United Kingdom AADC-Deficiency Therapeutics Market 2023 | Estimate CAGR Analysis, Technological Development Overview, Growth Trends and Competitive Research till 2028

Report Overview

The UK AADC-Deficiency Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

The AADC deficiency is an ultra-rare autosomal recessive disease caused by a non-functional AADC enzyme, which normally synthesizes serotonin and dopamine, among other important compounds. As a result, patients with AADC deficiency lack crucial neurotransmitters, including serotonin, dopamine, norepinephrine, epinephrine, and melatonin, and have severe developmental and motor deficiencies. Symptom onset typically occurs during the first months of life.

The majority of patients present a severe phenotype with early-onset hypotonia, oculogyric crises, ptosis, dystonia, hypokinesia, impaired development, and autonomic dysfunction, a few patients with a milder disease course are known. Patients with AADC deficiency also present with autonomic dysfunction that is characterized by impairment of the sympathetic regulation of heart rate and blood pressure, paroxysmal sweating, emotional instability, and sleep disturbance. This leads to a complex syndrome characterized by motor, behavioral and autonomic symptoms.

The life expectancy of patients with severe AADC deficiency has been reported to be less than a decade. In patients identified with AADC deficiency where gender is reported, the distribution is estimated as; approximately 57% are male and 43% are female. There are around 120 confirmed diagnoses of AADC deficiency worldwide documented in the literature. As per the cases documented in the literature, the average age of symptom onset for AADC deficiency is 2.7 months. Despite the early onset of symptoms, the average age of diagnosis is 3.5 years. The predicted birth rate of individuals with AADC deficiency is estimated to be approximately 1:118,000 in the EU, and these birth rates translate into a current estimate of about 853 living patients with AADC deficiency in the EU.

Market Growth Drivers

The introduction of novel therapies, robust product pipeline, rising government initiatives for creating awareness regarding the AADC-deficiency diagnosis and its treatment, and its available therapeutic options are expected to significantly fuel the market growth over the forecast period.

Additionally, changes in government policies, demand and supply of the market, government intervention programs, and major market share are also playing an essential role to boost the market growth over the forecast period. Moreover, the rise in the number of patients suffering from AADC deficiency is likely to fuel the market growth during the forecast period.

In addition, rise in healthcare expenses and government initiatives for the betterment of health. Increased R&D activities to counter AADC deficiency in children are more likely to propel the market demand during the forecast period.

Market Restraints

Increasing collaboration and technological advancements are projected to bring new growth avenues for market players during the forecast period. While the high cost of approved therapeutics and lower treatment rates hampers the market.

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Top Major Players

The AADC-deficiency treatment market is a niche segment and currently has only a few players in the UK, and these are planning to adopt various strategies to garner maximum market share. The inorganic and organic strategies will pave a way for the expansion of the business and customer base of market players. PTC Therapeutics Limited is one of the major players operating in the UK market. Its Eladocagene exuparvovec (PTC-AADC, Upstaza) is indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency and expected to account for maximum market share over the forecast periods.

The United Kingdom AADC-Deficiency Therapeutics Market 2023 Report makes available the current and forthcoming technical and financial details of the industry. It is one of the most comprehensive and important additions to the Prudent Markets archive of market research studies. It offers detailed research and analysis of key aspects of the global United Kingdom AADC-Deficiency Therapeutics market. This report explores all the key factors affecting the growth of the global United Kingdom AADC-Deficiency Therapeutics market, including demand-supply scenario, pricing structure, profit margins, production, and value chain analysis.

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Market Segmentation

By Diagnosis:

Based on the diagnosis, the AADC-deficiency treatment market can be bifurcated into

physical examination
complete blood count test
measurement of electrolyte levels
magnetic resonance imaging (MRI)
measurement of AADC-enzyme levels
spinal tap or lumbar puncture
genetic testing
Others

A complete clinical evaluation and a high index of suspicion are required to make the diagnosis. The evaluation of a child starts with a complete physical examination. Although many tests, such as a complete blood count, measurement of electrolyte levels, and magnetic resonance imaging of the brain are usually performed in the diagnostic workup of a child presenting with neurodevelopmental delay. Additionally, some other diagnostic tests typically involve a blood test taken to measure levels of the AADC-enzyme and a spinal tap or lumbar puncture to collect cerebrospinal fluid (CSF). Levels of dopamine and serotonin in the CSF can help to diagnose AADC deficiency. Finally, genetic testing can also be performed to detect mutations in the DDC gene and confirm the diagnosis.

By Therapeutics:

In terms of therapeutics, the AADC-deficiency treatment market is classified into

gene replacement therapy
physiotherapy
speech therapy
dietary supplements therapy
psychotherapy
dopamine agonists
monoamine oxidase (MAO) inhibitors
pyridoxal phosphate
pyridoxine
anticholinergic agents
folinic acid
L-dopa with carbidopa
L-dopa without carbidopa
5-hydroxytryptophan
Benzodiazepines
Melatonin
selective serotonin reuptake inhibitors (SSRIs).

Current research and clinical trials in AADC deficiency are mostly focused on gene replacement therapy, which aims to replace the non-working gene. The gene replacement therapy is injected via a surgical procedure into an area of the brain called the putamen. Thus, gene replacement therapy is expected to witness maximum market share during the forecast period.

By Route of Administration:

Based on route of administration, the AADC-deficiency treatment of the market can be segmented into oral, parenteral, and others. The parenteral route is anticipated to dominate the route of the administration segment as Eladocagene exuparvovec is delivered as four 0.080 mL infusions. Till now, it is only the effective gene replacement therapy that has been approved in the UK, thus further projected to control its market share during the forecast period.

By Distribution Channels:

In terms of distribution channels, the AADC-deficiency treatment market has been bifurcated into

Hospital pharmacies
Retail pharmacies
Online pharmacies

The hospital pharmacies segment generated the highest revenue in 2020 and is anticipated to witness growth during the forecast period due to most major products are prescription products, which are available in hospital pharmacies.

Top Major Players

The AADC-deficiency treatment market is a niche segment and currently has only a few players in the UK, and these are planning to adopt various strategies to garner maximum market share. The inorganic and organic strategies will pave a way for the expansion of the business and customer base of market players. PTC Therapeutics Limited is one of the major players operating in the UK market. Its Eladocagene exuparvovec (PTC-AADC, Upstaza) is indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency and expected to account for maximum market share over the forecast periods.

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The report covers the competitive analysis of the market. As the demand is driven by a buyer's paying capacity and the rate of item development, the report shows the important regions that will direct growth. This section exclusively shares insight into the budget reports of big-league members of the market helping key players and new entrants understand the potential of investments in the Global United Kingdom AADC-Deficiency Therapeutics Market. It can be better employed by both traditional and new players in the industry for complete know-how of the market.

Strategic Points Covered in Table of Content of Global United Kingdom AADC-Deficiency Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom AADC-Deficiency Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom AADC-Deficiency Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom AADC-Deficiency Therapeutics
Chapter 4: Presenting the United Kingdom AADC-Deficiency Therapeutics Market Factor Analysis, Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.
Chapter 5: Displaying the by Type, End User and Region/Country
Chapter 6: Evaluating the leading manufacturers of the United Kingdom AADC-Deficiency Therapeutics market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile
Chapter 7: To evaluate the market by segments, by countries and by Manufacturers/Company with revenue share and sales by key countries in these various regions
Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

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