Batten Disease Market Size
DelveInsight's Batten Disease- Market Insights, Epidemiology and Market Forecast-2028 report delivers an in-depth understanding of the disease, historical and 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.
• The United States
• EU5 (Germany, France, Italy, Spain and the United Kingdom)
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 DelveInsight's 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 DelveInsight's 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 DelveInsight 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
• This DelveInsight 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.