DelveInsight’s Urea Cycle Disorders Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the Urea Cycle Disorders Market Size and Share in the 7MM (i.e. the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).
The Urea Cycle Disorders market report covers emerging drugs, current treatment practices, market share of the individual therapies, current and forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Urea Cycle Disorders: Overview
A Urea Cycle Disorder (UCD) is a genetic disorder that results in a deficiency of some major enzymes in the urea cycle. These enzymes are responsible for removing ammonia from the bloodstream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is changed to a compound called urea and removed from the blood. Normally, the urea is removed from the body through the urine. In urea cycle disorders, nitrogen builds up in the blood in the form of ammonia, a highly toxic substance, resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma, and/or death. The onset and severity of urea cycle disorders is highly variable. The severity correlates with the amount of urea cycle enzyme function.
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Some of the key facts of the Urea Cycle Disorders Market Report
According to the literature survey, Urea Cycle Disorders can occur irrespective of sex-based differences among children. However, there may be differences in the number based on the type of the UCD.
As per a study conducted by Susanne Nettesheim et al. (2017), titled, “Incidence, disease onset and short-term outcome in urea cycle disorders-cross-border surveillance in Germany, Austria and Switzerland”, the estimated incidence or Urea Cycle Disorders below 16 years of age at about 1 in 52,000 live births in Germany.
A study conducted by Elena Martín-Hernández et al. (2014), titled, “Urea cycle disorders in Spain: an observational, cross-sectional and multicentric study of 104 cases”, reported that Ornithine transcarbamylase (OTC) deficiency was the most frequent condition followed by type 1 citrullinemia and argininosuccinic aciduria.
Urea Cycle Disorders market size is anticipated to increase during the study period owing to the increasing incident population and rising awareness of Urea Cycle Disorders in the 7MM. The expected launch of emerging therapies and the research and development activities of pharmaceutical companies will also fuel the market growth during the forecast period.
The market outlook section of the report helps to build a detailed comprehension of the historic, current, and forecasted Urea Cycle Disorders market size by analyzing the impact of current and emerging therapies in the market. It also provides a detailed assessment of the market drivers & barriers, unmet needs, and emerging technologies.
The report gives a thorough detail of the Urea Cycle Disorders market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MoA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.
Urea Cycle Disorders Market Insights
There are a number of treatments that can be effective in urea cycle disorders, but still, there are many unmet needs. Alternative pathway therapy has limited effectiveness in preventing hyperammonemia and it needs to be combined with effective dietary management. Therefore, in children with a neonatal-onset disease or in those with very poor metabolic control, liver transplantation is also considered. So, there is an immediate need for R&D activities for the development of novel therapies that can overcome the limitations of existing therapies.
Urea Cycle Disorders Treatment Market
Recent advances in the treatment of inborn errors of urea synthesis have significantly decreased mortality. The treatment of urea cycle disorders consists of dietary management to limit ammonia production, in conjunction with medications and/or supplements that provide alternative pathways for the removal of ammonia from the bloodstream. The aim is to correct biochemical abnormalities and ensure adequate nutritional intake. Treatment involves compounds that increase the removal of nitrogen waste. These compounds convert nitrogen into products other than urea, which are then excreted; hence, the load on the urea cycle is reduced. The first compounds to be used were sodium benzoate and arginine. Later, phenylacetate was used, which has now been replaced by phenylbutyrate.
Urea Cycle Disorders Epidemiology
The epidemiology section covers insights about the historical and current Urea Cycle Disorders patient pool and forecasted trends for every seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.
Urea Cycle Disorders Drugs & Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Urea Cycle Disorders market or expected to get launched in the market during the study period. The analysis covers Urea Cycle Disorders market uptake by drugs; patient uptake by therapies; and sales of each drug.
Report’s Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
The report also covers the Urea Cycle Disorders Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II, and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses the recent Development such as collaborations, acquisitions, and mergers, licensing patent details, and other information for emerging therapies.
Urea Cycle Disorders Emerging Therapy Assessment
The dynamics of the Urea Cycle Disorders market are anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and the expected launch of emerging therapies during the forecast period of 2020–2030.
Urea Cycle Disorders Companies includes-
Kaleido Biosciences and others
Urea Cycle Disorders Therapies includes-
Buphenyl, and others
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Table of content
Competitive Intelligence Analysis
Market Overview at a Glance
Disease Background and Overview
Epidemiology and Patient Population
Treatment Algorithm, Current Treatment, and Medical Practices
Key Endpoints of Urea Cycle Disorders Treatment
Seven Major Market Analysis
7MM: Market Outlook
Access and Reimbursement Overview
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