Urea Cycle Disorders—A Remaining Challenge for the Intensivist?” The yearly incidence in the United States is 1: 35,000 births which exemplifies 113 new cases each year. Therefore, there is a great burden of UCDs on the United States healthcare system, growing the demand for its diagnostics and therapeutics, hence, boosting the market.

Furthermore, product development and related technological innovation will provide more advantages and overcome the present challenges, intending to profitable market growth. For instance, in October 2021, Acer Therapeutics Inc. and RELIEF THERAPEUTICS Holding SA, broadcasted that the U.S. Food and Drug Administration (FDA) had approved for filing of the New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for the cure of patients with Urea Cycle Disorders (UCDs).

Moreover, increasing research and development activities in the market aids to evaluate the efficiencies of prevailing treatment and increase the pipeline of drugs, thus fuelling the market. For instance, a study was initiated in March 2021 to conduct post-marketing surveillance of carglumic acid (Carbaglu) to achieve long-term clinical safety evidence. Carglumic acid was permitted by the United States Food and Drug Administration (FDA) for dealing of acute hyperammonemia owing to N-acetyl glutamate synthase (NAGS) scarcity. Thus, positive results will boost the acceptance of therapeutics, thus driving the market’s growth.
Therefore, due to the abovementioned factors, the studied market is expected to lead to lucrative growth of the market in North America.

Key Companies Insights
The market for urea cycle disorders treatment is moderately competitive. With the rising applications of urea cycle disorders treatment, new players are planning to enter the market. The companies are also involved in events like joint ventures, partnerships, acquisitions, mergers, and collaborations. These activities aid in growing the effect of the players in the Urea cycle disorders treatment market, ultimately boosting the market growth.

Some of the key companies working in the global 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
• Other players

Some of the Recent Developments:
• In February 2022, Relief Therapeutics Holding SA and Acer Therapeutics, were issued a new patent from the United States Patent and Trademark Office (USPTO) for certain claims related to ACER-001 (sodium phenylbutyrate) related to ACER-001's multi-particulate dosage formulation for oral management as a possible treatment for Urea Cycle Disorders (UCDs) and Maple Syrup Urine Disease (MSUD).

• In January 2022, Acer Therapeutics Inc. and Relief Therapeutics Holding SA declared the approval of four ACER-001 abstracts for poster presentations in Orlando, Florida, and in Las Vegas, Nevada.

By Treatment Type
• Amino Acid Supplements
• Sodium Phenylbutyrate
• Glycerol Phenylbutyrate
• Sodium Benzoate
• Others
By Enzyme Deficiency Type
• Ornithine Transcarbamylas (OTC)
• Argininosuccinate Synthetase (citrullinemia) (AS)
• Arginase (AG)
• Argininosuccinate Lyase (AL)
• Carbamoyl Phosphate Synthase (CPS1)
• N-acetylglutamate Synthase (NAGS)

By Route of Administration
• Oral
• Injectable

By Geography
• North America
o U.S.
o Canada
o Mexico
• Europe
o U.K.
o Germany
o France
o Italy
o Spain
o Russia
• Asia-Pacific
o Japan
o China
o India
o Australia
o South Korea
• Latin America
o Brazil
o Argentina
o Colombia
o South Africa
o Saudi Arabia
o Egypt
Base Year: 2023
Historic Year: 2016-2022
Forecast: 2024-2035

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