United Kingdom Batten's Disease (CLN-2) Therapeutics Market Evolution Segmentation and Insight of Trends 2023 to 2028 with Top Countries Data

Report Overview

The UK Batten's Disease (CLN-2) 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 Batten disease (Jansky-Bielschowsky disease) is the common name for a family of severe and progressive neurological disorders (also known as neuronal ceroid lipofuscinoses - NCLs) that primarily affect children. Additionally, Batten disease is the most common inherited pediatric neurodegenerative disorder and the most common form of childhood dementia. Batten disease is a genetic disease, caused by mutations in ceroid lipofuscinosis neuronal (CLN) genes. The different types of Batten disease are classified according to the defective CLN gene that is causing the disorder (e.g., CLN1, CLN2, CLN3).

The Batten disease is also commonly described by reference to the childs age when symptoms typically appear in that form of Batten disease (e.g., CLN2 disease is known as late infantile Batten disease and CLN3 disease is known as juvenile Batten disease). The onset of seizures is typically followed rapidly by progressive motor and language decline between the ages of 4 to 6, loss of ability to swallow and talk, myoclonic jerks, epilepsy, blindness by age 7 to 10 years, and childhood dementia. Children also often have sleep disturbance and behavioral symptoms. Untreated, most affected children die between the ages of 6-12 years.

According to the Batten Disease Family Association UK, it is estimated that approximately 1 3 children are diagnosed with an infantile form of the disease each year, meaning there are probably between 15 and 30 affected children in the UK. Approximately 7 10 children are diagnosed with a late-infantile (including variants) form of the disease each year, meaning there are probably between 30 and 60 affected children and young people in the UK. And, approximately 3 4 children are diagnosed with a juvenile form of the disease each year, meaning there are probably between 30 40 affected children and young people in the UK. Therefore, in summary, we can estimate that there are approximately 100 150 children, young people, and adults currently living with an NCL diagnosis in the UK.

Market Growth Drivers

Increased incidence and prevalence of batten disease and clinical trials are expected to spur the growth of the batten disease treatment market. Various companies are conducting R&D for the development of new and effective drugs for the treatment of Batten disease are expected to support the growth of the batten disease treatment market.

The batten disease market is further anticipated to rise as the biotechnology and pharmaceutical firms engage more in R&D activities, the demand for new treatment methods grows, andhealthcare expenditure rises.

Market Restraints

However, the treatment is expensive, the lack of awareness among manufacturers and the patient population as well as unavailability of treatment for this disease due to scarcity of healthcare professionals are greatly hampering the growth of the batten disease treatment market.

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Top Major Players

The Batten's disease treatment market has limited players and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major players operating in the market include BioMarin Pharmaceuticals, Amicus Therapeutics UK Ltd, Pfizer UK, and Abeona Therapeutics Inc. among others.

The United Kingdom Batten's Disease (CLN-2) 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 Batten's Disease (CLN-2) Therapeutics market. This report explores all the key factors affecting the growth of the global United Kingdom Batten's Disease (CLN-2) Therapeutics market, including demand-supply scenario, pricing structure, profit margins, production, and value chain analysis.

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Market Segmentation

By Drugs Type:

Several drugs are available to treat or reduce the symptoms of Batten disease, and ultimately provide relief to the affected individuals which can be bifurcated as

Brineura
Anticonvulsants
Immunosuppressants
Others

The potential drugs include Brineura® is containing cerliponase alfa as the active ingredient. Brineura (cerliponase alfa) is the recombinant human tripeptidyl-peptidase, an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device; a year's treatment costs more than £500,000 per patient in the UK. The NICE's committee agreed that Brineura is an innovative treatment and effective in the neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency, however it is not a cost-effective treatment option.

Additionally, the Anticonvulsants and Immunosuppressants remain the mainstays for the treatment of this disease, to keep the patient clinically comfortable, to prevent seizures and other associated symptoms with the use of these medical interventions.

By Therapy Type:

In terms of therapy types, the Batten's Disease treatment market can be classified into

Stem-cell therapy
Enzyme-replacement therapy
Gene therapy
Occupational therapy
Vitamins supplemental therapy
Others

The stem-cell therapy, enzyme replacement therapy, and gene therapy are expected to increase the demand for batten disease treatment during the forecast period. In addition, there is also fatal stem cell material which may be possible to treat the brain of affected children and further anticipated to increase the growth of the batten disease treatment market. Additionally, receiving vitamin supplements also helps affected children and resulted in a slowing of the Batten disease which is expected to increase the growth of the batten disease treatment market during the forecast period.

By Routes of administration:

In terms of routes of administration, the Batten's Disease treatment market can be segmented into

Oral
Intravenous
Intracerebroventricular
Intrathecal
Others

Intracerebroventricular route of administration has been anticipated to gain maximum market share during the forecast period as BioMarin's Brineura is administered by intracerebroventricular route only.

By Distribution Channel:

In terms of distribution channels, the Batten's disease treatment market has been segmented into

Hospital pharmacies
Retail pharmacies
Online pharmacies

The hospital pharmacies segment has accounted for the highest market share in the past years as the major therapeutics are prescription products. Therefore, hospital pharmacies are further anticipated to witness higher market growth during the forecast period.

Top Major Players

The Batten's disease treatment market has limited players and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major players operating in the market include BioMarin Pharmaceuticals, Amicus Therapeutics UK Ltd, Pfizer UK, and Abeona Therapeutics Inc. among others.

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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 Batten's Disease (CLN-2) 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 Batten's Disease (CLN-2) Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom Batten's Disease (CLN-2) Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom Batten's Disease (CLN-2) Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom Batten's Disease (CLN-2) Therapeutics
Chapter 4: Presenting the United Kingdom Batten's Disease (CLN-2) 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 Batten's Disease (CLN-2) 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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