Niemann-Pick Disease Type C Market to Expand Significantly by 2034, States DelveInsight Report | Zevra Therapeutics, Azafaros A.G, IntraBio, Cyclo Therapeutics, Azafaros A.G

The Key Niemann-Pick Disease Type C Companies in the market include - Zevra Therapeutics, Azafaros A.G, IntraBio, Cyclo Therapeutics, Azafaros A.G., and others.

DelveInsight's "Niemann-Pick Disease Type C Market Insights, Epidemiology, and Market Forecast-2034 report offers an in-depth understanding of the Niemann-Pick Disease Type C, historical and forecasted epidemiology as well as the Niemann-Pick Disease Type C market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Niemann-Pick Disease Type C Market Report:

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The Niemann-Pick Disease Type C market size was valued ~USD 34 million in 2022 and is anticipated to grow with a significant CAGR during the study period (2020-2034)

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In April 2025, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a commercial-stage company dedicated to developing treatments for rare diseases, today announced the publication of the study titled "Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-Pick type C disease" in the journal Molecular Genetics and Metabolism.

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In February 2025, A Phase III trial investigating a treatment for the rare genetic condition Niemann-Pick disease reported that 87% of participants-each under the age of three-experienced either stabilization or improvement. US-based Cyclo Therapeutics shared early findings from its TransportNPC study (NCT04860960), which is evaluating a hydroxypropyl beta cyclodextrin formulation known as Trappsol Cyclo in eight patients.

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In January 2025, Azafaros B.V. announced that its lead candidate, nizubaglustat, has received orphan drug designation from regulatory agencies in both the United States and the European Union for the treatment of GM1 gangliosidosis. Furthermore, the company's Clinical Trial Application (CTA) for two global Phase 3 trials evaluating the drug's safety and efficacy in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC) has been approved by several European countries. Azafaros plans to begin these international studies in the second quarter of 2025.

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The market size for Niemann-Pick Disease Type C (NPC) in the US was USD 9 million in 2022. According to DelveInsight's analysis, the market is expected to grow due to the rising prevalence of the disease.

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DelveInsight's analysis shows that the market size for Niemann-Pick Disease Type C in the EU-4 and the UK was around USD 23 million in 2022. This market is projected to grow during the forecast period from 2023 to 2034.

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According to DelveInsight's analysis, there were 787 diagnosed prevalent cases of Niemann-Pick Disease Type C in the 7MM in 2022, with a projected increase in cases expected during the forecast period of 2023 to 2034.

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In 2022, the EU4 countries and the UK accounted for 328 cases of Niemann-Pick Disease Type C. Among these countries, Germany had the highest number of diagnosed prevalent cases, with 84 cases, representing 26% of the total in these four European nations. This number is expected to rise during the study period from 2020 to 2034.

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In 2022, Spain had the lowest number of diagnosed prevalent cases of Niemann-Pick Disease Type C among the EU4 countries.

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The diagnosed prevalent cases of Niemann-Pick Disease Type C in the 7MM differed by gender, with females having higher numbers than males. According to DelveInsight's analysts, there were 433 diagnosed prevalent cases in females and 354 in males in 2022. These numbers are expected to increase during the forecast period from 2023 to 2034.

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According to DelveInsight's analysts, Japan had 37 diagnosed prevalent cases of the juvenile type (ages 6 to under 15) of Niemann-Pick Disease Type C in 2022.

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In 2022, the market size for Niemann-Pick Disease Type C (NPC) in Japan was USD 2 million, representing 6% of the total market size across the 7MM. It is anticipated to increase by 2034.

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Key Niemann-Pick Disease Type C Companies: Zevra Therapeutics, Azafaros A.G, IntraBio, Cyclo Therapeutics, Azafaros A.G., and others

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Key Niemann-Pick Disease Type C Therapies: Arimoclomol, AZ-3102, IB1001, N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, AZ-3102, and others

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The Niemann-Pick Disease Type C epidemiology based on gender analyzed that Niemann Pick Disease Type C is more prevalent in females than in males

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The Niemann-Pick Disease Type C market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Niemann-Pick Disease Type C pipeline products will significantly revolutionize the Niemann-Pick Disease Type C market dynamics.

Niemann-Pick Disease Type C Overview

Niemann-Pick Disease Type C (NPC) is a rare, inherited, neurodegenerative disorder characterized by the abnormal accumulation of lipids (fats) in various tissues of the body, particularly in the brain, liver, and spleen. This accumulation is due to mutations in the NPC1 or NPC2 genes, which are responsible for regulating the transport of cholesterol and other lipids within cells. These mutations disrupt the normal processing and movement of lipids, leading to their buildup and subsequent cellular dysfunction and damage.

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Niemann-Pick Disease Type C Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Niemann-Pick Disease Type C Epidemiology Segmentation:

The Niemann-Pick Disease Type C market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Niemann-Pick Disease Type C

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Prevalent Cases of Niemann-Pick Disease Type C by severity

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Gender-specific Prevalence of Niemann-Pick Disease Type C

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Diagnosed Cases of Episodic and Chronic Niemann-Pick Disease Type C

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Niemann-Pick Disease Type C Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Niemann-Pick Disease Type C market or expected to get launched during the study period. The analysis covers Niemann-Pick Disease Type C market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Niemann-Pick Disease Type C Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Niemann-Pick Disease Type C Therapies and Key Companies

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Arimoclomol: Zevra Therapeutics

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AZ-3102: Azafaros A.G

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IB1001: IntraBio

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N-Acetyl-L-Leucine: IntraBio Inc

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arimoclomol: ZevraDenmark

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Hydroxypropyl-beta-cyclodextrin: Cyclo Therapeutics

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AZ-3102: Azafaros A.G.

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Niemann-Pick Disease Type C Market Drivers

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Advances in genetic testing and diagnostic technologies

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Increasing awareness and improved disease identification

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Rising prevalence of NPC globally

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Growing research and development initiatives for novel therapies

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Supportive regulatory environment facilitating drug approvals

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Expansion of healthcare infrastructure and accessibility

Niemann-Pick Disease Type C Market Barriers

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Limited understanding of disease mechanisms and variability

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High costs associated with treatment and care

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Challenges in early diagnosis and screening

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Limited therapeutic options and efficacy of current treatments

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Regulatory complexities in drug development and approval

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Accessibility issues in healthcare systems, especially in developing regions

Scope of the Niemann-Pick Disease Type C Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Niemann-Pick Disease Type C Companies: Zevra Therapeutics, Azafaros A.G, IntraBio, Cyclo Therapeutics, Azafaros A.G., and others

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Key Niemann-Pick Disease Type C Therapies: Arimoclomol, AZ-3102, IB1001, N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, AZ-3102, and others

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Niemann-Pick Disease Type C Therapeutic Assessment: Niemann-Pick Disease Type C current marketed and Niemann-Pick Disease Type C emerging therapies

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Niemann-Pick Disease Type C Market Dynamics: Niemann-Pick Disease Type C market drivers and Niemann-Pick Disease Type C market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Niemann-Pick Disease Type C Unmet Needs, KOL's views, Analyst's views, Niemann-Pick Disease Type C Market Access and Reimbursement

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Table of Contents

1. Niemann-Pick Disease Type C Market Report Introduction

2. Executive Summary for Niemann-Pick Disease Type C

3. SWOT analysis of Niemann-Pick Disease Type C

4. Niemann-Pick Disease Type C Patient Share (%) Overview at a Glance

5. Niemann-Pick Disease Type C Market Overview at a Glance

6. Niemann-Pick Disease Type C Disease Background and Overview

7. Niemann-Pick Disease Type C Epidemiology and Patient Population

8. Country-Specific Patient Population of Niemann-Pick Disease Type C

9. Niemann-Pick Disease Type C Current Treatment and Medical Practices

10. Niemann-Pick Disease Type C Unmet Needs

11. Niemann-Pick Disease Type C Emerging Therapies

12. Niemann-Pick Disease Type C Market Outlook

13. Country-Wise Niemann-Pick Disease Type C Market Analysis (2020-2034)

14. Niemann-Pick Disease Type C Market Access and Reimbursement of Therapies

15. Niemann-Pick Disease Type C Market Drivers

16. Niemann-Pick Disease Type C Market Barriers

17. Niemann-Pick Disease Type C Appendix

18. Niemann-Pick Disease Type C Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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