Niemann - Pick Disease - A Pipeline Analysis report
- Published: Jul 2018
- Pages: 68
- SKU: IRTNTR23335
This pipeline analysis report provides detailed insights into the clinical trials landscape of the drug development for Niemann-pick disease including molecules at pre-clinical and discovery stage. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations such as therapy, route of administration, and target.
Overview of the drug development for Niemann-pick disease
Niemann-Pick disease is a rare, inherited disease that affects the body’s ability to metabolize cholesterol and lipids within cells. Niemann-Pick disease can affect the brain, nerves, liver, spleen, bone marrow, and lungs. People with Niemann-Pick disease experience symptoms related to the progressive function loss of the nerves, the brain, and other organs. Niemann-Pick disease can occur at any age, but it mainly affects children. The disease has no known cure and is sometimes fatal. According to the US National Library of Medicines, Neimann-Pick disease type A and B are estimated to affect 1 in 250,000 individuals. It occurs more frequently among individuals of Ashkenazi, an Eastern and Central European Jewish descent, than in the general population. The disease occurs mostly among the individuals of the French-Acadian descent in Nova Scotia. In Nova Scotia, a population of affected French-Acadians was previously designated as having Niemann-Pick disease type D. Consequently, the rising incidences of the prevalence of this disease are likely to boost the drug development for Niemann-pick disease.
According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for Niemann-Pick disease type A. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.
This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of Niemann-Pick disease. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are –
- CTD Holdings
Therapeutic assessment of the drug development for Niemann-pick disease by route of administration
The oral route of administration (ROA) involves the application of the drug directly through the mouth cavity, which will have a more direct effect on the target cells.
Therapeutic assessment of the drug development for Niemann-pick disease by therapy
According to this pipeline analysis report, all the molecules that are currently in the drug development for Niemann-pick disease are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.
Key questions answered in the report include
- What are the drug molecules in the various development stages for Niemann-pick disease?
- What are the companies that are currently involved in the development of drug molecules for Niemann-pick disease?
- Insight into discontinued/inactive molecules with appropriate reasoning?
- What are the major regulatory authorities approving drugs in various regions?
- Detailed profiling of each active molecule
Technavio also offers customization on reports based on specific client requirement.
PART 01: EXECUTIVE SUMMARY
PART 02: SCOPE OF THE REPORT
PART 03: RESEARCH METHODOLOGY
PART 04: INTRODUCTION
PART 05: MAJOR REGULATORY AUTHORITIES
PART 06: PIPELINE LANDSCAPE
PART 07: COMPARATIVE ANALYSIS
- Discovery stage molecules
- Pre-clinical stage molecules
- Inactive and discontinued molecules
PART 08: INDICATION ANALYSIS
PART 09: THERAPEUTIC ASSESSMENT (THERAPY BASED)
PART 10: THERAPEUTIC ASSESSMENT (ROA BASED)
PART 11: THERAPEUTIC ASSESSMENT BY TARGET
PART 12: KEY COMPANIES
- Active companies: Category and parameters
PART 13: APPENDIX
- List of abbreviations
- Market size and forecast
- Market segmentation
- Geographical insights
- Competitive landscape
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