Niemann-Pick Disease Type C Pipeline 2024: Clinical Trials Assessment, FDA Approvals, Therapies, and Key Companies Involved by DelveInsight | Actelion Pharmaceuticals, Cyclo Therapeutics, IntraBio, Zevra Therapeutics, Azafaros A.G

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Niemann-Pick Disease Type C pipeline constitutes 10+ key companies continuously working towards developing 15+ Niemann-Pick Disease Type C treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Niemann-Pick Disease Type C Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Niemann-Pick Disease Type C Market.

The Niemann-Pick Disease Type C 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.

Some of the key takeaways from the Niemann-Pick Disease Type C Pipeline Report: https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Niemann-Pick Disease Type C treatment therapies with a considerable amount of success over the years.
• Niemann-Pick Disease Type C companies working in the treatment market are Actelion Pharmaceuticals, Cyclo Therapeutics, IntraBio, Zevra Therapeutics, Azafaros A.G, and others, are developing therapies for the Niemann-Pick Disease Type C treatment
• Emerging Niemann-Pick Disease Type C therapies in the different phases of clinical trials are- ZAVESCA/BRAZAVES (miglustat), Trappsol Cyclo, IB10001, Arimoclomol, AZ-3102, IB1001, N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, and others are expected to have a significant impact on the Niemann-Pick Disease Type C market in the coming years.
• In July 2024, Azafaros has announced positive topline results from the Phase II RAINBOW clinical trial of nizubaglustat for treating patients with GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The trial, conducted at three sites in Brazil, enrolled 13 participants aged 12 and older. It evaluated the safety, pharmacokinetics, and pharmacodynamics of two different doses of nizubaglustat.
• In March 2024, Zevra Therapeutics announced that the United States Food and Drug Administration (FDA) has extended the review timeline for the New Drug Application (NDA) for arimoclomol, intended for treating Niemann-Pick disease type C. The FDA has established a new Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024.
• In March 2024, The US FDA has accepted IntraBio's New Drug Application (NDA) and granted a priority review for IB1001 as a treatment for Niemann-Pick disease type C. The application has been assigned a Priority Review with a target action date under the Prescription Drug User Fee Act (PDUFA) of September 24, 2024.

Niemann-Pick Disease Type C Overview
Niemann-Pick Disease Type C (NPC) is a rare, genetic disorder that impairs the body's ability to metabolize cholesterol and other lipids within cells. This leads to an abnormal accumulation of these substances, particularly in the brain, liver, and spleen, causing progressive cellular damage. NPC is caused by mutations in either the NPC1 or NPC2 gene, which are responsible for regulating lipid transport within cells. Symptoms can vary widely but often include difficulty with movement, speech, and swallowing, as well as liver and lung dysfunction. NPC is progressive and life-limiting, with treatment focusing on symptom management and supportive care, as there is currently no cure.

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Emerging Niemann-Pick Disease Type C Drugs Under Different Phases of Clinical Development Include:
• ZAVESCA/BRAZAVES (miglustat): Actelion Pharmaceuticals
• Trappsol Cyclo: Cyclo Therapeutics
• IB10001: IntraBio
• Arimoclomol: Zevra Therapeutics
• AZ-3102: Azafaros A.G
• IB1001: IntraBio
• N-Acetyl-L-Leucine: IntraBio Inc
• arimoclomol: ZevraDenmark
• Hydroxypropyl-beta-cyclodextrin: Cyclo Therapeutics
• AZ-3102: Azafaros A.G.

Niemann-Pick Disease Type C Route of Administration
Niemann-Pick Disease Type C pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Niemann-Pick Disease Type C Molecule Type
Niemann-Pick Disease Type C Products have been categorized under various Molecule types, such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Niemann-Pick Disease Type C Pipeline Therapeutics Assessment
• Niemann-Pick Disease Type C Assessment by Product Type
• Niemann-Pick Disease Type C By Stage and Product Type
• Niemann-Pick Disease Type C Assessment by Route of Administration
• Niemann-Pick Disease Type C By Stage and Route of Administration
• Niemann-Pick Disease Type C Assessment by Molecule Type
• Niemann-Pick Disease Type C by Stage and Molecule Type

DelveInsight's Niemann-Pick Disease Type C Report covers around 15+ 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

Further Niemann-Pick Disease Type C product details are provided in the report. Download the Niemann-Pick Disease Type C pipeline report to learn more about the emerging Niemann-Pick Disease Type C therapies at:
https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Some of the key companies in the Niemann-Pick Disease Type C Therapeutics Market include:
Key companies developing therapies for Niemann-Pick Disease Type C are - Azafaros A.G., Cyclo Therapeutics, Inc., ZevraDenmark, Genzyme, IntraBio Inc, Mandos Health, and others.

Niemann-Pick Disease Type C Pipeline Analysis:
The Niemann-Pick Disease Type C pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the treatment of Niemann-Pick Disease Type C with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Niemann-Pick Disease Type C Treatment.
• Niemann-Pick Disease Type C key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Niemann-Pick Disease Type C Drugs 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.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Niemann-Pick Disease Type C 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.

Download Sample PDF Report to know more about Niemann-Pick Disease Type C drugs and therapies- https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Niemann-Pick Disease Type C Pipeline Market Drivers
• Rising Research and Development, Orphan Drug Designations and Incentives, Improved Diagnostic Techniques, Growing Awareness and Advocacy, are some of the important factors that are fueling the Niemann-Pick Disease Type C Market.

Niemann-Pick Disease Type C Pipeline Market Barriers
• However, High R&D Costs, Complexity of Disease Mechanism, Limited Patient Population, Regulatory Hurdles, High Treatment Costs, and other factors are creating obstacles in the Niemann-Pick Disease Type C Market growth.

Scope of Niemann-Pick Disease Type C Pipeline Drug Insight
• Coverage: Global
• Key Niemann-Pick Disease Type C Companies: Actelion Pharmaceuticals, Cyclo Therapeutics, IntraBio, Zevra Therapeutics, Azafaros A.G, and others
• Key Niemann-Pick Disease Type C Therapies: ZAVESCA/BRAZAVES (miglustat), Trappsol Cyclo, IB10001, Arimoclomol, AZ-3102, IB1001, N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, 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

Request for Sample PDF Report for Niemann-Pick Disease Type C Pipeline Assessment and clinical trials- https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Table of Contents
1. Niemann-Pick Disease Type C Report Introduction
2. Niemann-Pick Disease Type C Executive Summary
3. Niemann-Pick Disease Type C Overview
4. Niemann-Pick Disease Type C- Analytical Perspective In-depth Commercial Assessment
5. Niemann-Pick Disease Type C Pipeline Therapeutics
6. Niemann-Pick Disease Type C Late Stage Products (Phase II/III)
7. Niemann-Pick Disease Type C Mid Stage Products (Phase II)
8. Niemann-Pick Disease Type C Early Stage Products (Phase I)
9. Niemann-Pick Disease Type C Preclinical Stage Products
10. Niemann-Pick Disease Type C Therapeutics Assessment
11. Niemann-Pick Disease Type C Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Niemann-Pick Disease Type C Key Companies
14. Niemann-Pick Disease Type C Key Products
15. Niemann-Pick Disease Type C Unmet Needs
16 . Niemann-Pick Disease Type C Market Drivers and Barriers
17. Niemann-Pick Disease Type C Future Perspectives and Conclusion
18. Niemann-Pick Disease Type C Analyst Views
19. Appendix
20. About DelveInsight

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About DelveInsight

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