Paroxysmal Nocturnal Hemoglobinuria Pipeline Drugs Analysis Report (2024 Updates): FDA Approvals, Clinical Trials, MOA, ROA by DelveInsight | Alexion Pharma, Apellis Pharma, Roche, Novartis, RA Pharma

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:

• Companies across the globe are diligently working toward developing novel Paroxysmal Nocturnal Hemoglobinuria treatment therapies with a considerable amount of success over the years. 

• Paroxysmal Nocturnal Hemoglobinuria companies working in the treatment market are Alexion Pharmaceuticals, BioCryst Pharmaceuticals, Apellis Pharmaceuticals, Hoffmann-La Roche, Omeros Corporation, Chengdu Suncadia Medicine, Wuhan Createrna Science, Novartis, Regeneron Pharmaceuticals, Kira Pharmacenticals, Hoffmann-La Roche, NovelMed Therapeutics, and others, are developing therapies for the Paroxysmal Nocturnal Hemoglobinuria treatment 

• Emerging Paroxysmal Nocturnal Hemoglobinuria therapies in the different phases of clinical trials are- Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906, HRS-5965, MY008211A tablets, Iptacopan, Pozelimab, KP104, Crovalimab, NM8074, Pegcetacoplan, and others are expected to have a significant impact on the Paroxysmal Nocturnal Hemoglobinuria market in the coming years.   

• In January 2024, Fabhalta (iptacopan) is a medication authorized to treat adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH), a rare and persistent blood condition. Switzerland-based pharmaceutical firm Novartis is responsible for the development of this factor B inhibitor.

• In December 2023, At the 2023 American Society of Hematology (ASH) Annual Meeting in San Diego, Kira Pharmaceuticals, a leading global biotechnology company specializing in innovative complement therapies for immune-mediated conditions, showcased preliminary safety and efficacy findings from its Phase 2 trial of KP104 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitors. KP104, a novel bifunctional biologic, is designed to block both the alternative and terminal complement pathways.

• 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 disorder of the blood characterized by the destruction of red blood cells (hemolysis), blood clots (thrombosis), and impaired bone marrow function. It is caused by a genetic mutation in the hematopoietic stem cells, leading to the deficiency or absence of certain proteins on the surface of blood cells, particularly red blood cells.

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Emerging Paroxysmal Nocturnal Hemoglobinuria Drugs Under Different Phases of Clinical Development Include:

• Eculizumab: Alexion Pharmaceuticals

• BCX9930: BioCryst Pharmaceuticals

• Pegcetacoplan: Apellis Pharmaceuticals

• Crovalimab: Hoffmann-La Roche

• OMS906: Omeros Corporation

• HRS-5965: Chengdu Suncadia Medicine

• MY008211A tablets: Wuhan Createrna Science

• Iptacopan: Novartis

• Pozelimab: Regeneron Pharmaceuticals

• KP104: Kira Pharmacenticals

• Crovalimab: Hoffmann-La Roche

• NM8074: NovelMed Therapeutics

• Pegcetacoplan: Apellis 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 

• Oral

• Parenteral

• Intravitreal

• Subretinal

• Topical

• Molecule Type

Paroxysmal Nocturnal Hemoglobinuria Molecule Type

Paroxysmal Nocturnal Hemoglobinuria Products have been categorized under various Molecule types, such as

• Monoclonal Antibody

• Peptides

• Polymer

• Small molecule

• Gene therapy

• Product Type

Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapeutics Assessment

• Paroxysmal Nocturnal Hemoglobinuria Assessment by Product Type

• Paroxysmal Nocturnal Hemoglobinuria By Stage and Product Type

• Paroxysmal Nocturnal Hemoglobinuria Assessment by Route of Administration

• Paroxysmal Nocturnal Hemoglobinuria By Stage and Route of Administration

• Paroxysmal Nocturnal Hemoglobinuria Assessment by Molecule Type

• 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

• 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 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 

• 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.

• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Paroxysmal Nocturnal Hemoglobinuria Treatment.

• Paroxysmal Nocturnal Hemoglobinuria key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• 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. 

• 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.

Download Sample PDF Report to know more about Paroxysmal Nocturnal Hemoglobinuria drugs and therapies

Paroxysmal Nocturnal Hemoglobinuria Pipeline Market Drivers

• 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

• 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    

• Coverage: Global

• Key Paroxysmal Nocturnal Hemoglobinuria Companies: Alexion Pharmaceuticals, BioCryst Pharmaceuticals, Apellis Pharmaceuticals, Hoffmann-La Roche, Omeros Corporation, Chengdu Suncadia Medicine, Wuhan Createrna Science, Novartis, Regeneron Pharmaceuticals, Kira Pharmacenticals, Hoffmann-La Roche, NovelMed Therapeutics, and others

• Key Paroxysmal Nocturnal Hemoglobinuria Therapies: Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906, HRS-5965, MY008211A tablets, Iptacopan, Pozelimab, KP104, Crovalimab, NM8074, Pegcetacoplan, and others

• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment: Paroxysmal Nocturnal Hemoglobinuria current marketed and Paroxysmal Nocturnal Hemoglobinuria emerging therapies

• Paroxysmal Nocturnal Hemoglobinuria Market Dynamics: Paroxysmal Nocturnal Hemoglobinuria market drivers and Paroxysmal Nocturnal Hemoglobinuria market barriers 

Request for Sample PDF Report for Paroxysmal Nocturnal Hemoglobinuria Pipeline Assessment and clinical trials

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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