Urea Cycle Disorders Market Growth Anticipated by 2032 | Major Players: Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, expected to boost the market

In the market landscape of Urea Cycle Disorders, an impressive surge is expected during the study period spanning 2019 to 2032, according to the latest report titled  “Urea Cycle Disorders Market Insights, Epidemiology and Market Forecast 2032” from DelveInsight. 

The Urea Cycle Disorders market report sheds light on Urea Cycle Disorders current treatment practices, upcoming drugs in the Urea Cycle Disorders pipeline, market shares of individual therapies, and the anticipated trajectory of the Urea Cycle Disorders market size from 2019 to 2032 across the 7MM (the United States, the EU-4 comprising Italy, Spain, France, and Germany, the United Kingdom, and Japan).

Driving Forces Behind the Urea Cycle Disorders Market Growth

The market size shall grow during the forecast period, i.e., 2023–2032 owing to the increasing prevalence of Urea Cycle Disorders. The market size is expected to increase during the study period.

Discover the Anticipated Evolution and Growth of the Market @ Urea Cycle Disorders Therapeutics Market Forecast

Therapeutic Advancements and Emerging Treatments:

• Urea Cycle Disorders Clinical Trial Progression: The market is set to experience significant growth, driven by the progression of emerging therapies expected for launch between 2023 and 2032. Pioneering companies, including Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others, are actively engaged in developing novel drugs for potential market entry.

• Urea Cycle Disorders Innovative Therapies: Ongoing research and development activities are fostering the introduction of innovative therapies designed to address the signs and symptoms of Urea Cycle Disorders. Therapies such as LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and others are driving the Urea Cycle Disorders market.

Urea Cycle Disorders Treatment Market

The treatment of disorders related to the urea cycle is a lifelong process aimed at managing symptoms and doesn't cure the disorder. Strategies include monitoring ammonia levels using blood tests (serum and plasma levels) at regular intervals and using the results to optimize treatment methods. In the current market scenario, recommended therapies to possibly slow the progression of the disease include sodium benzoate, sodium phenylacetate, Glycerol phenylbutyrate, sodium phenylbutyrate, and antiemetic agents. 

These drugs lower blood ammonia concentrations by conjugation reactions involving acylation of amino acids. For neonates with hyperammonemia, the immediate treatment goal is a rapid lowering of ammonia and reducing dietary protein intake. Hemodialysis is very effective at reducing plasma ammonia and should immediately be initiated if elevated hyperammonemia is observed. Ammonia scavenger medications such as AMMONUL IV are also useful. AMMONUL IV acts by removing glycine and glutamate from plasma thereby their reducing contribution to ammonia formation.

Urea Cycle Disorders Treatment Market

The dynamics of the Urea Cycle Disorders market are expected to change owing to a robust pipeline products. Some of the key players involved in the development of novel drugs are Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others.

LOARGYS (pegzilarginase) is an engineered human arginase I enzyme designed to degrade the amino acid arginine. Pegzilarginase is being developed to treat two extremes of arginine metabolism, including arginine excess in patients with Arginase 1 Deficiency, as well as some cancers which have been shown to have a metabolic dependency on arginine. The company has completed a Phase III clinical study which demonstrated reduction in blood arginine levels in patients with ARG1-D. The therapy was well tolerated among the patients. Recently, in October 2023, Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending marketing authorization of LOARGYS (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D) in patients two years and older. 

DTX301 is an investigational gene therapy being developed for the treatment of individuals with ornithine transcarbamylase (OTC) deficiency which is the most common urea cycle disorder. DTX301 is designed by Ultragenyx Pharmaceutical to deliver OTC gene expression in a durable fashion, with the goal of preventing or reducing the occurrence of complications associated with OTC deficiency. It has been granted Orphan Drug Designation in the United States, EU and United Kingdom and Fast Track Designation in the United States. Long term Phase I/II data demonstrated an acceptable safety profile and durable metabolic control and sustained responses. The company is conducting a Phase III (NCT05345171) trial to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

LUNAR-OTC (ARCT-810) is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP). Arcturus Therapeutics is developing mRNA medicines that enable OTC patients to make healthy functional OTC enzyme in the liver cells. The early phase studies demonstrated efficacy of the therapy in restoration of the urea cycle function to normal levels and increased survival. Currently, ARCT-810 is being investigated in Phase II (NCT05526066) clinical development to evaluate the safety, tolerability and pharmacokinetics of ARCT-810 in adolescent and adult participants with ornithine transcarbamylase deficiency.

Leading Urea Cycle Disorders Companies and Emerging Drugs: Pioneering companies such as Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, among others, are actively developing novel drugs for potential entry into the Urea Cycle Disorders market.

Urea Cycle Disorders Therapeutic Landscape: Key therapies identified for Urea Cycle Disorders treatment include LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and more.

Urea Cycle Disorders Overview:

Urea cycle disorders (UCDs) are a group of rare genetic disorders that affect the body's ability to eliminate ammonia, a waste product formed during the breakdown of proteins. Normally, ammonia is converted into urea in the liver through a series of biochemical reactions known as the urea cycle. Urea is then excreted from the body through urine.

In individuals with urea cycle disorders, one of the enzymes involved in the urea cycle is deficient or dysfunctional, leading to the accumulation of ammonia in the blood. This elevated ammonia, known as hyperammonemia, can be toxic to the brain and nervous system, causing neurological damage and potentially life-threatening complications.

There are several types of urea cycle disorders, each caused by mutations in specific genes that encode enzymes critical to the urea cycle. These disorders include:

• Ornithine Transcarbamylase (OTC) Deficiency: This is the most common urea cycle disorder, caused by a deficiency in the OTC enzyme. It is an X-linked disorder, primarily affecting males. Symptoms can range from mild to severe and may appear shortly after birth or later in life.

• Carbamoyl Phosphate Synthetase (CPS) Deficiency: CPS deficiency is a rare autosomal recessive disorder caused by mutations in the CPS1 gene, leading to reduced or absent activity of the CPS enzyme. Symptoms can be severe and appear soon after birth.

• Argininosuccinic Acid Synthetase (AS) Deficiency: AS deficiency results from mutations in the ASS1 gene, leading to decreased production of the AS enzyme. Symptoms can range from mild to severe and may present at different ages, from infancy to adulthood.

• Arginase Deficiency: This disorder is caused by mutations in the ARG1 gene, resulting in a deficiency of the arginase enzyme. Symptoms often appear in early childhood and can vary in severity.

Symptoms of urea cycle disorders can include vomiting, lethargy, irritability, seizures, developmental delay, coma, and even death if not promptly treated. Diagnosis involves blood tests to measure ammonia levels and specific metabolites related to the urea cycle, as well as genetic testing to identify the specific enzyme deficiency.

Treatment aims to reduce ammonia levels and prevent its accumulation. This often involves dietary restrictions, medications like ammonia-scavenging drugs, and in severe cases, dialysis to remove excess ammonia from the blood. Long-term management by metabolic specialists and genetic counselors is essential for individuals with urea cycle disorders. Early detection and intervention are critical to minimize the risk of neurological damage and improve outcomes for affected individuals.

Key Facts Urea Cycle Disorders Market Report:

• Key players such as Aeglea BioTherapeutics, Immedica Pharma, Ultragenyx Pharmaceutical, Arcturus Therapeutics, and others are investigating its candidates for Urea Cycle Disorders.

• Urea Cycle Disorders pipeline includes the major therapies such as LOARGYS (pegzilarginase), DTX301, LUNAR-OTC (ARCT-810), and others. 

• In December 2022, the US FDA approved OLPRUVA (sodium phenylbutyrate) to treat certain patients living with urea cycle disorders (UCDs). Acer Therapeutics presented data from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) at the 44th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) on March 18th to 21st.

Urea Cycle Disorders Epidemiology Segmentation:

According to NORD, OTC deficiency affects males more often than females and most males are symptomatic in nature. In males, symptoms typically begin during the first few days of life.

• According to reports published by the Centers for Disease Control and Prevention (2022), the anticipated incidence of UCD is 1 in 8,500 births. The research findings suggest that many of the cases of UCD persist undiagnosed. In some of the cases, the newborn with UCD dies without a conclusive diagnosis.

• According to the American Association for Clinical Chemistry (2019), the incidence of Urea Cycle Disorders, or UCDs, in the US is estimated to be around 1 in 8,200 births. The mortality rate is 24% in neonatal cases, and 11% in later onset cases.

For in-depth insights, access the full report @ Urea Cycle Disorders Market Outlook 2032