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Niemann-Pick Disease (NPD) Global Market Report 2025
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Niemann-Pick disease (NPD) is a rare inherited lysosomal storage disorder caused by mutations in the SMPD1 (Types A and B) or NPC1/NPC2 (Type C) genes, resulting in excessive lipid accumulation in cells. This buildup leads to progressive damage to the brain, liver, spleen, and other organs. The symptoms of NPD vary by type but may include neurodegeneration, hepatosplenomegaly (enlargement of the liver and spleen), respiratory complications, and developmental delays.

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 are Type A, Type B, and Type C. Niemann-Pick disease type A is a rare and severe genetic disorder caused by a deficiency of the enzyme acid sphingomyelinase (ASM), leading to the accumulation of sphingomyelin in various organs. Treatment for this type includes drugs, physical therapy, and other interventions, administered through various routes such as oral, intravenous, and subcutaneous. Distribution channels for these treatments include hospital pharmacies, retail pharmacies, and others, and they are used by various end users, including hospitals, clinics, and home care settings.

The niemann-pick disease (NPD) market research report is one of a series of new reports from The Business Research Company that provides niemann-pick disease (NPD) market statistics, including niemann-pick disease (NPD) industry global market size, regional shares, competitors with a niemann-pick disease (NPD) market share, detailed niemann-pick disease (NPD) market segments, market trends and opportunities, and any further data you may need to thrive in the niemann-pick disease (NPD) industry. This niemann-pick disease (NPD) 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 (NPD) market size has grown rapidly in recent years. It will grow from$0.97 billion in 2024 to $1.07 billion in 2025 at a compound annual growth rate (CAGR) of 11.3%. The growth during the historic period can be attributed to the rising number of genetic counselors, increased funding for rare disease research, growing investment from venture capital firms, the increasing use of biomarkers, and enhanced healthcare access.

The niemann-pick disease (NPD) market size is expected to see rapid growth in the next few years. It will grow to$1.63 billion in 2029 at a compound annual growth rate (CAGR) of 11.0%. The growth during the forecast period can be attributed to the growing demand for personalized medicine, increased interest in lysosomal storage disorders, rising regulatory approvals for orphan drugs, increasing demand for targeted therapies, and the growing need for advanced diagnostic tools. Key trends in the forecast period include advancements in treatment options, technological innovations in diagnostic tools, integration of artificial intelligence in diagnosis, innovation in enzyme replacement therapies (ERT), and the development of clinical trials for new drugs.

The increasing adoption of gene therapy is expected to drive the growth of the Niemann-Pick disease (NPD) market in the coming years. Gene therapy is a medical technique that modifies or manipulates an individual's genes to treat or prevent diseases. The rise in gene therapy is fueled by advancements in genetic engineering, which enable precise and effective gene modifications that improve treatment outcomes for genetic disorders. In the case of Niemann-Pick disease (NPD), gene therapy targets the underlying genetic mutations to correct enzyme deficiencies that cause the disorder and enhance cellular function. For example, in January 2024, the American Society of Gene and Cell Therapy, a U.S.-based organization focused on gene and cell therapy, reported that the number of gene therapies in Phase III trials increased by 10% in the fourth quarter of 2023 compared to the previous quarter, marking the first rise since the third quarter of 2022. Thus, the growing adoption of gene therapy is fueling the expansion of the Niemann-Pick disease (NPD) market.

Key players in the Niemann-Pick disease (NPD) market are focusing on developing advanced treatments, such as combination therapy, to improve treatment efficacy and patient outcomes. Combination therapy involves using two or more therapeutic agents or strategies to treat a disease, enhancing effectiveness and reducing resistance to single-agent treatments. For example, in September 2024, Zevra Therapeutics Inc., a U.S.-based biopharmaceutical company, received U.S. Food and Drug Administration (FDA) approval for MIPLYFFA, combined with miglustat, for treating the neurological symptoms of Niemann-Pick disease type C (NPC) in both adult and pediatric patients aged 2 years and older. MIPLYFFA (arimoclomol) works by boosting the production of heat shock proteins (HSPs), which help clear toxic lipid buildup and protect neurons from damage. Meanwhile, miglustat inhibits glucosylceramide synthase, reducing lipid accumulation. Together, these therapies help slow the progression of neurological decline in NPC patients.

In May 2022, Zevra Therapeutics Inc. (formerly KemPharm Inc.), a U.S.-based pharmaceutical company, acquired arimoclomol from Orphazyme A/S for $12.8 million. This acquisition allows Zevra to strengthen its rare disease pipeline by gaining access to arimoclomol, a promising heat shock protein amplifier developed for treating Niemann-Pick disease type C (NPC). Orphazyme A/S, a Denmark-based biopharmaceutical company, specializes in developing treatments for rare neurodegenerative diseases, including Niemann-Pick disease type C (NPC).

Major players in the niemann-pick disease (NPD) market are Sanofi S.A., Mayo Clinic, Cincinnati Children's Hospital Medical Center, Boston Children's Hospital, Mallinckrodt Pharmaceuticals plc, Myriad Genetics, CENTOGENE N.V., The Florey Institute of Neuroscience and Mental Health, National Organization for Rare Disorders (NORD), Evox Therapeutics Limited, Zevra Therapeutics Inc., IntraBio Inc., Dipharma SA, Okklo Life Sciences, Bloomsbury Genetic Therapies Ltd., Azafaros A.G, Cyclo Therapeutics Inc., Mandos Health, CarelonRx, Rainbow Children's Hospital, and Vtesse Inc.

North America was the largest region in the niemann-pick disease (NPD) market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in niemann-pick disease (NPD report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the niemann-pick disease (NPD) market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The niemann-pick disease (NPD) market consists of revenues earned by entities by providing services such as diagnostic testing, genetic counseling, patient support programs, clinical trial management, drug development, and regulatory consulting. The market value includes the value of related goods sold by the service provider or included within the service offering. The niemann-pick disease (NPD) market also includes sales of gene therapy products, enzyme replacement tools, diagnostic kits, biomarker testing tools, and supportive care products. 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.

Niemann-Pick Disease (NPD) 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 (npd) 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.

Reasons to Purchase

Where is the largest and fastest growing market for niemann-pick disease (npd) ? 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 (npd) 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.

Scope

Table of Contents

1. Executive Summary

2. Niemann-Pick Disease (NPD) Market Characteristics

3. Niemann-Pick Disease (NPD) Market Trends And Strategies

4. Niemann-Pick Disease (NPD) Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market

5. Global Niemann-Pick Disease (NPD) Growth Analysis And Strategic Analysis Framework

6. Niemann-Pick Disease (NPD) Market Segmentation

7. Niemann-Pick Disease (NPD) Market Regional And Country Analysis

8. Asia-Pacific Niemann-Pick Disease (NPD) Market

9. China Niemann-Pick Disease (NPD) Market

10. India Niemann-Pick Disease (NPD) Market

11. Japan Niemann-Pick Disease (NPD) Market

12. Australia Niemann-Pick Disease (NPD) Market

13. Indonesia Niemann-Pick Disease (NPD) Market

14. South Korea Niemann-Pick Disease (NPD) Market

15. Western Europe Niemann-Pick Disease (NPD) Market

16. UK Niemann-Pick Disease (NPD) Market

17. Germany Niemann-Pick Disease (NPD) Market

18. France Niemann-Pick Disease (NPD) Market

19. Italy Niemann-Pick Disease (NPD) Market

20. Spain Niemann-Pick Disease (NPD) Market

21. Eastern Europe Niemann-Pick Disease (NPD) Market

22. Russia Niemann-Pick Disease (NPD) Market

23. North America Niemann-Pick Disease (NPD) Market

24. USA Niemann-Pick Disease (NPD) Market

25. Canada Niemann-Pick Disease (NPD) Market

26. South America Niemann-Pick Disease (NPD) Market

27. Brazil Niemann-Pick Disease (NPD) Market

28. Middle East Niemann-Pick Disease (NPD) Market

29. Africa Niemann-Pick Disease (NPD) Market

30. Niemann-Pick Disease (NPD) Market Competitive Landscape And Company Profiles

31. Niemann-Pick Disease (NPD) Market Other Major And Innovative Companies

32. Global Niemann-Pick Disease (NPD) Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Niemann-Pick Disease (NPD) Market

34. Recent Developments In The Niemann-Pick Disease (NPD) Market

35. Niemann-Pick Disease (NPD) Market High Potential Countries, Segments and Strategies

36. Appendix

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