Summary

The 7 major Batten disease markets are expected to exhibit a CAGR of 5.55% during 2024-2034.

The Batten disease market has been comprehensively analyzed in IMARC's new report titled "Batten Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Batten disease, also known as neuronal ceroid lipofuscinosis, refers to a rare and progressive genetic disorder that primarily affects the nervous system. It is characterized by the accumulation of lipopigments, specifically ceroid and lipofuscin, in various tissues throughout the body, particularly in the nerve cells of the brain. Afflicting mainly children, it begins stealthily, often with subtle signs like vision problems, clumsiness, personality changes, etc. As the disease advances, affected individuals suffer from seizures, cognitive decline, as well as motor impairments, such as loss of coordination and muscle weakness. Vision deterioration persists, leading to blindness, and speech difficulties arise, making communication challenging. In its later stages, Batten disease ravages the nervous system, causing severe dementia, complete loss of motor function, and, ultimately, a shortened life expectancy. The diagnostic process often involves a combination of clinical evaluation, genetic testing, neuroimaging studies to check seizure activity, visual assessment, and biochemical analysis of blood and urine samples to detect biomarkers associated with the ailment. Neuropsychological assessments are also crucial in evaluating cognitive function. Besides this, genetic testing, including next-generation sequencing, plays a central role in confirming the diagnosis by identifying pathogenic variants in genes associated with Batten disease.

The rising cases of mutations in various genes responsible for the production of enzymes involved in lysosomal function are primarily driving the Batten disease market. Besides this, the increasing utilization of electroencephalography (EEG) for assessing and characterizing seizures in patients is also augmenting the market growth. Specific EEG patterns, such as slow waves and epileptiform discharges, can aid in differentiating the ailment from other epileptic conditions. Furthermore, the widespread adoption of chaperone therapy, which aims to develop small molecules that can stabilize misfolded proteins and improve their function, thereby potentially slowing disease progression, is creating a positive outlook for the market. Additionally, several key players are making extensive investments in R&D activities to introduce drugs that enhance lysosomal function, reduce neuroinflammation, promote neuroprotection, and modify disease-related pathways. This, in turn, is further acting as another significant growth-inducing factor. Moreover, the emerging popularity of autophagy modulation, since it promotes the removal of damaged proteins and organelles, thereby reducing cellular stress as well as promoting neuronal health, is expected to drive the Batten disease market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Batten disease market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Batten disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Batten disease market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Batten disease market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Batten disease market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Batten disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the Batten disease market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Batten disease market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Batten disease market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of Batten disease across the seven major markets?
What is the number of prevalent cases (2018-2034) of Batten disease by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Batten disease by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of Batten disease by type across the seven major markets?
How many patients are diagnosed (2018-2034) with Batten disease across the seven major markets?
What is the size of the Batten disease patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Batten disease?
What will be the growth rate of patients across the seven major markets?

Batten Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Batten disease drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Batten disease market?
What are the key regulatory events related to the Batten disease market?
What is the structure of clinical trial landscape by status related to the Batten disease market?
What is the structure of clinical trial landscape by phase related to the Batten disease market?
What is the structure of clinical trial landscape by route of administration related to the Batten disease market?

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Table of Contents

​1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Batten Disease - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Batten Disease - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Batten Disease - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Epidemiology by Type (2018-2034)
7.2.6 Diagnosed Cases (2018-2034)
7.2.7 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Epidemiology by Type (2018-2034)
7.3.6 Diagnosed Cases (2018-2034)
7.3.7 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Epidemiology by Type (2018-2034)
7.4.6 Diagnosed Cases (2018-2034)
7.4.7 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Epidemiology by Type (2018-2034)
7.5.6 Diagnosed Cases (2018-2034)
7.5.7 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Epidemiology by Type (2018-2034)
7.6.6 Diagnosed Cases (2018-2034)
7.6.7 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Epidemiology by Type (2018-2034)
7.7.6 Diagnosed Cases (2018-2034)
7.7.7 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Epidemiology by Type (2018-2034)
7.8.6 Diagnosed Cases (2018-2034)
7.8.7 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Epidemiology by Type (2018-2034)
7.9.6 Diagnosed Cases (2018-2034)
7.9.7 Patient Pool/Treated Cases (2018-2034)

8 Batten Disease - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Batten Disease - Unmet Needs

10 Batten Disease - Key Endpoints of Treatment

11 Batten Disease - Marketed Products
11.1 List of Batten Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Brineura (Cerliponase alfa) - BioMarin Pharmaceutical
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Batten Disease - Pipeline Drugs
12.1 List of Batten Disease Pipeline Drugs Across the Top 7 Markets
12.1.1 AT GTX 502 - Amicus Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 NGN101 - Neurogene Inc
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 RGX381 - REGENXBIO
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Batten Disease - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Batten Disease – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Batten Disease - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Batten Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Batten Disease - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Batten Disease - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Batten Disease - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Batten Disease - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Batten Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Batten Disease - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Batten Disease - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Batten Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Batten Disease - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Batten Disease - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Batten Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Batten Disease - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Batten Disease - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Batten Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Batten Disease - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Batten Disease - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Batten Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Batten Disease - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Batten Disease - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Batten Disease - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Batten Disease - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Batten Disease - Access and Reimbursement Overview

16 Batten Disease - Recent Events and Inputs From Key Opinion Leaders

17 Batten Disease Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Batten Disease Market – Strategic Recommendations

19 Appendix