UK Batten’s Disease (CLN-2) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2595

  • Format: PPT, PDF

UK Batten’s Disease (CLN-2) Therapeutics Market: Segmented by Drugs Type, Therapy type, Routes of Administration, and Distribution Channel – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

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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 child’s 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, and healthcare 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.

TABLE OF CONTENTS

1. UK Batten’s Disease (CLN-2) Therapeutics Market Overview………………..
A. Market Size
2. Market Growth Drivers & Restraints……………………………………………….
Market Growth Drivers
A. Increased Incidence and Prevalence of Batten Disease
B. Biotechnology & Pharmaceutical Firms more engaged in R&D Activities
C. Development of Novel Therapeutics
Market Restraints
A. Huge Treatment Costs
B. Scarcity of Healthcare Professionals
3. Major Types of Batten’s Disease…………………………………………………………….
A. Infantile NCL
B. Late Infantile NCL
C. Juvenile NCL
D. Adult NCL
4. UK Batten’s Disease (CLN-2) Therapeutics Market Segmentation………..
A. By Drugs Type
I. Brineura
II. Anticonvulsants
III. Immunosuppressants
IV. Others
B. By Therapy Type
I. Stem Cell Therapy
II. Enzyme Replacement Therapy
III. Gene Therapy
IV. Occupational Therapy
V. Vitamins Supplemental Therapy
VI. Others
C. By Routes of Administration
I. Oral
II. Intravenous
III. Intracerebroventricular
IV. Intrathecal
V. Others
D. By Distribution Channel
I. Hospital Pharmacy
II. Retail Pharmacy
III. Online Pharmacy
5. UK Batten’s Disease (CLN-2) Major Products Market Share………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles……………………………………………………………………
A. BioMarin Company overview, Product & Services, Strategies & Financials
B. Amicus Company overview, Product & Services, Strategies & Financials
C. Pfizer Company overview, Product & Services, Strategies & Financials
D. Abeona Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………
A. New Trends and Development of Batten’s disease (CLN-2) Therapeutics Market
B. Future Opportunities
10. Conclusion

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.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

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The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

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We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

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