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Batten Disease- Market Insights, Epidemiology and Market Forecast-2028

  • ID: 4790885
  • Drug Pipelines
  • March 2019
  • Region: Global
  • 179 pages
  • DelveInsight
1 of 3

FEATURED COMPANIES

  • Amicus Therapeutics
  • BioMarin Pharmaceuticals
  • MORE
Batten Disease- Market Insights, Epidemiology and Market Forecast-2028 report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Batten Disease in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan

The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Batten Disease from 2017 to 2028 segmented by the seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain and the United Kingdom)
  • Japan
Study Period: 2017-2028

Batten Disease – Disease Understanding and Treatment Algorithm

Batten disease, referred as Neuronal Ceroid Lipofuscinosis (NCL), is a rare and debilitating autosomal recessive neurodegenerative lysosomal storage disorder being caused by cellular accumulation of abnormal auto-fluorescent lipoproteins lipofuscin. This neurodegenerative disorder primarily affects the nervous system and is characterized by seizures and a progressive loss in motor, language, visual, and cognitive skills. There are 14 different forms of Batten disease, which are numerically abbreviated as CLN1, CLN2, CLN3, CLN4, CLN5, CLN6, CLN7, CLN8, CLN9 and CLN10, CLN11, CLN12, CLN13, CLN14

The Batten Disease market report gives the thorough understanding of the Batten Disease by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for Batten Disease in the US, Europe, and Japan

Batten Disease – Epidemiology

The Batten Disease epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 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 as well as forecasted epidemiology [segmented by Total Diagnosed Prevalent Population, and Type – Specific Diagnosed Prevalent Population] scenario of Batten Disease in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2017-2028

As per analysis, total diagnosed prevalent population of Batten Disease in the 7MM was assessed to be 1,606 in 2017, which is expected to increase at a CAGR of XXX% for the study period i.e. 2017-2028. The estimates show higher diagnosed prevalence of Batten Disease in the United States, among all 7MM countries. Furthermore, among the EU-5 countries, Germany had the highest diagnosed prevalent population of Batten Disease, followed by the United Kingdom and France. On the other hand, Spain had the lowest diagnosed prevalent population of less than 100 cases assessed in 2017. The estimates show that Japan had 64 prevalent cases diagnosed for Batten Disease in 2017
Batten disease has 14 known forms, with mutations in 13 genes. Progressions of the different forms of the disease vary (the phenotype) based on the type of genetic mutation (the genotype) and other factors. The trend in prevalent cases of the disease, based on the form of gene mutation, varies among the 7MM countries. However, CLN1, CLN2, CLN3, CLN6 and CLN7 are the most commonly occurring in all the 7MM countries. In contrast, for majority of the countries, CLN9 was observed to contribute least towards the patient population

Batten Disease – Drug Chapters

This segment of the Batten Disease report encloses the detailed analysis of marketed drugs and late stage (Phase III, II, and I/II) pipeline drugs. It also helps to understand the 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

Brineura (cerliponase alfa) is a recombinant form of human tripeptidyl peptidase 1 (TPP1) being marketed by BioMarin Pharmaceuticals for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). It is administered via intracerebroventricular (ICV) infusion and intended to be used in combination with a delivery device. BioMarin is utilizing its patented technology for the direct delivery of Brineura into the fluid surrounding the brain (cerebrospinal fluid) with an ultimate aim to reach out the cells of the brain and central nervous system. The U.S. Food and Drug Administration has approved Brineura to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) and became the first treatment approved to treat children with CLN2 disease, a form of Batten disease

Brineura’s approval was based on the results of Phase I/II open-label, a dose-escalation trial conducted in 24 patients with CLN2 disease, ages 3 to 8, and results from an ongoing extension study. These clinical trials demonstrated the safety and tolerability of Brineura administered in patients’ brains and assessed the therapy’s effectiveness as measured by a CLN2 disease-specific rating scale score, in comparison with natural history data, after 48 and 72 weeks of treatment. This scale measured the children’s ability to walk or crawl, ranging from loss of walking or crawling to regular walking or crawling, as well as motor-language scores

Batten Disease – Market Outlook

The Batten Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market 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 market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of 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

The market size of Batten Disease in the seven major markets is expected to grow at a CAGR of 29.76% for the study period (2017-2028). Among the 7MM countries, the United States had the highest market size of Batten disease in 2017, which accounts for approximately 80% of the total market. The growth of market size for Batten disease is attributed to currently approved treatment regimens as well as emerging therapies

Among the EU5 countries, Germany had the highest market size, while Spain had the lowest market size of Batten Disease. Unlike other 7MM countries, Japan does not have any approved therapy for any of the forms of Batten Disease, therefore, the therapeutic market size in the country is completely dependent on expected launch of upcoming therapies

Batten Disease – Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers 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 allows 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

No specific treatment is yet available to cure or slow the progression of Batten disease, but treatments do exist to manage the symptoms and make the child more comfortable. The current therapeutic landscape for Batten Disease predominantly focuses on the disease-associated symptoms. On the whole, Anti-convulsants 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 medical interventions

The most widely observed symptoms include epilepsy or seizures. In order to control or reduce seizures, anti-convulsant therapies are prescribed by a doctor, such as valproic acid and lamotrigine, alone or in combination with clonazepam. Other anti-convulsant therapies that may be prescribed include carbamazepine, oxcarbazepine, and phenytoin

Many new therapies are in the pipeline for the treatment of different forms of NCLs. Most of these may halt or slow the progression of the disease but there remains a dearth of information to prove whether these can completely reverse the disease. Companies across the globe have shifted their focus to the development of drugs to cure the various forms of the disease. To name a few of these: REGENXBIO, Polaryx Therapeutics, Spark Therapeutics, Abeona Therapeutics, Amicus Therapeutics, and others. While, the majority of these companies have recently shifted their focus towards this therapeutic area, and only Amicus Therapeutics has its drug in the early stage of clinical development

Batten Disease Report Insights
  • Patient Population
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Batten Disease Report Key Strengths
  • 10 Years Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Key Cross Competition
  • Market Size by Therapies
  • Drugs Uptake
Batten Disease Report Assessment
  • Pipeline Product Profiles
  • Key Products and Key Players
  • Market Drivers and Barriers
Key Benefits
  • This report will help to develop Business Strategies by understanding the trends shaping and driving Batten Disease market
  • Organize sales and marketing efforts by identifying the best opportunities for Batten Disease market
  • To understand the future market competition in the Batten Disease market
Note: Product cover images may vary from those shown
2 of 3

FEATURED COMPANIES

  • Amicus Therapeutics
  • BioMarin Pharmaceuticals
  • MORE
1. Key Insights

2. Batten Disease: Market Overview at a Glance
2.1. Total Market Share (%) Distribution of Batten Disease in 2017
2.2. Total Market Share (%) Distribution of Batten Disease in 2028

3. Batten disease: Disease Background and Overview
3.1. Introduction
3.2. History of Neuronal Ceroid Lipofuscinosis
3.3. Clinical Presentation
3.4. Causes
3.5. Pathophysiology
3.6. Inheritance Pattern
3.7. New nomenclature for NCL
3.8. Classification of Batten Disease
3.9. Diagnosis
3.9.1. Diagnostic Algorithm

4. Epidemiology and Patient Population
4.1. Key Findings
4.2. Assumptions and Rationale
4.3. Total Diagnosed Prevalent Population of Batten Disease in 7MM
4.4. Type-Specific Diagnosed Prevalent Population of Batten Disease in the 7MM

5. United States Epidemiology
5.1. Assumptions and Rationale
5.2. Total Diagnosed Prevalent Population of Batten Disease in the United States
5.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in the United States

6. EU5 Epidemiology
6.1. Assumptions and Rationale
6.2. KOL Interviews
6.3. Germany Epidemiology
6.3.1. Assumptions and Rationale
6.3.2. Total Diagnosed Prevalent Population of Batten Disease in Germany
6.3.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in Germany
6.4. France Epidemiology
6.4.1. Assumptions and rationale
6.4.2. Total Diagnosed Prevalent Population of Batten Disease in France
6.4.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in France
6.5. Italy Epidemiology
6.5.1. Assumptions and Rationale
6.5.2. Total Diagnosed Prevalent Population of Batten Disease in Italy
6.5.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in Italy
6.6. Spain Epidemiology
6.6.1. Assumptions and Rationale
6.6.2. Total Diagnosed Prevalent Population of Batten Disease in Spain
6.6.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in Spain
6.7. United Kingdom Epidemiology
6.7.1. Assumptions and Rationale
6.7.2. Total Diagnosed Prevalent Population of Batten Disease in the UK
6.7.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in the UK

7. Japan Epidemiology
7.1. Assumptions and Rationale
7.2. Total Diagnosed Prevalent Population of Batten Disease in the Japan
7.3. Type-Specific Diagnosed Prevalent Population of Batten Disease in the Japan

8. Treatment Algorithm, Current Treatment, and Medical Practices
8.1. Treatment of Batten Disease
8.2. Management of CLN2 Disease

9. Unmet Needs

10. Marketed Products
10.1. Brineura: BioMarin Pharmaceuticals
10.1.1. Drug Description
10.1.2. Mechanism of Action
10.1.3. Regulatory Milestones
10.1.4. Product Development Activities
10.1.5. Advantages & Disadvantages
10.1.6. Safety and Efficacy
10.1.7. Product Profile

11. Emerging Therapies: Clinical
11.1. Key Cross Competition
11.1.1. AAV9-CLN3: Amicus Therapeutics
11.1.2. Drug Description
11.1.3. Other Development Activities
11.1.4. Clinical Development
11.1.5. Product Profile
11.2. scAVV9.CB.CLN6: Amicus Therapeutics
11.2.1. Drug Description
11.2.2. Other Development Activities
11.2.3. Clinical Development
11.2.4. Safety and Efficacy
11.2.5. Product Profile

12. Batten Disease: 7MM Market Analysis
12.1. Key Findings
12.2. Market Size of Batten Disease in 7MM
12.3. Market Forecast Assumptions

13. United States: Market Outlook
13.1. United States Market Size
13.1.1. Total Market size of Batten Disease
13.1.2. Market Size of Batten Disease by Therapies in the US

14. EU-5 countries: Market Outlook
14.1. Germany Market Size
14.1.1. Total Market size of Batten Disease
14.1.2. Market Size of Batten Disease by therapies in Germany
14.2. France Market Size
14.2.1. Total Market size of Batten Disease
14.2.2. Market Size of Batten Disease by therapies in France
14.3. Italy Market Size
14.3.1. Total Market size of Batten Disease
14.3.2. Market Size of Batten Disease by therapies in Italy
14.4. Spain Market Size
14.4.1. Total Market size of Batten Disease
14.4.2. Market Size of Batten Disease by therapies in Spain
14.5. United Kingdom Market Size
14.5.1. Total Market size of Batten Disease
14.5.2. Market Size of Batten Disease by therapies in the UK

15. Japan Market Outlook
15.1. Japan Market Size
15.1.1. Total Market size of Batten Disease
15.1.2. Market Size of Batten Disease by therapies in Japan

16. Market Drivers

17. Market Barriers

18. Appendix
18.1. Report Methodology

19. Publisher Capabilities

20. Disclaimer

21. About the Publisher

List of Tables
Table 1:Classification by Batten Disease (BD) Support and Research Association
Table 2 NCL Classification
Table 3 Classical Forms of Batten Disease
Table 4 Classification of NCL Based on associated genes and age of onset
Table 5 Classification of NCLs based on the age at the onset of clinical manifestations
Table 6 NCL Phenotypes and their associated genes
Table 7 Electron Microscopic Findings and Enzyme Activity by NCL Genotype
Table 8 Molecular Genetic Testing Used in NCL
Table 9 Diagnostic Algorithm for NCL Disease
Table 10 Total Prevalent Population of Batten Disease in the 7MM (2017-2028)
Table 11 Type-Specific Diagnosed Prevalent Population of BD in the 7MM (2017-2028)
Table 12 Total Diagnosed Prevalent Population of Batten Disease in the US (2017-2028)
Table 13 Type-Specific Diagnosed Prevalent Population of BD in the US (2017-2028)
Table 14 Total Diagnosed Prevalent Population of Batten Disease in Germany (2017-2028)
Table 15 Type-Specific Diagnosed Prevalent Population of BD in Germany (2017-2028)
Table 16 Total Prevalent Population of Batten Disease in France (2017-2028)
Table 17 Type-Specific Diagnosed Prevalent Population of BD in France (2017-2028)
Table 18 Total Prevalent Population of Batten Disease in Italy (2017-2028)
Table 19 Type-Specific Diagnosed Prevalent Population of Batten Disease in Italy (2017-2028)
Table 20 Total Diagnosed Prevalent Population of Batten Disease in Spain (2017-2028)
Table 21 Type-Specific Diagnosed Prevalent Population of BD in Spain (2017-2028)
Table 22 Total Diagnosed Prevalent Population of Batten Disease in the UK (2017-2028)
Table 23 Type-Specific Diagnosed Prevalent Population of BD in the UK (2017-2028)
Table 24 Total Diagnosed Prevalent Population of Batten Disease in Japan (2017-2028)
Table 25 Type-Specific Diagnosed Prevalent Population of BD in Japan (2017-2028)
Table 26 Overview on medication for palliative therapies in NCL
Table 27 Common Medication for Batten Disease
Table 28 Goals evolve as CLN2 disease progresses
Table 29 AAV9-CLN3,Clinical Trial Description, 2019
Table 30 scAVV9.CB.CLN6,Clinical Trial Description, 2019
Table 31 Market Size of Batten Disease in 7MM in USD Million (2017-2028)
Table 32 The US Market Size of Batten Disease in USD Million (2017-2028)
Table 33 Market Size of Batten Disease by therapies in the US in USD Million (2017-2028)
Table 34 Germany Market Size of Batten Disease in USD Million (2017-2028)
Table 35 Market Size of Batten Disease by therapies in Germany in USD Million (2017-2028)
Table 36 France Market Size of Batten Disease in USD Million (2017-2028)
Table 37 Market Size of Batten Disease by therapies in France in USD Million (2017-2028)
Table 38 Italy Market Size of Batten Disease in USD Million (2017-2028)
Table 39 Market Size of Batten Disease by therapies in Italy in USD Million (2017-2028)
Table 40 Spain Market Size of Batten Disease in USD Million (2017-2028)
Table 41 Market Size of Batten Disease by therapies in Spain in USD Million (2017-2028)
Table 42 The UK Market Size of Batten Disease in USD Million (2017-2028)
Table 43 Market Size of Batten Disease by therapies in the UK in USD Million (2017-2028)
Table 44 Japan Market Size of Batten Disease in USD Million (2017-2028)
Table 45 Market Size of Batten Disease by therapies in Japan in USD Million (2017-2028)

List of Figures
Figure 1:CLN protein distribution and their link to autophagy defects in neuronal ceroid lipofuscinoses
Figure 2 Batten’s Disease inheritance pattern
Figure 3 Forms of Batten Disease
Figure 4 Recommended Diagnostic Algorithm
Figure 5 Diagnostic Algorithm for Batten Disease
Figure 6 Recommended Diagnostic Algorithm for CLN2
Figure 7 Total Prevalent Population of Batten Disease in the 7MM (2017-2028)
Figure 8 Type-Specific Diagnosed Prevalent Population of Batten Disease in the 7MM (2017-2028)
Figure 9 Total Diagnosed Prevalent Population of Batten Disease in the US (2017-2028)
Figure 10 Type-Specific Diagnosed Prevalent Population of Batten Disease in the US (2017-2028)
Figure 11 Total Diagnosed Prevalent Population of Batten Disease in Germany (2017-2028)
Figure 12 Type-Specific Diagnosed Prevalent Population of Batten Disease in Germany (2017-2028)
Figure 13 Total Diagnosed Prevalent Population of Batten Disease in France (2017-2028)
Figure 14 Type-Specific Diagnosed Prevalent Population of Batten Disease in France (2017-2028)
Figure 15 Total Diagnosed Prevalent Population of Batten Disease in Italy (2017-2028)
Figure 16 Type-Specific Diagnosed Prevalent Population of Batten Disease in Italy (2017-2028)
Figure 17 Total Diagnosed Prevalent Population of Batten Disease in Spain (2017-2028)
Figure 18 Type-Specific Diagnosed Prevalent Population of Batten Disease in Spain (2017-2028)
Figure 19 Total Diagnosed Prevalent Population of Batten Disease in the UK (2017-2028)
Figure 20 Type-Specific Diagnosed Prevalent Population of Batten Disease in the UK (2017-2028)
Figure 21 Total Diagnosed Prevalent Population of Batten Disease in Japan (2017-2028)
Figure 22 Type-Specific Diagnosed Prevalent Population of Batten Disease in Japan (2017-2028)
Figure 23 Palliative care framework for CLN2 management facilitates comprehensive care of patients
Figure 24 Unmet Needs of Batten Disease
Figure 25 Market Size of Batten Disease in the 7MM in USD Million (2017-2028)
Figure 26 Market Size of Batten Disease in the US, USD Millions (2017-2028)
Figure 27 Market Size of Batten Disease by therapies in the United States in USD Million (2017-2028)
Figure 28 Market Size of Batten Disease in Germany, USD Millions (2017-2028)
Figure 29 Market Size of Batten Disease by therapies in Germany in USD Million (2017-2028)
Figure 30 Market Size of Batten Disease in France, USD Millions (2017-2028)
Figure 31 Market Size of Batten Disease by therapies in France in USD Million (2017-2028)
Figure 32 Market Size of Batten Disease in Italy, USD Millions (2017-2028)
Figure 33 Market Size of Batten Disease by therapies in Italy in USD Million (2017-2028)
Figure 34 Market Size of Batten Disease in Spain, USD Millions (2017-2028)
Figure 35 Market Size of Batten Disease by therapies in Spain in USD Million (2017-2028)
Figure 36 Market Size of Batten Disease in the UK, USD Millions (2017-2028)
Figure 37 Market Size of Batten Disease by therapies in the UK in USD Million (2017-2028)
Figure 38 Market Size of Batten Disease in the Japan, USD Millions (2017-2028)
Figure 39 Market Size of Batten Disease by therapies in Japan in USD Million (2017-2028)
Figure 40 Market Drivers
Figure 41 Market Barriers
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