PUBLISHER: The Business Research Company | PRODUCT CODE: 1751057
PUBLISHER: The Business Research Company | PRODUCT CODE: 1751057
Niemann-Pick disease type C (NPC) treatment refers to the medical strategies used to manage and slow the progression of Niemann-Pick disease type C, a rare inherited neurodegenerative disorder that impairs the body's ability to metabolize cholesterol and other lipids within cells. Treatment typically involves pharmacological and supportive therapies aimed at reducing neurological symptoms, stabilizing the disease, and improving the quality of life for affected individuals.
Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.
The main types of Niemann-Pick disease type C treatment focus on Niemann-Pick disease type C1 and Niemann-Pick disease type C2. Niemann-Pick disease type C1 (NPC1) is a rare, progressive, and fatal lysosomal storage disorder caused by mutations in the NPC1 gene. This leads to impaired cholesterol trafficking within cells, resulting in the accumulation of lipids in lysosomes, and causing severe neurological deterioration such as cognitive decline, motor dysfunction, and organ damage. NPC treatment spans various age groups, including infantile, juvenile, and adult populations, with end-users including hospitals, specialty clinics, and research and academic institutions.
The niemann-Pick disease type C treatment market research report is one of a series of new reports from The Business Research Company that provides niemann-Pick disease type C treatment market statistics, including niemann-Pick disease type C treatment industry global market size, regional shares, competitors with a niemann-Pick disease type C treatment market share, detailed niemann-Pick disease type C treatment market segments, market trends and opportunities, and any further data you may need to thrive in the niemann-Pick disease type C treatment industry. This niemann-Pick disease type C treatment 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 niemann-Pick disease type C treatment market size has grown strongly in recent years. It will grow from $2.68 billion in 2024 to $2.93 billion in 2025 at a compound annual growth rate (CAGR) of 9.2%. The growth observed during the historic period can be attributed to the expansion of healthcare infrastructure, a rising number of cases involving mutations in the NPC1 and NPC2 genes, a growing prevalence of Niemann-Pick disease, increased government funding for research into rare disease treatments, and heightened awareness and advocacy efforts.
The niemann-Pick disease type C treatment market size is expected to see strong growth in the next few years. It will grow to $4.14 billion in 2029 at a compound annual growth rate (CAGR) of 9.1%. The growth projected during the forecast period can be attributed to increasing research and development activities, a growing number of pipeline drugs, a high prevalence of rare diseases, the emerging popularity of gene therapy, and significant advancements in genetic therapies. Key trends during this period include advancements in technology, the rise of personalized medicine for lysosomal storage disorders, progress in genetic and molecular research, breakthroughs in biotechnology, and the development of innovative therapeutics.
The growing prevalence of Alzheimer's disease is expected to accelerate the expansion of the Niemann-Pick disease type C (NPC) treatments market. Alzheimer's disease is a progressive neurodegenerative disorder characterized by the buildup of beta-amyloid plaques and tau protein tangles in the brain, leading to cognitive decline, memory loss, and difficulties with daily functioning, ultimately resulting in severe dementia and neurodegeneration. The increasing prevalence of Alzheimer's disease is largely due to the aging global population. As life expectancy increases, especially in developed nations, the risk of age-related diseases such as Alzheimer's also rises, since advancing age is the most significant known risk factor for the condition. NPC treatment contributes to Alzheimer's disease research by offering insights into lysosomal storage disorders and dysfunctions in lipid metabolism. For example, in May 2024, the Alzheimer's Society, a UK-based health organization, estimated that 982,000 people in the UK were living with dementia in 2024, with more than a third undiagnosed. This number is expected to reach 1.4 million by 2040. Therefore, the growing prevalence of Alzheimer's disease is driving the growth of the Niemann-Pick disease type C treatments market.
Leading companies in the Niemann-Pick disease type C (NPC) treatment market are focusing on developing innovative therapies, such as neuroprotective treatments, aimed at slowing disease progression and improving patient outcomes. Neuroprotective therapies are designed to protect the brain and nervous system from damage or degeneration, with the goal of preserving neurological function in conditions such as neurodegenerative diseases. For example, in September 2024, IntraBio Inc., a US-based biopharmaceutical company, received approval from the United States Food and Drug Administration (FDA) for AQNEURSA (levacetylleucine), the first stand-alone therapy for Niemann-Pick Disease Type C (NPC). This approval followed promising Phase III trial results, showing significant neurological improvements in NPC patients within 12 weeks. AQNEURSA offers a long-awaited treatment option for individuals suffering from this rare lysosomal storage disorder, which affects movement, cognition, and daily function. IntraBio also intends to explore AQNEURSA's potential in treating other neurodegenerative and neurodevelopmental disorders.
In December 2023, Cyclo Therapeutics, Inc., a US-based biotechnology firm, merged with Applied Molecular Transport Inc. for $10.2 million. This merger is aimed at strengthening Cyclo Therapeutics' financial and strategic position by utilizing additional resources, extending its financial runway, and advancing the clinical development and regulatory approval process for Trappsol Cyclo, a potential treatment for Niemann-Pick Disease Type C1 (NPC1). This treatment will undergo the pivotal Phase 3 global study (TransportNPC). Applied Molecular Transport Inc. is a US-based clinical-stage biopharmaceutical company.
Major players in the niemann-pick disease type c treatment market are Merck & Co. Inc., Mayo Clinic, Boston Children's Hospital, Cincinnati Children's Hospital Medical Center, Mallinckrodt Pharmaceuticals, Myriad Genetics Inc., Rainbow Children's Hospital, CENTOGENE N.V., The Florey Institute of Neuroscience and Mental Health, Insilico Medicine Inc., National Organization for Rare Disorders (NORD), Zevra Therapeutics Inc., Azafaros A.G., IntraBio Inc., National Niemann-Pick Disease Foundation (NNPDF), Bloomsbury Genetic Therapies Ltd., CTD Holdings Inc., Okklo Life Sciences, Vtesse Inc., Cyclo Therapeutics Inc., Mandos Health.
North America was the largest region in the niemann-pick disease type C treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in niemann-Pick disease type C treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the niemann-Pick disease type C treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The niemann-Pick disease type C treatment market includes revenues earned by entities through medication management, symptom management, neurological care, liver care, and genetic counseling. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.
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.
Niemann-Pick Disease Type C Treatment Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses on niemann-pick disease type c treatment 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.
Where is the largest and fastest growing market for niemann-pick disease type c treatment ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The niemann-pick disease type c treatment market global report from the Business Research Company 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 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.