Niemann Pick C Disease Treatment Market Size in 7MM is expected to grow at a decent CAGR by 2034, estimates DelveInsight | Mandos Health, Cycle Therapeutics, Intra Bio, Zevra Therapeutics

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

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Key Takeaways from the Niemann-Pick Disease Type C Market Report
• In March 2025, Actelion announced a phase IV confirmatory study.The study is conducted in Chinese subjects aged 4 years and older with Niemann Pick Type C disease (NPC). Approximately 19 subjects will be enrolled in this study. The study will be conducted at 2 sites in China. Patients will be treated with miglustat for 12 months, efficacy and safety outcomes will be measured.
• In March 2025, Azafaros A.G. organized a phase 2 study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.
• The United States accounted for the highest number of Niemann-Pick disease type C Prevalent Cases in the 7MM in 2023.
• Among gene-specific cases, Niemann-Pick disease type C1 accounted for most of the cases.
• Among the gender-specific cases, Niemann-Pick disease type C1 affects males and females equally.
• The leading Niemann-Pick Disease Type C Companies such as Mandos Health, Cycle Therapeutics, Intra Bio, Zevra Therapeutics, and others
• Promising Niemann-Pick Disease Type C Therapies such as N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, AZ-3102, and others.

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Niemann-Pick Disease Type C Epidemiology Segmentation in the 7MM
• Total Diagnosed Prevalent cases
• Gender- specific Diagnosed prevalent Cases
• Distribution of diagnosed Prevalent Cases of Niemann-Pick disease Type C by age

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Niemann-Pick Disease Type C Marketed Drugs
• ZAVESCA/BRAZAVES (miglustat): Actelion Pharmaceuticals
ZAVESCA/BRAZAVES is a small iminosugar molecule that reversibly inhibits glycosphingolipid synthesis and blocks the synthesis of glycosphingolipids, one of the substances that accumulate in the brain of individuals with Niemann-Pick disease type C and can slow the progression of neurological symptoms associated with Niemann-Pick disease type C. It was approved by the EMA in 2009. It has been denied approval for the treatment of Niemann-Pick disease type C, by the US FDA on the grounds of insufficient data. However, it is being prescribed to the Niemann-Pick disease type C patients as an off-label therapy.

Emerging Niemann-Pick Disease Type C Drugs
• Trappsol Cyclo: Cyclo Therapeutics
Trappsol by Cyclo Therapeutics is a proprietary formulation of hydroxypropyl betacyclodextrin and in multiple clinical studies has shown encouraging results to effectively manage the transportation of cholesterol. Taking the place of the defective Niemann-Pick disease type C protein, Trappsol Cyclo, with its cyclic structure, captures cholesterol within the cell and transports it out of the cell. The drug is currently being evaluated in Phase III TransportNPC pivotal trial in pediatric and adult patients with Niemann-Pick disease type C1. It has received an Orphan drug designation by the US FDA and the EMA. It has also received Rare Pediatric Disease Designation, and Fast-track designation by the US FDA for the treatment of Niemann-Pick disease type C.

• IB10001: IntraBio
IntraBio's lead drug series, IB1000s, is a set of orally administered, modified amino acids, N-acetyl-leucine, categorized by a well-established safety and tolerability profile. IB1001 (N-acetyl-l-leucine) is being developed for Niemann-Pick Disease Type C besides other orphan indications like Tay-Sachs and Sandhoff Disease, and inherited Cerebellar Ataxias. IB1000s have an effect on the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels and have been observed to have symptomatic and neuroprotective properties and disease-modifying potential. The US FDA has accepted IntraBio's NDA and granted priority review for IB1001 for the treatment of Niemann-Pick disease type C. The application has been granted Priority Review and a PDUFA target action date of September 24, 2024.

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Niemann-Pick Disease Type C Market Outlook
Various supportive therapies are available for managing symptoms linked with Niemann-Pick disease type C, including antiepileptics for seizures, tricyclic antidepressants, CNS stimulants for cataplexy, and anticholinergics for dystonia and tremor. However, these therapies contribute minimally to market revenue for Niemann-Pick disease type C1 treatment. ZAVESCA, prescribed to approximately half of Niemann-Pick disease type C patients, offers hope amidst the lack of disease-targeting therapies.

Scope of the Niemann-Pick Disease Type C Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Niemann-Pick Disease Type C Companies- Mandos Health, Cycle Therapeutics, Intra Bio, Zevra Therapeutics, and others
• Niemann-Pick Disease Type C Therapies- N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, AZ-3102, and others.
• Niemann-Pick Disease Type C Therapeutic Assessment: Niemann-Pick Disease Type C Current Marketed and Niemann-Pick Disease Type C Emerging Therapies
• Niemann-Pick Disease Type C Market Dynamics: Niemann-Pick Disease Type C market drivers and Niemann-Pick Disease Type C market barriers
• 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 Content
1. Key Insights
2. Report Introduction
3. Executive Summary
4. Niemann-Pick Disease Type C Market Overview at a Glance
5. Key Events
6. Epidemiology and Market Forecast Methodology
7. Disease Background and Overview
8. Niemann-Pick Disease Type C Epidemiology and Patient Population
9. Japan
10. Niemann-Pick Disease Type C Patient Journey
11. Niemann-Pick Disease Type C Marketed Drugs
12. Emerging Therapies
13. Niemann-Pick Disease Type C: Seven Major Market Analysis
14. Niemann-Pick Disease Type C Unmet Needs
15. SWOT Analysis
16. KOL Views
17. Reimbursement and Market Access
18. Appendix
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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