The NPC market remains constrained by the scarcity of approved therapies and a substantial unmet medical need. Nonetheless, upcoming treatments, including Cyclo Therapeutics’ Trappsol Cyclo, Azafaros’ oral small-molecule nizubaglustat (AZ-3102), and the early-stage candidate Adrabetadex (VTS-270), combined with increasing disease awareness, are anticipated to support moderate market growth and broaden future treatment options.
DelveInsight’s Niemann-Pick Disease Type C Market Insights report includes a comprehensive understanding of current treatment practices, emerging Niemann-Pick disease type C drugs, market share of individual therapies, and current and forecasted Niemann-Pick disease type C market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan).
Niemann-Pick Disease Type C Market Summary
- The Niemann-Pick disease type C market across major regions was valued at about USD 60 million in 2024. The United States represented the largest share, making up nearly 60% of the total 7MM market, outpacing the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
- In 2024, the 7MM recorded roughly 1,000 diagnosed prevalent cases of Niemann-Pick disease type C, underscoring its classification as an ultra-rare disorder.
- Leading companies such as Cyclo Therapeutics, Azafaros, and Mandos are advancing innovative treatment candidates.
- Prominent therapies under clinical development include Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin), Nizubaglustat (AZ-3102), and Adrabetadex (VTS-270). These emerging treatments are expected to enter the market over the forecast period and significantly reshape the therapeutic landscape.
Discover which Niemann-Pick disease type C medications are expected to grab the market share @ Niemann-Pick Disease Type C Market Report
What is Niemann-Pick Disease Type C?
Niemann-Pick disease type C is a chronic, progressive lysosomal lipid storage disorder that leads to irreversible and debilitating complications involving visceral organs, the nervous system, and cognitive function. It results from defective intracellular cholesterol transport, leading to the accumulation of unesterified cholesterol and glycosphingolipids within neurovisceral tissues. The majority of cases stem from mutations in the NPC1 gene, with a smaller portion linked to NPC2 mutations, both disrupting normal cholesterol movement in late endosomes and lysosomes. The disease also triggers a secondary reduction in ASM activity, establishing a biological link with other Niemann-Pick subtypes.
Niemann-Pick Disease Type C Epidemiology Segmentation
The NPC epidemiology section provides insights into the historical and current Niemann-Pick disease type C patient pool and forecasted trends for the leading markets. In 2024, the highest burden of NPC in the US was observed in the juvenile age group (6 to <15 years) with approximately 120 cases, followed by around 110 cases in the adolescent/adult-onset group (=15 years). The NPC market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into:
- Prevalent Cases of NPC
- Total Diagnosed Prevalent Cases of NPC
- Age-specific Cases of NPC
- Gender-specific Cases of NPC
- Mutation-specific Cases of NPC
- Total Treated Cases of NPC
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Niemann-Pick Disease Type C Market Analysis
Current care for Niemann–Pick disease type C remains symptomatic, mainly individualized. Swallowing difficulties require vigilant monitoring because of the risk of aspiration. Early management strategies include modifying food textures, such as softening solids and thickening liquids, and engaging speech therapists to improve swallowing function. In more advanced cases, a gastrostomy tube may be required to maintain adequate nutrition. This procedure places a thin tube directly into the stomach through a small incision in the abdomen, allowing direct delivery of food and medication.
Two therapies, AQNEURSA (levacetylleucine) and MIPLYFFA (arimoclomol), are currently available to help manage neurological manifestations of NPC, each with defined eligibility requirements. MIPLYFFA became the first FDA-approved oral therapy for this ultra-rare disorder in September 2024 and is approved for use with miglustat in patients aged two years and older, marking a key step forward in symptomatic care. Zevra Therapeutics submitted an MAA to the European Medicines Agency in July 2025 for arimoclomol, intended to address the underlying lipid buildup characteristic of NPC.
However, no existing treatment has demonstrated the ability to modify the disease’s fundamental progression. Available options primarily alleviate symptoms rather than offering a cure or altering the course of NPC. Miglustat remains a key therapy, approved in Europe (2009) and Japan (2012), for reducing symptoms and slowing neurological decline, and it is frequently used off-label in the U.S. Its continued use underscores the substantial unmet need for true disease-modifying therapies capable of altering long-term outcomes in NPC.
Learn more about the NPC treatment options @ Niemann-Pick Disease Type C Treatment Market
Key Factors Driving the Growth of the Niemann-Pick Disease Type C Market
Increasing Prevalence of NPC
DelveInsight’s analysis estimates that nearly 1,000 diagnosed prevalent cases of Niemann-Pick Disease Type C (NPC) were recorded across the 7MM in 2024. This number is expected to rise steadily throughout the forecast period (2025–2034).
Progress in Genetic Research and Gene Therapy
Advances in understanding NPC-related genetic mutations, particularly involving the NPC1 and NPC2 genes, are driving the development of more precise treatment strategies. Innovative gene therapy approaches, including gene replacement and CRISPR-Cas9–based editing, are emerging as potential curative solutions for this rare condition.
Introduction of New NPC Treatment Candidates
The NPC treatment pipeline is expanding with candidates such as Cyclo Therapeutics’ Trappsol Cyclo, Azafaros’ oral small molecule nizubaglustat (AZ-3102), and Mandos’ early-stage therapy Adrabetadex (VTS-270). These candidates represent multiple therapeutic strategies targeting different aspects of the disease.
Growing Focus on Cyclodextrin-Based Treatments
Cyclodextrin-based therapies like Trappsol Cyclo work by clearing accumulated cholesterol from cells and restoring normal cell function. Their ability to address the core lipid-storage defect in NPC is contributing to increased interest and market momentum for these treatments.
Niemann-Pick Disease Type C Competitive Landscape
Pipeline candidates such as Cyclo Therapeutics’ Trappsol Cyclo, Azafaros’ Nizubaglustat, and Mandos’ Adrabetadex (VTS-270), among others, are expected to contribute significantly to the growth of the NPC market.
Trappsol Cyclo, developed by Cyclo Therapeutics, is a specialized hydroxypropyl β-cyclodextrin formulation that has demonstrated promising outcomes across multiple clinical studies aimed at correcting cholesterol transport. Acting as a functional replacement for the impaired NPC1 protein, its cyclic structure helps extract accumulated cholesterol from lysosomes, thereby restoring normal processing and clearance within cells. The therapy is currently being assessed in clinical trials as a potential treatment for Niemann-Pick disease type C (NPC), a rare, progressive, and life-threatening genetic disorder.
Azafaros’ Nizubaglustat (formerly AZ-3102) is an orally delivered, brain-penetrant azasugar with a unique dual mechanism of action, designed to address rare lysosomal storage disorders with neurological symptoms, including GM1 and GM2 gangliosidoses and NPC. It works by modulating lipid metabolism to decrease the buildup of toxic lipids in lysosomes. By targeting this core cellular defect, Nizubaglustat may serve as a disease-modifying therapy for NPC, offering an at-home treatment option that can enhance patient convenience and quality of life.
The expected introduction of these advanced NPC therapies is set to redefine the NPC treatment landscape in the coming years. As these innovative treatments progress through development and secure regulatory approvals, they are anticipated to establish new standards of care while opening avenues for medical innovation and market expansion.
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Recent Developments in the NPC Market
- In July 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorisation for AQNEURSA to treat neurological manifestations of NPC.
- In July 2025, Azafaros announced the initiation of two global Phase III studies with Nizubaglustat in NPC and GM1/GM2 gangliosidoses, respectively.
- In March 2025, Rafael Holdings completed a merger with Cyclo Therapeutics following shareholder approvals.
- In February 2025, Cyclo Therapeutics presented preliminary data from the Phase III TransportNPC open-label sub-study at the 21st Annual WORLDSymposium 2025, showing that among NPC1 patients under 3 years old, 87% demonstrated stabilization or improvement on the CGI-C scale at 24 weeks and 86% at 48 weeks.
| Niemann-Pick Disease Type C Market Report Metrics | Details |
| Study Period | 2020–2034 |
| Niemann-Pick Disease Type C Market Report Coverage | 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] |
| Niemann-Pick Disease Type C Market CAGR | 14.8% |
| Niemann-Pick Disease Type C Market Size | USD 60 Million |
| Key Niemann-Pick Disease Type C Companies | Cyclo Therapeutics, Azafaros, Mandos, IntraBio, Zevra Therapeutics, and others |
| Key Niemann-Pick Disease Type C Therapies | Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin), Nizubaglustat (AZ-3102), Adrabetadex (VTS-270), AQNEURSA, MIPLYFFA, and others |
Scope of the Niemann-Pick Disease Type C Market Report
- Niemann-Pick Disease Type C Therapeutic Assessment: Niemann-Pick Disease Type C current marketed and emerging therapies
- Niemann-Pick Disease Type C Market Dynamics: Conjoint Analysis of Emerging Niemann-Pick Disease Type C Drugs
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Niemann-Pick Disease Type C Market Unmet Needs, KOL’s views, Analyst’s views, Niemann-Pick Disease Type C Market Access and Reimbursement
Discover more about NPC drugs in development @ Niemann-Pick Disease Type C Clinical Trials
Table of Contents
| 1 | Niemann-Pick Disease Type C Market Key Insights |
| 2 | Niemann-Pick Disease Type C Market Report Introduction |
| 3 | NPC Market Overview at a Glance |
| 3.1 | Market Share (%) Distribution of NPC by Therapies in 2024 in the 7MM |
| 3.2 | Market Share (%) Distribution of NPC by Therapies in 2034 in the 7MM |
| 4 | Epidemiology and Market Methodology |
| 5 | Executive Summary |
| 6 | Key Events |
| 7 | Disease Background and Overview |
| 7.1 | Introduction |
| 7.2 | Classification of NPD |
| 7.3 | Niemann-Pick Disease Type C Causes |
| 7.4 | Niemann-Pick Disease Type C Inheritance Patterns |
| 7.5 | Niemann-Pick Disease Type C Clinical Manifestations |
| 7.6 | Niemann-Pick Disease Type C Pathophysiology |
| 7.7 | Niemann-Pick Disease Type C Diagnosis |
| 8 | Niemann-Pick Disease Type C Treatment and Management |
| 9 | Epidemiology and Patient Population |
| 9.1 | Key Findings |
| 9.2 | Assumptions and Rationale |
| 9.3 | Total Diagnosed Prevalent Cases of NPC in the 7MM |
| 9.4 | The United States |
| 9.4.1 | Prevalent Cases of NPC in the United States |
| 9.4.2 | Total Diagnosed Prevalent Cases of NPC in the United States |
| 9.4.3 | Age-specific Cases of NPC in the United States |
| 9.4.4 | Gender-specific Cases of NPC in the United States |
| 9.4.5 | Mutation-specific Cases of NPC in the United States |
| 9.4.6 | Total Treated Cases of NPC in the United States |
| 9.5 | EU4 and the UK |
| 9.6 | Japan |
| 10 | Niemann-Pick Disease Type C Patient Journey |
| 11 | Marketed Niemann-Pick Disease Type C Therapies |
| 11.1 | Key Cross Competition |
| 11.2 | AQNEURSA (levacetylleucine): IntraBio |
| 11.2.1 | Product Description |
| 11.2.2 | Regulatory Milestones |
| 11.2.3 | Other Developmental Activities |
| 11.2.4 | Safety and efficacy |
| 11.2.5 | Analyst Views |
| 11.3 | MIPLYFFA (arimoclomol): Zevra Therapeutics (Formerly Known as KemPharm) |
| 12 | Emerging Niemann-Pick Disease Type C Therapies |
| 12.1 | Key Cross Competition |
| 12.2 | Trappsol Cyclo (Hydroxypropyl β-cyclodextrin): Cyclo Therapeutics |
| 12.2.1 | Product Description |
| 12.2.2 | Other Developmental Activities |
| 12.2.3 | Ongoing Clinical Development activity |
| 12.2.4 | Safety and Efficacy |
| 12.2.5 | Analyst Views |
| 12.3 | Nizubaglustat: Azafaros |
| 13 | NPC Market – Seven Major Market Analysis |
| 13.1 | Key Findings |
| 13.2 | Niemann-Pick Disease Type C Market Outlook |
| 13.3 | Conjoint Analysis |
| 13.4 | Key Niemann-Pick Disease Type C Market Forecast Assumptions |
| 13.5 | Total Market Size of NPC in the 7MM |
| 13.6 | Market Size of NPC by Therapies in the 7MM |
| 13.7 | Niemann-Pick Disease Type C Market Size in the United States |
| 13.7.1 | Total Market Size of NPC in the United States |
| 13.7.2 | Market Size of NPC by Therapies in the United States |
| 13.8 | Niemann-Pick Disease Type C Market Size in EU4 and the UK |
| 13.9 | Niemann-Pick Disease Type C Market Size in Japan |
| 14 | KOL Views on Niemann-Pick Disease Type C |
| 15 | Niemann-Pick Disease Type C Market SWOT Analysis |
| 16 | Niemann-Pick Disease Type C Market Unmet Needs |
| 17 | Niemann-Pick Disease Type C Market Access and Reimbursement |
| 17.1 | United States |
| 17.2 | EU4 and the UK |
| 17.3 | Japan |
| 17.4 | Reimbursement Scenario in NPC |
| 18 | Bibliography |
| 19 | Niemann-Pick Disease Type C Market Report Methodology |
Related Reports
Niemann-Pick Disease Type C Clinical Trial Analysis
Niemann-Pick Disease Type C Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key NPC companies, including Cyclo Therapeutics, Inc., Azafaros A.G., Cyclarity Therapeutics, Cyclolab, among others.
Niemann Pick Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Niemann Pick disease companies, including Azafaros A.G., Cyclo Therapeutics, Inc., ZevraDenmark, Genzyme, Mandos Health, Zevra Therapeutics, IntraBio, among others.
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