Paroxysmal Nocturnal Hemoglobinuria Pipeline 2025: Therapies, MOA Insights, and Key Clinical Trial Updates by DelveInsight | Alexion Pharma, Apellis Pharma, Hoffmann-La Roche, Novartis, Regeneron Pharma, RA Pharma

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Paroxysmal Nocturnal Hemoglobinuria pipeline constitutes 20+ key companies continuously working towards developing 25+ Paroxysmal Nocturnal Hemoglobinuria treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Paroxysmal Nocturnal Hemoglobinuria Market.

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.

Some of the key takeaways from the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report: https://www.delveinsight.com/sample-request/paroxysmal-nocturnal-hemoglobinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• 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 Hoffmann-La Roche (Chugai Pharmaceutical), AstraZeneca (Alexion Pharmaceuticals), Apellis Pharmaceuticals, Swedish Orphan Biovitrum, Regeneron, Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation, BioCryst Pharmaceuticals, Novartis, Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, Regeneron Pharma, Kira Pharma, 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- PIASKY (Crovalimab), SOLIRIS (eculizumab), EMPAVELI/ASPAVELI (pegcetacoplan), Pozelimab (REGN3918), Zaltenibart (OMS906), Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906 study drug, HRS-5965 tablets, MY008211A tablets, Iptacopan, Pozelimab, KP104, NM8074, Danicopan, Pegcetacoplan, and others are expected to have a significant impact on the Paroxysmal Nocturnal Hemoglobinuria market in the coming years.
• In June 2025, Novartis has reported positive outcomes from the APPULSE-PNH Phase 3B trial, which evaluated the safety and effectiveness of iptacopan (Fabhalta), a twice-daily oral monotherapy, in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had hemoglobin (Hb) levels of ≥10 g/dL and transitioned from anti-C5 treatments such as eculizumab or ravulizumab.
• In May 2025, NovelMed announced encouraging 12-week interim findings from its ongoing multi-dose Phase II study of Ruxoprubart, an innovative complement-targeting immunotherapy, in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH). The interim results show that Ruxoprubart, used as a monotherapy, was safe, well-tolerated, and successfully met all primary efficacy goals. The treatment led to transfusion avoidance, higher hemoglobin levels, lower LDH levels, and an increase in PNH clone size-key indicators of better disease management and improved quality of life for individuals with PNH.
• In December 2024, Novartis has announced positive topline results from the multicenter Phase IIIB APPULSE-PNH trial evaluating oral Fabhalta (iptacopan) for adults with paroxysmal nocturnal hemoglobinuria (PNH). Participants in the study transitioned from anti-C5 therapies and had a baseline hemoglobin (Hb) level of at least 10g/dL following treatment with eculizumab or ravulizumab.
• In December 2024, Omeros Corporation (Nasdaq: OMER) announced that two posters on zaltenibart (OMS906), its investigational MASP-3 inhibitor targeting the alternative complement pathway, were presented yesterday at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego. The posters, focused on zaltenibart's potential in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder, highlighted positive Phase 2 clinical data and clinical pharmacology analyses supporting dose selection for the upcoming Phase 3 program. Enrollment for the Phase 3 trials in PNH is expected to begin in early 2025.
• In August 2024, The EMA approved PIASKY (crovalimab) for treating adults and adolescents (aged 12 and older, weighing at least 40 kg) with Paroxysmal Nocturnal Hemoglobinuria, regardless of prior treatment with C5 inhibitors. Previously, in June 2024, the US FDA also granted approval for PIASKY to be used in adult and pediatric patients aged 13 and older with the same condition, provided they weigh at least 40 kg.

Paroxysmal Nocturnal Hemoglobinuria Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disorder of the blood characterized by the premature destruction of red blood cells (hemolysis), blood clotting issues, and the potential development of blood clots (thrombosis). PNH arises due to a mutation in the bone marrow stem cells, leading to the deficiency or absence of certain proteins that help protect blood cells from the immune system's attack.

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Emerging Paroxysmal Nocturnal Hemoglobinuria Drugs Under Different Phases of Clinical Development Include:
• PIASKY (Crovalimab): Hoffmann-La Roche (Chugai Pharmaceutical)
• SOLIRIS (eculizumab): AstraZeneca (Alexion Pharmaceuticals)
• EMPAVELI/ASPAVELI (pegcetacoplan): Apellis Pharmaceuticals/Swedish Orphan Biovitrum
• Pozelimab (REGN3918): Regeneron Pharmaceuticals/Alnylam Pharmaceuticals
• Zaltenibart (OMS906): Omeros Corporation
• Eculizumab: Alexion Pharmaceuticals
• BCX9930: BioCryst Pharmaceuticals
• Pegcetacoplan: Apellis Pharmaceuticals, Inc.
• Crovalimab: Hoffmann-La Roche
• OMS906 study drug: Omeros Corporation
• HRS-5965 tablets: Chengdu Suncadia Medicine Co., Ltd.
• MY008211A tablets: Wuhan Createrna Science
• Iptacopan: Novartis
• Pozelimab: Regeneron Pharma
• KP104: Kira Pharma
• NM8074: NovelMed Therapeutics
• Danicopan: Alexion Pharmaceuticals, Inc.
• Pegcetacoplan: Apellis Pharma

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
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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.

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
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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: Hoffmann-La Roche (Chugai Pharmaceutical), AstraZeneca (Alexion Pharmaceuticals), Apellis Pharmaceuticals, Swedish Orphan Biovitrum, Regeneron, Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation, BioCryst Pharmaceuticals, Novartis, Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, Regeneron Pharma, Kira Pharma, NovelMed Therapeutics, and others
• Key Paroxysmal Nocturnal Hemoglobinuria Therapies: PIASKY (Crovalimab), SOLIRIS (eculizumab), EMPAVELI/ASPAVELI (pegcetacoplan), Pozelimab (REGN3918), Zaltenibart (OMS906), Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906 study drug, HRS-5965 tablets, MY008211A tablets, Iptacopan, Pozelimab, KP104, NM8074, Danicopan, 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

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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

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About DelveInsight
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