Niemann-Pick Type C Disease - Pipeline Insight, 2021
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DelveInsight’s, “Niemann-Pick Type C Disease – Pipeline Insight, 2021,” report provides comprehensive insights about 13+ companies and 13+ pipeline drugs in Niemann-Pick Type C Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Niemann-Pick Type C Disease: Overview
Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. NPC belongs to a larger group of more than 50 disorders known as lysosomal storage disorders. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. Individuals with NPC can have onset of symptoms at different ages that have been grouped historically as: perinatal (shortly before and after birth), early infantile (3 months to < 2 years), late infantile (2 to < 6 years), juvenile (6 to < 15 years), and adult (15 years and greater). Individuals with NPC have mutations in one of two genes, NPC1 or NPC2 and are inherited in an autosomal recessive manner. Approximately 95% of affected individuals have mutations in NPC1. Niemann-Pick disease type C is diagnosed based on characteristic symptoms obtained from a thorough clinical evaluation, and confirmed by a variety of specialized tests. Proper diagnosis of NPC requires physicians to suspect the diagnosis based upon symptoms, and to follow up with appropriate laboratory tests to evaluate the function of the protein or the presence of accumulated byproducts (biochemical tests), and to identify mutations in the NPC1 or NPC2 gene (gene sequencing). Current treatment is directed toward the specific symptoms apparent in each individual. Several therapies have been studied or are currently being studied to assess their long-term safety and effectiveness as potential treatments for individuals with NPC.
'Niemann-Pick Type C Disease - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann-Pick Type C Disease pipeline landscape is provided which includes the disease overview and Niemann-Pick Type C Disease treatment guidelines. The assessment part of the report embraces, in depth Niemann-Pick Type C Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Niemann-Pick Type C Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Niemann-Pick Type C Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Niemann-Pick Type C Disease Emerging Drugs
Further product details are provided in the report.
Niemann-Pick Type C Disease: Therapeutic Assessment
This segment of the report provides insights about the different Niemann-Pick Type C Disease drugs segregated based on following parameters that define the scope of the report, such as:
Niemann-Pick Type C Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann-Pick Type C Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Niemann-Pick Type C Disease drugs.
Niemann-Pick Type C Disease Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Niemann-Pick Type C Disease – Pipeline Insight, 2021,” report provides comprehensive insights about 13+ companies and 13+ pipeline drugs in Niemann-Pick Type C Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Niemann-Pick Type C Disease: Overview
Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. NPC belongs to a larger group of more than 50 disorders known as lysosomal storage disorders. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. Individuals with NPC can have onset of symptoms at different ages that have been grouped historically as: perinatal (shortly before and after birth), early infantile (3 months to < 2 years), late infantile (2 to < 6 years), juvenile (6 to < 15 years), and adult (15 years and greater). Individuals with NPC have mutations in one of two genes, NPC1 or NPC2 and are inherited in an autosomal recessive manner. Approximately 95% of affected individuals have mutations in NPC1. Niemann-Pick disease type C is diagnosed based on characteristic symptoms obtained from a thorough clinical evaluation, and confirmed by a variety of specialized tests. Proper diagnosis of NPC requires physicians to suspect the diagnosis based upon symptoms, and to follow up with appropriate laboratory tests to evaluate the function of the protein or the presence of accumulated byproducts (biochemical tests), and to identify mutations in the NPC1 or NPC2 gene (gene sequencing). Current treatment is directed toward the specific symptoms apparent in each individual. Several therapies have been studied or are currently being studied to assess their long-term safety and effectiveness as potential treatments for individuals with NPC.
'Niemann-Pick Type C Disease - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann-Pick Type C Disease pipeline landscape is provided which includes the disease overview and Niemann-Pick Type C Disease treatment guidelines. The assessment part of the report embraces, in depth Niemann-Pick Type C Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Niemann-Pick Type C Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Niemann-Pick Type C Disease R&D. The therapies under development are focused on novel approaches to treat/improve Niemann-Pick Type C Disease.
This segment of the Niemann-Pick Type C Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Niemann-Pick Type C Disease Emerging Drugs
- Arimoclomol: Orphazyme
- Adrabetadex: Mandos, LLC
Further product details are provided in the report.
Niemann-Pick Type C Disease: Therapeutic Assessment
This segment of the report provides insights about the different Niemann-Pick Type C Disease drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Niemann-Pick Type C Disease
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Niemann-Pick Type C Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann-Pick Type C Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Niemann-Pick Type C Disease drugs.
Niemann-Pick Type C Disease Report Insights
- Niemann-Pick Type C Disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Niemann-Pick Type C Disease drugs?
- How many Niemann-Pick Type C Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Niemann-Pick Type C Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Niemann-Pick Type C Disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Niemann-Pick Type C Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
Introduction
Executive Summary
Niemann-Pick Type C Disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Late Stage Products (Preregistration)
Comparative Analysis
Arimoclomol: Orphazyme
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Mid Stage Products (Phase II)
Comparative Analysis
IB1001: IntraBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Preclinical stage products
Comparative Analysis
Research programme: Niemann-Pick disease gene therapies - Sarepta Therapeutics/StrideBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Inactive Products
Comparative Analysis
Niemann-Pick Type C Disease Key Companies
Niemann-Pick Type C Disease Key Products
Niemann-Pick Type C Disease- Unmet Needs
Niemann-Pick Type C Disease- Market Drivers and Barriers
Niemann-Pick Type C Disease- Future Perspectives and Conclusion
Niemann-Pick Type C Disease Analyst Views
Niemann-Pick Type C Disease Key Companies
Appendix
Executive Summary
Niemann-Pick Type C Disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Late Stage Products (Preregistration)
Comparative Analysis
Arimoclomol: Orphazyme
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Mid Stage Products (Phase II)
Comparative Analysis
IB1001: IntraBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Preclinical stage products
Comparative Analysis
Research programme: Niemann-Pick disease gene therapies - Sarepta Therapeutics/StrideBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Inactive Products
Comparative Analysis
Niemann-Pick Type C Disease Key Companies
Niemann-Pick Type C Disease Key Products
Niemann-Pick Type C Disease- Unmet Needs
Niemann-Pick Type C Disease- Market Drivers and Barriers
Niemann-Pick Type C Disease- Future Perspectives and Conclusion
Niemann-Pick Type C Disease Analyst Views
Niemann-Pick Type C Disease Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Niemann-Pick Type C Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Niemann-Pick Type C Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Niemann-Pick Type C Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Niemann-Pick Type C Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products