Paroxysmal Nocturnal Hemoglobinuria Treatment Pipeline Shows Strong Momentum as 20+ Pharma Companies in the Race | DelveInsight

DelveInsight's, "Paroxysmal Nocturnal Haemoglobinuria Pipeline Insight 2026" report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Paroxysmal Nocturnal Haemoglobinuria 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.

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Key Takeaways from the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report
• On January 08, 2026- Chengdu Suncadia Medicine Co., Ltd. announced phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules.
• DelveInsight's Paroxysmal Nocturnal Hemoglobinuria pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline therapies for Paroxysmal Nocturnal Hemoglobinuria treatment.
• The leading Paroxysmal Nocturnal Hemoglobinuria Companies such as Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio and others.
• Promising Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapies such as HSK39297 tablets, Coversin, MY008211A tablets, Eculizumab Injection, Pegcetacoplan, Crovalimab, Ravulizumab, Iptacopan, LNP023, and others.

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Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs Profile
• Crovalimab: Hoffman-La-Roche
Crovalimab (RG6107) is a humanised complement inhibitor C5 monoclonal antibody discovered by Chugai using recycling antibody technology. By blocking the cleavage of C5 to C5a and C5b, it is expected to inhibit complement activation, which is the cause of a number of diseases. As the complement system is a key innate immune defense mechanism, we plan to study the potential of this antibody in a broader range of complement-mediated diseases. A phase III clinical trial is evaluating crovalimab for the treatment of paroxysmal nocturnal hemoglobinuria.

• Pozelimab: Regeneron Pharmaceuticals
Pozelimab is an investigational, fully-human monoclonal antibody designed to block complement factor C5 and prevent the destruction of red blood cells (hemolysis) that cause the symptoms of PNH and other diseases mediated by complement pathway activity. It is an IgG4 antibody that binds with high affinity to wild-type and variant human C5 and blocks its activity. Pozelimab was invented using Regeneron's proprietary VelocImmune technology, which uses a unique genetically-humanized mouse to produce optimized fully-human antibodies. Pozelimab is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.

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 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

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Paroxysmal Nocturnal Hemoglobinuria Companies
Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio and others.

Paroxysmal Nocturnal Haemoglobinuria 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 Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
• Product Type

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Scope of the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report
• Coverage- Global
• Paroxysmal Nocturnal Hemoglobinuria Companies- Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio and others.
• Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapies- HSK39297 tablets, Coversin, MY008211A tablets, Eculizumab Injection, Pegcetacoplan, Crovalimab, Ravulizumab, Iptacopan, LNP023, and others.
• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content
1. Introduction
2. Executive Summary
3. Paroxysmal Nocturnal Haemoglobinuria: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Paroxysmal Nocturnal Haemoglobinuria - DelveInsight's Analytical Perspective
7. In-depth Commercial Assessment
8. Paroxysmal Nocturnal Haemoglobinuria Collaboration Deals
9. Late Stage Products (Phase III)
10. Crovalimab: Hoffman-La-Roche
11. Mid Stage Products (Phase II)
12. BCX9930: BioCryst pharmaceuticals
13. Eary Stage Products (Phase I)
14. CAN106: CanBridge
15. Inactive Products
16. Paroxysmal Nocturnal Haemoglobinuria Key Companies
17. Paroxysmal Nocturnal Haemoglobinuria Key Products
18. Paroxysmal Nocturnal Haemoglobinuria- Unmet Needs
19. Paroxysmal Nocturnal Haemoglobinuria- Market Drivers and Barriers
20. Paroxysmal Nocturnal Haemoglobinuria- Future Perspectives and Conclusion
21. Paroxysmal Nocturnal Haemoglobinuria Analyst Views
22. Paroxysmal Nocturnal Haemoglobinuria Key Companies
23. Appendix

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