Niemann Pick Disease Marketed and Emerging Pipeline Drugs Assessment | Clinical Trials, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, Competitive Landscape, and Key Companies

Niemann Pick Disease Marketed and Emerging Pipeline Drugs Assessment | Clinical Trials, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, Competitive Landscape, and Key Companies
Delveinsight Business Research LLP
As per DelveInsight’s assessment, about 20+ key pharma and biotech companies are working on 20+ pipeline drugs in the Niemann Pick Disease 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 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 Market. 

The Niemann Pick Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, 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 Pipeline Analysis

Niemann Pick Disease Pipeline Analysis

The report provides insights into: 

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

  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the 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 market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.  

Analysis of Emerging Therapies by Phases

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 & Inactive candidates


Route of Administration

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

  • Intra-articular

  • Intraocular

  • Intrathecal

  • Intravenous

  • Ophthalmic

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Transdermal


Molecule Type

The emerging products have been categorized under various Molecule types, such as

  • Oligonucleotide

  • Peptide

  • Small molecule


Learn How the Ongoing Clinical & Commercial Activities will Affect the Niemann Pick Disease Therapeutic Segment @

Niemann Pick Disease Therapeutics Landscape

There are approx. 20+ key companies developing therapies for Niemann-Pick-Disease. Currently, Orphazyme is leading the therapeutics market with its Niemann-Pick-Disease drug candidates in the most advanced stage of clinical development.

The Leading Companies in the Niemann Pick Disease Therapeutics Market Include:

  • Orphazyme

  • Cyclo Therapeutics

  • Mandos LLC

  • IntraBio

  • E-scape Bio

  • Scenic Biotech

  • Synaptogenix

  • Evox Therapeutics

  • StrideBio

  • SOM Biotech


  • Oraxion Therapeutics

  • Polaryx Therapeutics

And Many More

Niemann-Pick-Disease Drugs Covered in the Report Include

  • Arimoclomol: Orphazyme

  • Trappsol Cyclo: Cyclo Therapeutics

  • VTS-270: Mandos LLC

  • ESB1609: E-scape Bio

And Many More

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @

Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Niemann Pick Disease Current Treatment Patterns

4. Niemann Pick Disease – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

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

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

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

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Niemann Pick Disease Discontinued Products

13. Niemann Pick Disease Product Profiles

14. Key Companies in the Niemann Pick Disease Market

15. Key Products in the Niemann Pick Disease Therapeutics Segment

16. Dormant and Discontinued Products

17. Niemann Pick Disease Unmet Needs

18. Niemann Pick Disease Future Perspectives

19. Niemann Pick Disease 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 @


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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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