DelveInsight’s “Batten Disease Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Batten Disease market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Batten Disease drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Batten Disease treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Batten Disease: An Overview
Batten disease, referred to as Neuronal Ceroid Lipofuscinosis (NCL), is a rare and debilitating autosomal recessive neurodegenerative lysosomal storage disorder 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.
Patients with Batten disease have defects in the involved process of lipofuscin breakdown. It accumulates the inner cells in lysosomes and thereby leads to the deterioration of neurons in both the brain and retina. Mutations in recessively inherited human genes generally cause the disease.
All individuals with neuronal ceroid-lipofuscinosis have a progressive decline and can experience evolving cognitive and motor disorders, and seizures. With the exception of Adult Neuronal Ceroid- Lipofuscinosis (ANCL) and Northern Epilepsy (NE), NCL phenotypes are usually associated with progressive loss of vision.
As per the National Institute of Neurological Disorders and Stroke, It is not known how many people have Batten disease, but by some estimates, it can be as frequent as 1 in 12,500 people in some populations. It affects an estimated 2 to 4 out of every 100,000 children in the United States. Many more individuals may be carriers of a defective gene that can cause any of the NCL diseases.
Among the EU5 countries, Germany had the highest market size, while Spain had the lowest market size for 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 the expected launch of upcoming therapies.
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 the cure for various forms of the disease. To name a few of these: REGENXBIO, Polaryx Therapeutics, Spark Therapeutics, Abeona Therapeutics, Neurogene, Lexeo Therapeutics, Amicus Therapeutics, Polaryx Therapeutics, and others.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Batten Disease pipeline therapies. It also thoroughly assesses the Batten Disease market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the market trend for each marketed Batten Disease drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Batten Disease Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Batten Disease epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Batten Disease epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Batten Disease Epidemiology, Segmented as –
Total Diagnosed Prevalent Population of Batten Disease in the 7MM (2019–2032)
Type-Specific Diagnosed Prevalent Population of Batten Disease in the 7MM (2019–2032)
Batten Disease Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Batten Disease market or expected to be launched during the study period. The analysis covers the Batten Disease market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Batten Disease drugs based on their sale and market share.
The report also covers the Batten Disease pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Batten Disease companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Learn How the Batten Disease Market Will Evolve and Grow by 2032 @
Batten Disease Therapeutics Analysis
Currently, there is no cure for Batten Disease. No specific treatment is yet available to cure or slow down the progression of Batten disease, but treatment exists to manage the symptoms and make the patient more comfortable. Only one drug is so far approved by the US Food and Drug Administration and European Medicines Agency (EMA), Brineura, specifically for the treatment of CLN2 form of the disease in the United States, EU-5 countries, and Japan.
To further improve the treatment scenario, several major pharma and biotech companies are developing therapies for Batten Disease. Currently, Polaryx Therapeutics is leading the therapeutics market with its Batten Disease drug candidates in the most advanced stage of clinical development.
Batten Disease Companies Actively Working in the Therapeutics Market Include
REGENXBIO, Polaryx Therapeutics, Spark Therapeutics, Abeona Therapeutics, Neurogene, Lexeo Therapeutics, Amicus Therapeutics, and others.
Emerging and Marketed Batten Disease Therapies Covered in the Report Include:
PLX-200: Polaryx Therapeutics
NGN-101: Neurogene Inc
And Many Others
The Report Covers an In-depth Assessment of Emerging Drugs and key Companies. Download the Sample Report to Learn More @
Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Batten Disease Competitive Intelligence Analysis
4. Batten Disease Market Overview at a Glance
5. Batten Disease Disease Background and Overview
6. Batten Disease Patient Journey
7. Batten Disease Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Batten Disease Treatment Algorithm, Current Treatment, and Medical Practices
9. Batten Disease Unmet Needs
10. Key Endpoints of Batten Disease Treatment
11. Batten Disease Marketed Therapies
12. Batten Disease Emerging Drugs and Latest Therapeutic Advances
13. Batten Disease Seven Major Market Analysis
14. Attribute Analysis
15. Batten Disease Market Outlook (In US, EU5, and Japan)
16. Batten Disease Companies Active in the Market
17. Batten Disease Access and Reimbursement Overview
18. KOL Views on the Batten Disease Market
19. Batten Disease Market Drivers
20. Batten Disease Market Barriers
22. DelveInsight Capabilities
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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