The Paroxysmal Nocturnal Hemoglobinuria 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.
“Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Paroxysmal Nocturnal Hemoglobinuria Market.
Some of the key takeaways from the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report:
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Companies across the globe are diligently working toward developing novel Paroxysmal Nocturnal Hemoglobinuria treatment therapies with a considerable amount of success over the years.
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Paroxysmal Nocturnal Hemoglobinuria companies working in the treatment market are Amyndas Pharmaceuticals, Arrowhead Pharmaceuticals, Kira Pharmaceuticals (US), LLC., Alexion AstraZeneca Rare Disease, Regeneron Pharmaceuticals, and others, are developing therapies for the Paroxysmal Nocturnal Hemoglobinuria treatment
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Emerging Paroxysmal Nocturnal Hemoglobinuria therapies in the different phases of clinical trials are- AMY 101, ARO-C3, KP104, Danicopan, Pozelimab, and others are expected to have a significant impact on the Paroxysmal Nocturnal Hemoglobinuria market in the coming years.
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In December 2023, Kira Pharmaceuticals, a worldwide biotech company developing groundbreaking complement therapies for immune-mediated diseases, shared interim safety and efficacy findings from its Phase 2 study of KP104 in complement inhibitor-naïve PNH patients. This was presented orally at the 2023 American Society of Hematology (ASH) Annual Meeting in San Diego on December 10th. Additionally, the presentation has been chosen for inclusion in the 2024 Highlights of ASH. KP104 is a pioneering bifunctional biologic designed to inhibit both the alternative and terminal complement pathways.
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In March 2023, Regeneron Pharmaceuticals has started a trial named “An Open-Label Extension Study for Assessing the Extended Safety, Tolerance, and Effectiveness of Pozelimab and Cemdisiran Combined Treatment in Individuals Affected by Paroxysmal Hemoglobinuria.” The main goal of this study is to outline the extended-term safety, tolerance, and effectiveness of the combined therapy involving Pozelimab and Cemdisiran in patients diagnosed with PNH. Presently, the trial is ongoing, involving 300 participants, and it’s expected to conclude by March 2028.
Paroxysmal Nocturnal Hemoglobinuria Overview
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood. It is characterized by the destruction of red blood cells (hemolysis), blood clots (thrombosis), and impaired bone marrow function. PNH is caused by a mutation in the PIGA gene in a hematopoietic stem cell, leading to the production of defective blood cells that are more prone to being attacked by the immune system.
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Emerging Paroxysmal Nocturnal Hemoglobinuria Drugs Under Different Phases of Clinical Development Include:
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AMY 101: Amyndas Pharmaceuticals
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ARO-C3: Arrowhead Pharmaceuticals
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KP104: Kira Pharmaceuticals (US), LLC.
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Danicopan: Alexion AstraZeneca Rare Disease
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Pozelimab: Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria Route of Administration
Paroxysmal Nocturnal Hemoglobinuria pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
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Oral
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Parenteral
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Intravitreal
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Subretinal
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Topical
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Molecule Type
Paroxysmal Nocturnal Hemoglobinuria Molecule Type
Paroxysmal Nocturnal Hemoglobinuria Products have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
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Product Type
Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapeutics Assessment
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Paroxysmal Nocturnal Hemoglobinuria Assessment by Product Type
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Paroxysmal Nocturnal Hemoglobinuria By Stage and Product Type
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Paroxysmal Nocturnal Hemoglobinuria Assessment by Route of Administration
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Paroxysmal Nocturnal Hemoglobinuria By Stage and Route of Administration
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Paroxysmal Nocturnal Hemoglobinuria Assessment by Molecule Type
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Paroxysmal Nocturnal Hemoglobinuria by Stage and Molecule Type
DelveInsight’s Paroxysmal Nocturnal Hemoglobinuria Report covers around 25+ products under different phases of clinical development like
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Further Paroxysmal Nocturnal Hemoglobinuria product details are provided in the report. Download the Paroxysmal Nocturnal Hemoglobinuria pipeline report to learn more about the emerging Paroxysmal Nocturnal Hemoglobinuria therapies
Some of the key companies in the Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market include:
Key companies developing therapies for Paroxysmal Nocturnal Hemoglobinuria are – Alexion Pharmaceuticals, Apellis Pharmaceuticals, Hoffmann-La Roche, Novartis, Regeneron Pharmaceuticals, RA Pharmaceuticals Inc., and others.
Paroxysmal Nocturnal Hemoglobinuria Pipeline Analysis:
The Paroxysmal Nocturnal Hemoglobinuria pipeline report provides insights into
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The report provides detailed insights about companies that are developing therapies for the treatment of Paroxysmal Nocturnal Hemoglobinuria with aggregate therapies developed by each company for the same.
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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Paroxysmal Nocturnal Hemoglobinuria Treatment.
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Paroxysmal Nocturnal Hemoglobinuria key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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Paroxysmal Nocturnal Hemoglobinuria 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.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Paroxysmal Nocturnal Hemoglobinuria 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.
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Paroxysmal Nocturnal Hemoglobinuria Pipeline Market Drivers
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Treatment approach for Paroxysmal Hemoglobinuria is rapidly evolving, increasing adoption of novel therapeutics and rising geriatric population are some of the important factors that are fueling the Paroxysmal Nocturnal Hemoglobinuria Market.
Paroxysmal Nocturnal Hemoglobinuria Pipeline Market Barriers
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However, high treatment cost, reimbursement Policies and other factors are creating obstacles in the Paroxysmal Nocturnal Hemoglobinuria Market growth.
Scope of Paroxysmal Nocturnal Hemoglobinuria Pipeline Drug Insight
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Coverage: Global
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Key Paroxysmal Nocturnal Hemoglobinuria Companies: Amyndas Pharmaceuticals, Arrowhead Pharmaceuticals, Kira Pharmaceuticals (US), LLC., Alexion AstraZeneca Rare Disease, Regeneron Pharmaceuticals, and others
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Key Paroxysmal Nocturnal Hemoglobinuria Therapies: AMY 101, ARO-C3, KP104, Danicopan, Pozelimab, and others
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Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment: Paroxysmal Nocturnal Hemoglobinuria current marketed and Paroxysmal Nocturnal Hemoglobinuria emerging therapies
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Paroxysmal Nocturnal Hemoglobinuria Market Dynamics: Paroxysmal Nocturnal Hemoglobinuria market drivers and Paroxysmal Nocturnal Hemoglobinuria market barriers
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Table of Contents
1. Paroxysmal Nocturnal Hemoglobinuria Report Introduction
2. Paroxysmal Nocturnal Hemoglobinuria Executive Summary
3. Paroxysmal Nocturnal Hemoglobinuria Overview
4. Paroxysmal Nocturnal Hemoglobinuria- Analytical Perspective In-depth Commercial Assessment
5. Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapeutics
6. Paroxysmal Nocturnal Hemoglobinuria Late Stage Products (Phase II/III)
7. Paroxysmal Nocturnal Hemoglobinuria Mid Stage Products (Phase II)
8. Paroxysmal Nocturnal Hemoglobinuria Early Stage Products (Phase I)
9. Paroxysmal Nocturnal Hemoglobinuria Preclinical Stage Products
10. Paroxysmal Nocturnal Hemoglobinuria Therapeutics Assessment
11. Paroxysmal Nocturnal Hemoglobinuria Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Paroxysmal Nocturnal Hemoglobinuria Key Companies
14. Paroxysmal Nocturnal Hemoglobinuria Key Products
15. Paroxysmal Nocturnal Hemoglobinuria Unmet Needs
16 . Paroxysmal Nocturnal Hemoglobinuria Market Drivers and Barriers
17. Paroxysmal Nocturnal Hemoglobinuria Future Perspectives and Conclusion
18. Paroxysmal Nocturnal Hemoglobinuria Analyst Views
19. Appendix
20. About DelveInsight
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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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