Batten Disease Market

Market Overview:

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, EU4 (Germany, Spain, Italy, and France), 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, 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.

Recent Developments:

• In April 2024, Theranexus and the Beyond Batten Disease Foundation (BBDF) revealed final good efficacy and safety findings from the Phase I/II trial of its therapeutic candidate Batten-1 in juvenile Batten disease (CLN3) following 18 months of treatment.

Key Highlights:

• Batten disease is the most prevalent neurological condition in childhood.
• It can originate from mutations in 1-13 genes, and the global prevalence of Batten disease is approximately one in 100,000 live births.
• In the United States, it affects an estimated two to four out of every 100,000 children.
• The CLN3 mutation has reported occurrences ranging from 0.02-4.8 per 100,000 people worldwide.
• The estimated frequencies for Finnish and non-Finnish Europeans are 1/558 and 1/380, respectively, while for Latinos and Americans, they are 1/1169 and 1/506.

Drugs:

BRINEURA is an enzyme replacement medication that is intended to decrease the loss of the ability to walk or crawl (ambulation) in juvenile patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), commonly known as tripeptidyl peptidase 1 (TPP1) deficiency, a kind of Batten disease.

AT-GTX-502 is an innovative gene therapy in Phase I/II clinical trials for CLN3 Batten disease. AT-GTX-502 is administered in a single infusion to deliver a functional copy of the CLN3 gene to cells in the central nervous system. The therapy is aimed at targeting the underlying enzyme shortage that causes increasing cell damage and neurodevelopmental and physical deterioration.

NGN-101 is under clinical development by Neurogene for the treatment of Batten disease. It is delivered by the intracerebroventricular (ICV) and intravitreal (IVT) methods. It operates by targeting CLN5. The therapy consists of an adeno-associated virus (AAV) that expresses a healthy copy of CLN5.

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?