This Brineura market report delivers an in-depth analysis of the market’s key characteristics, including size, growth potential, and segmentation. It provides a detailed breakdown of the market across major regions and leading countries, highlighting historical data and future growth projections. The report also examines the competitive landscape, market share insights, emerging trends, and strategic developments shaping the market.
Market growth during the historic period can be attributed to factors such as the rising prevalence of Batten disease, increased awareness of rare neurodegenerative disorders, growth in pediatric-focused therapies, improvements in diagnostic capabilities, and more regulatory approvals for enzyme replacement therapies.
The growth is expected to be driven by factors such as rising healthcare expenditure, increased adoption of orphan drugs, a growing focus on personalized medicine, improved access to rare disease diagnostics, and higher investments in biologics. Key trends during this period include the expansion of gene therapy research, the growing use of AI in drug discovery, a focus on patient-centric therapies, increased collaborations between pharmaceutical companies, and a rise in the adoption of digital health solutions.
The increasing prevalence of genetic disorders is expected to drive the growth of the brineura market in the future. Genetic disorders are medical conditions caused by mutations or abnormalities in an individual’s DNA, whether in genes or chromosomes. The rising incidence of these disorders is attributed to factors such as improved detection and awareness through advanced diagnostic tools, better access to healthcare, ongoing advancements in genetic research, and changes in reproductive trends. Brineura is used in the treatment of genetic disorders to provide enzyme replacement therapy directly to the central nervous system, helping slow disease progression and improve neurological function. For example, a report from the United Kingdom Hemophilia Centre Doctors' Organization (UKHCDO) in November 2022 noted an increase in new registrations for Hemophilia A in the UK, from 16 cases in 2021 to 19 in 2022. As a result, the growing prevalence of genetic disorders is driving the expansion of the brineura market.
The rise in healthcare expenditure is also expected to contribute to the growth of the brineura market. Healthcare expenditure refers to the total financial resources spent on healthcare services, including treatment, infrastructure, and programs. The increase in healthcare spending is driven by factors such as an aging population, advancements in medical technology, a rise in chronic diseases, inflation, and higher expectations for quality care. Healthcare expenditure plays a critical role in making Brineura more accessible, as the high cost of the treatment requires substantial funding for rare disease therapies, affecting insurance coverage and affordability. For instance, according to a report published by the Office for National Statistics in May 2024, total healthcare expenditure in the UK increased by 5.6% in nominal terms between 2022 and 2023, compared to a growth of 0.9% in 2022. Therefore, the rise in healthcare expenditure is expected to drive the growth of the brineura market.
A significant trend in the brineura market is the development of innovative pediatric therapies for neurodegenerative disorders, particularly those designed for early intervention in children of all ages, including presymptomatic cases, to slow disease progression and improve outcomes. Pediatric neurodegenerative therapies are treatments tailored to address progressive neurological disorders in children, which lead to the degeneration of nerve cells and can cause severe cognitive and motor impairments. For example, in July 2024, BioMarin Pharmaceutical Inc., a U.S.-based biotechnology company, announced that the FDA had approved Brineura (cerliponase alfa) for children under the age of 3 with CLN2 disease. This approval expanded its use to all ages, including presymptomatic patients, marking a significant step forward in early intervention for this rare genetic condition. Clinical data showed that early treatment slowed motor decline and helped preserve motor function in children under 3, providing evidence of the benefits of timely intervention.
The key company operating in the brineura market is BioMarin Pharmaceutical Inc.
North America was the largest region in the brineura market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in brineura report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the brineura market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Brineura is a biologic enzyme replacement therapy designed to treat neuronal ceroid lipofuscinosis type 2 (CLN2), a rare genetic lysosomal storage disorder that affects the central nervous system. It works by replacing the deficient enzyme tripeptidyl peptidase 1 (TPP1) in order to slow the progression of motor function decline in pediatric patients with CLN2 disease.
The main indications for brineura are neuronal ceroid lipofuscinosis type 2 (CLN2) and other lysosomal storage disorders. CLN2 is a rare, inherited neurodegenerative disorder marked by the progressive loss of cognitive and motor skills, leading to severe impairment and early death due to the accumulation of lipofuscin in the brain. Brineura is used in various healthcare settings, including hospitals, clinics, ambulatory care, and home care. It is distributed through multiple channels, such as direct sales, pharmaceutical wholesalers, online pharmacies, and retail pharmacies.
The brineura market research report is one of a series of new reports that provides brineura market statistics, including brineura industry global market size, regional shares, competitors with a brineura market share, detailed brineura market segments, market trends and opportunities, and any further data you may need to thrive in the brineura industry. This brineura market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The brineura market consists of sales of brineura injections, brineura administration kits, dosage adjustments, and special formulations. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Market growth during the historic period can be attributed to factors such as the rising prevalence of Batten disease, increased awareness of rare neurodegenerative disorders, growth in pediatric-focused therapies, improvements in diagnostic capabilities, and more regulatory approvals for enzyme replacement therapies.
The growth is expected to be driven by factors such as rising healthcare expenditure, increased adoption of orphan drugs, a growing focus on personalized medicine, improved access to rare disease diagnostics, and higher investments in biologics. Key trends during this period include the expansion of gene therapy research, the growing use of AI in drug discovery, a focus on patient-centric therapies, increased collaborations between pharmaceutical companies, and a rise in the adoption of digital health solutions.
The increasing prevalence of genetic disorders is expected to drive the growth of the brineura market in the future. Genetic disorders are medical conditions caused by mutations or abnormalities in an individual’s DNA, whether in genes or chromosomes. The rising incidence of these disorders is attributed to factors such as improved detection and awareness through advanced diagnostic tools, better access to healthcare, ongoing advancements in genetic research, and changes in reproductive trends. Brineura is used in the treatment of genetic disorders to provide enzyme replacement therapy directly to the central nervous system, helping slow disease progression and improve neurological function. For example, a report from the United Kingdom Hemophilia Centre Doctors' Organization (UKHCDO) in November 2022 noted an increase in new registrations for Hemophilia A in the UK, from 16 cases in 2021 to 19 in 2022. As a result, the growing prevalence of genetic disorders is driving the expansion of the brineura market.
The rise in healthcare expenditure is also expected to contribute to the growth of the brineura market. Healthcare expenditure refers to the total financial resources spent on healthcare services, including treatment, infrastructure, and programs. The increase in healthcare spending is driven by factors such as an aging population, advancements in medical technology, a rise in chronic diseases, inflation, and higher expectations for quality care. Healthcare expenditure plays a critical role in making Brineura more accessible, as the high cost of the treatment requires substantial funding for rare disease therapies, affecting insurance coverage and affordability. For instance, according to a report published by the Office for National Statistics in May 2024, total healthcare expenditure in the UK increased by 5.6% in nominal terms between 2022 and 2023, compared to a growth of 0.9% in 2022. Therefore, the rise in healthcare expenditure is expected to drive the growth of the brineura market.
A significant trend in the brineura market is the development of innovative pediatric therapies for neurodegenerative disorders, particularly those designed for early intervention in children of all ages, including presymptomatic cases, to slow disease progression and improve outcomes. Pediatric neurodegenerative therapies are treatments tailored to address progressive neurological disorders in children, which lead to the degeneration of nerve cells and can cause severe cognitive and motor impairments. For example, in July 2024, BioMarin Pharmaceutical Inc., a U.S.-based biotechnology company, announced that the FDA had approved Brineura (cerliponase alfa) for children under the age of 3 with CLN2 disease. This approval expanded its use to all ages, including presymptomatic patients, marking a significant step forward in early intervention for this rare genetic condition. Clinical data showed that early treatment slowed motor decline and helped preserve motor function in children under 3, providing evidence of the benefits of timely intervention.
The key company operating in the brineura market is BioMarin Pharmaceutical Inc.
North America was the largest region in the brineura market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in brineura report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the brineura market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Brineura is a biologic enzyme replacement therapy designed to treat neuronal ceroid lipofuscinosis type 2 (CLN2), a rare genetic lysosomal storage disorder that affects the central nervous system. It works by replacing the deficient enzyme tripeptidyl peptidase 1 (TPP1) in order to slow the progression of motor function decline in pediatric patients with CLN2 disease.
The main indications for brineura are neuronal ceroid lipofuscinosis type 2 (CLN2) and other lysosomal storage disorders. CLN2 is a rare, inherited neurodegenerative disorder marked by the progressive loss of cognitive and motor skills, leading to severe impairment and early death due to the accumulation of lipofuscin in the brain. Brineura is used in various healthcare settings, including hospitals, clinics, ambulatory care, and home care. It is distributed through multiple channels, such as direct sales, pharmaceutical wholesalers, online pharmacies, and retail pharmacies.
The brineura market research report is one of a series of new reports that provides brineura market statistics, including brineura industry global market size, regional shares, competitors with a brineura market share, detailed brineura market segments, market trends and opportunities, and any further data you may need to thrive in the brineura industry. This brineura market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The brineura market consists of sales of brineura injections, brineura administration kits, dosage adjustments, and special formulations. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Brineura Market Characteristics4. Brineura Market Trends and Strategies5. Brineura Market -Macro Economic Scenario Including the Impact of Interest Rates, Inflation, Geopolitics, and the Recovery from COVID-19 on the Market7. Global Brineura Pricing Analysis & Forecasts30. Global Brineura Market Competitive Benchmarking and Dashboard31. Key Mergers and Acquisitions in the Brineura Market32. Recent Developments in the Brineura Market
3. Brineura Market Biologic Drug Characteristics
6. Global Brineura Growth Analysis and Strategic Analysis Framework
8. Brineura Market Segmentation
9. Global Brineura Epidemiology of Clinical Indications
10. Brineura Market Regional and Country Analysis
11. Asia-Pacific Brineura Market
12. China Brineura Market
13. India Brineura Market
14. Japan Brineura Market
15. Australia Brineura Market
16. South Korea Brineura Market
17. Western Europe Brineura Market
18. UK Brineura Market
19. Germany Brineura Market
20. France Brineura Market
21. Eastern Europe Brineura Market
22. North America Brineura Market
23. USA Brineura Market
24. Canada Brineura Market
25. South America Brineura Market
26. Middle East Brineura Market
27. Africa Brineura Market
28. Brineura Market Competitive Landscape and Company Profiles
29. Global Brineura Market Pipeline Analysis
33. Brineura Market High Potential Countries, Segments and Strategies
34. Appendix
Executive Summary
Brineura Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on brineura market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for brineura? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The brineura market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) by Indication: Neuronal Ceroid Lipofuscinosis Type 2 (CLN2); Other Lysosomal Storage Disorders2) by End Use: Hospitals; Clinics; Ambulatory Care; Home Care
3) by Distribution Channel: Direct Sales; Pharmaceutical Wholesalers; Online Pharmacies; Retail Pharmacies
Key Companies Mentioned: BioMarin Pharmaceutical Inc.
Countries: Australia, India, China, South Korea, Japan, UK, France, Germany, USA, Canada
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
- BioMarin Pharmaceutical Inc.
