Paroxysmal Nocturnal Hemoglobinuria Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, and Companies

DelveInsight's, "Paroxysmal Nocturnal Haemoglobinuria Pipeline Insight 2025" 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 August 19, 2025, Regeneron Pharmaceuticals announced the initiation of a clinical study investigating a novel treatment approach with its two investigational drugs, pozelimab and cemdisiran, for individuals diagnosed with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this trial is to assess the long-term safety and efficacy of the pozelimab + cemdisiran combination therapy, which is collectively referred to as the "study drugs" within the trial framework.
* On August 14, 2025, Hoffmann-La Roche disclosed the commencement of a Phase I/II, first-in-human trial designed with four sequential parts and an open-label extension (OLE). In Part 1, the study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab in healthy volunteers. Subsequently, in Parts 2, 3, 4, and the OLE, the trial will focus on assessing the safety, tolerability, PK, and PD of multiple doses of crovalimab in patients with PNH. Additionally, the efficacy of crovalimab will be examined during Parts 2, 3, and 4.
* 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

* Introduction
* Executive Summary
* Paroxysmal Nocturnal Haemoglobinuria: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Paroxysmal Nocturnal Haemoglobinuria - DelveInsight's Analytical Perspective
* In-depth Commercial Assessment
* Paroxysmal Nocturnal Haemoglobinuria Collaboration Deals
* Late Stage Products (Phase III)
* Crovalimab: Hoffman-La-Roche
* Mid Stage Products (Phase II)
* BCX9930: BioCryst pharmaceuticals
* Eary Stage Products (Phase I)
* CAN106: CanBridge
* Inactive Products
* Paroxysmal Nocturnal Haemoglobinuria Key Companies
* Paroxysmal Nocturnal Haemoglobinuria Key Products
* Paroxysmal Nocturnal Haemoglobinuria- Unmet Needs
* Paroxysmal Nocturnal Haemoglobinuria- Market Drivers and Barriers
* Paroxysmal Nocturnal Haemoglobinuria- Future Perspectives and Conclusion
* Paroxysmal Nocturnal Haemoglobinuria Analyst Views
* Paroxysmal Nocturnal Haemoglobinuria Key Companies
* Appendix

About Us

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