Argininosuccinic aciduria (ASA) - Market Insights, Epidemiology, and Market Forecast - 2030
発行: DelveInsight Business Research LLP
ページ情報: 英文 119 Pages
DelveInsight's "Argininosuccinic Aciduria (ASA) - Market Insights, Epidemiology, and Market Forecast -2030" report delivers an in-depth understanding of the historical and forecasted epidemiology and the market trends of argininosuccinic aciduria (ASA) in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
The argininosuccinic aciduria (ASA) market report provides current treatment practices, emerging drugs, and their market share of the individual therapies, current and forecasted argininosuccinic aciduria (ASA) market Size from 2017 to 2030, segmented by seven major markets. The report also covers the current treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.
Study Period: 2017-2030
Argininosuccinic Aciduria (ASA) Overview
Argininosuccinic aciduria (ASA) is one of the inherited disorders belonging to 'urea cycle disorders,' which causes ammonia to accumulate in the blood. Patients with ASA lack 'argininosuccinate lyase,' one of the liver enzymes needed to get rid of excess nitrogen. In the absence of this enzyme, excess nitrogen accumulates in the body in the form of ammonia, which can be toxic at high levels, especially to the brain.
The disease's symptoms usually appear in the first few days of life and include lethargy (lack of energy), vomiting, loss of appetite, seizures (fits), and coma. It is a long-term debilitating and life-threatening disease that leads to mental retardation and is associated with poor overall survival. ASA can manifest as either a severe neonatal-onset form with hyperammonemia within the first few days after birth or as a Late-onset form with episodic hyperammonemia and/or long-term complications that include liver dysfunction, neurocognitive deficits, and hypertension.
Treatment of acute metabolic decompensations with hyperammonemia involves discontinuing oral protein intake, supplementing oral intake with intravenous lipids and/or glucose, and the use of intravenous arginine and nitrogen-scavenging therapy. Dietary restriction of protein and dietary supplementation with arginine are the mainstays in long-term management. Orthotopic liver transplantation (OLT) is best considered only in patients with recurrent hyperammonemia or metabolic decompensations resistant to conventional medical therapy.
Ammonia scavengers may be required in those who have metabolic decompensations. Recent evidence also suggests that ASL is required to synthesize nitric oxide (NO) and that NO deficiency in this disorder may lead to hypertension. Various nitrogen scavengers have been approved to treat urea cycle disorders, including sodium benzoate, Buphenyl (Ammonaps in Europe), and Ravicti. Liver transplant should be an option for ASA patients to prevent further neurologic deterioration and improve their quality of life.
Moreover, while acute hyperammonemia in ASA should continue to be treated with high dose intravenous arginine, in the context of chronic management, lower dose arginine in conjunction with nitrogen-scavenging therapy, especially in patients with elevated liver enzyme tests, should be considered.
Argininosuccinic Aciduria (ASA) Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnosis available in the argininosuccinic aciduria (ASA) market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The DelveInsight Argininosuccinic Aciduria (ASA) market report gives a thorough understanding of Argininosuccinic aciduria (ASA) by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides treatment algorithms and treatment guidelines for Argininosuccinic Aciduria (ASA) in the US, Europe, and Japan.
The persistent argininosuccinic aciduria (ASA) epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical and forecasted argininosuccinic aciduria (ASA) epidemiology segmented as the total incident cases of argininosuccinic aciduria (ASA) and early-onset and late-onset cases of argininosuccinic aciduria. The report includes the Incident scenario of argininosuccinic aciduria (ASA) in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.
The epidemiology segment also provides the argininosuccinic aciduria (ASA) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total Incident population of argininosuccinic aciduria (ASA) in 7MM countries was estimated to be 393 in 2017.
The drug chapter segment of the argininosuccinic aciduria (ASA) report encloses the detailed analysis of argininosuccinic aciduria (ASA) marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the argininosuccinic aciduria (ASA) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
Treatment of ASA may require the coordinated efforts of a team of specialists ranging from pediatricians, geneticists, dieticians, and physicians who are familiar with metabolic disorders to occupational, speech-language, and physical therapists. A comprehensive approach to treatment is needed to treat children with wide disabilities. Genetic counseling is also recommended for affected individuals and their families. Furthermore, treatment recommendations may be separated into two scenarios: treatment for rapid control of hyperammonemia during metabolic decompensations and interval therapy to prevent the primary manifestations and long-term complications.
Products detail in the report…
Argininosuccinic Aciduria (ASA) Emerging Drugs
ACER-001 is a proprietary, fully taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPB) developed using a microencapsulation process. NaPB helps control blood ammonia levels in conjunction with a restricted diet for people with UCDs. For the treatment of UCDs, NaPB is a prodrug that metabolizes quickly to become phenylacetate that binds to glutamine via acetylation to form phenylacetylglutamine. Phenylacetylglutamine is similar to urea; it contains two moles of nitrogen and is excreted by the kidneys. Through this process, NaPB is an alternate way to excrete excessive nitrogen created by the ineffective processing of ammonia during the urea cycle.
Products detail in the report…
The argininosuccinic aciduria (ASA) market outlook of the report builds the detailed comprehension of the historical, current, and forecasted argininosuccinic aciduria (ASA) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand of better technology.
This segment gives a thorough detail of Argininosuccinic Aciduria (ASA) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, Argininosuccinic Aciduria (ASA) market in 7MM is expected to change in the study period 2017-2030.
The management and diagnosis of ASA have not advanced significantly in the past decade, but given recent advances in understanding the timing and potential pathogenesis of ASA, it is hopeful that the next decade will bring early diagnostics and novel therapeutics.
This section includes a glimpse of the argininosuccinic aciduria (ASA) market in the 7MM. The market size of argininosuccinic aciduria (ASA) in the seven major markets was found to be USD 40.3 million in 2017.
This section provides the total Argininosuccinic Aciduria (ASA) market size and market size by therapies in the United States.
The United States accounts for the highest market size of Argininosuccinic Aciduria (ASA) in comparison to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
The treatment of ASA aims to prevent the formation of excessive ammonia or the removal of excess ammonia during a hyperammonemic episode. Treatment recommendations may be separated into two scenarios, i.e., treatment for rapid control of hyperammonemia during metabolic decompensations and interval therapy, to prevent the primary manifestations and long-term complications.
Dietary restrictions in individuals with ASA are aimed at limiting the amount of protein intake to avoid the development of excess ammonia. However, enough protein must be taken in by an affected infant to ensure proper growth.
In addition to dietary restrictions and supplements, individuals with ASA are treated by medications that stimulate the removal of nitrogen from the body. These medications provide an alternative method to the urea cycle in converting and removing nitrogen waste; this is known as alternative pathway therapy or nitrogen scavenging therapy and includes sodium benzoate, sodium phenylbutyrate glycerol tri-phenylbutyrate, and others.
Oral nitrogen-scavenging therapy, patients who have had metabolic decompensations or episodes of elevated ammonia despite being on a protein-restricted diet and arginine base supplementation are candidates for oral nitrogen-scavenging therapy, in which sodium benzoate and sodium phenylbutyrate stimulate the excretion of nitrogen in the form of hippuric acid and phenylacetylglutamine, respectively.
There is no cure for urea cycle disorders other than a liver transplant. However, a liver transplant is limited by donor availability and patient eligibility. It is also associated with significant risks and complications, including perioperative morbidity and mortality, liver rejection, and vulnerability to infection due to lifelong immunosuppressant medication.
For the prevention of secondary complications, recommendations for salt restriction, and the use of antihypertensive therapy in those with elevated blood pressures are not different from those for the general population. Potassium supplementation must be considered in those with hypokalemia.
The total argininosuccinic aciduria (ASA) market size and market size by therapies in EU-5 are also mentioned.
The total argininosuccinic aciduria (ASA) market size and market size by therapies in Japan are also mentioned.
This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017-2030. The analysis covers argininosuccinic aciduria (ASA) market uptake by drugs, patient uptake by therapies, and sales of each drug.
This 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 based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.
Argininosuccinic Aciduria (ASA) Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase II and Phase III stages. It also analyses argininosuccinic aciduria (ASA) key players involved in developing targeted therapeutics.
Major players include Acer Therapeutics, Horizon Therapeutics, and Evox Therapeutics, whose key products are expected to get launched in the US market by 20XX.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for argininosuccinic aciduria (ASA) emerging therapies.
To keep up with current market trends, we take KOLs and SME's opinion working in the argininosuccinic aciduria (ASA) domain through primary research to fill the data gaps and validate our secondary research. Their opinions help to understand and validate current and emerging therapies treatment patterns or argininosuccinic aciduria (ASA) market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.
Competitive Intelligence Analysis
We perform Competitive & Market Intelligence analysis of the Argininosuccinic Aciduria (ASA) Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies: