Niemann-Pick Disease Type C (NPC) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Mandos, Orphazyme, IntraBio, SOM Biotech

Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, about 13+ key pharma and biotech companies are working on 13+ pipeline drugs in the Niemann-Pick Disease Type C therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action (MOA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Niemann-Pick Disease Type C (NPC) Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Niemann-Pick Disease Type C Market

The Niemann-Pick Disease Type C Pipeline report embraces in-depth commercial, regulatory, and Niemann-Pick Disease Type C clinical trial assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the emerging Niemann-Pick Disease Type C drugs, including the mechanism of action, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Niemann-Pick Disease Type C Pipeline Analysis

The report provides insights into: 

The report provides detailed insights into the emerging therapies for Niemann-Pick Disease Type C treatment and the aggregate therapies developed by major pharma companies.

It accesses the different Niemann-Pick Disease Type C therapies segmented into early-stage, mid-stage, and late-stage of clinical development.

It outlines the major Niemann-Pick Disease Type C companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

The report evaluates the Niemann-Pick Disease Type C drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Niemann-Pick Disease Type C therapeutic market.

Niemann-Pick Disease Type C Therapeutics Landscape

Treatment of NPC requires the coordinated efforts of a team of specialists. Pediatricians, neurologists, ophthalmologists, pulmonologists, gastroenterologists, and other healthcare professionals may need to systematically and comprehensively plan an affected child’s treatment. Psychosocial support for the entire family is essential as well. Genetic counseling benefits affected individuals and their families. Current treatment is directed toward the specific symptoms apparent in each individual. 

There is no cure for NPC, although patients benefit from palliative treatments. Occupational therapy can be used to help with posture, speech, and movement. Physical therapy is an important part of treatment to help maintain mobility as long as possible.

The only approved therapy in Europe and Japan is ZAVESCA/BRAZAVES [miglustat], a substrate reduction therap developed by Actelion Pharmaceuticals. It has been denied approval for the treatment of NPC, by the US Food and Drug Administration (FDA) on the grounds of insufficient data. The US FDA requested for more data, which would take years to get, and almost impossible to satisfy the high standards of the FDA, mainly restrained by the limited or lack of patient population that could be recruited in the trials.

There are approx. 13+ key companies are developing therapies for Niemann-Pick Disease Type C. Currently, Orphazyme is leading the therapeutics market with its Niemann-Pick Disease Type C drug candidates in the most advanced stage of clinical development.

Niemann-Pick Disease Type C Companies Actively Working in the Therapeutic Market Include:

Mandos LLC

Cyclo Therapeutics


IntraBio Inc


Evox Therapeutics

SOM Biotech

Sarepta Therapeutics



Amicus Therapeutics

And Many Others

Emerging and Marketed Niemann-Pick Disease Type C Drugs Covered in the Report Include:

Arimoclomol: Orphazyme

Adrabetadex: Mandos, LLC

VTS-270: Mallinckrodt

Arimoclomol: Orphazyme

Trappsol Cyclo: CTD Holdings, Inc.

And Many Others

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Niemann-Pick Disease Type C Companies Working in the Market:

The report covers the emerging products under different phases of clinical development like –

Late-stage products (Phase III)

Mid-stage products (Phase II)

Early-stage product (Phase I) 

Pre-clinical and Discovery stage candidates

Discontinued and inactive candidates

Route of Administration

Niemann-Pick Disease Type C pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Niemann-Pick Disease Type C Products have been categorized under various ROAs such as






Molecule Type

Niemann-Pick Disease Type C Products have been categorized under various Molecule types such as

Monoclonal Antibody



Small molecule

Gene therapy

Learn How the Niemann-Pick Disease Type C Treatment Outlook will Evolve with the Ongoing Clinical & Commercial Activities in the Therapeutic Market @

Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Niemann-Pick Disease Type C Treatment Patterns

4. Niemann-Pick Disease Type C – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Niemann-Pick Disease Type C Late Stage Products (Phase-III)

7. Niemann-Pick Disease Type C Mid-Stage Products (Phase-II)

8. Niemann-Pick Disease Type C Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Niemann-Pick Disease Type C Discontinued Products

13. Niemann-Pick Disease Type C Product Profiles

14. Major Niemann-Pick Disease Type C Companies in the Market

15. Key Products in the Niemann-Pick Disease Type C Therapeutics Segment

16. Dormant and Discontinued Products

17. Niemann-Pick Disease Type C Unmet Needs

18. Niemann-Pick Disease Type C Future Perspectives

19. Niemann-Pick Disease Type C Analyst Review  

20. Appendix

21. Report Methodology

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download Sample PDF to Explore the Key Offerings of the Report @



About DelveInsight

DelveInsight is a leading 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.

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