Paroxysmal Nocturnal Hemoglobinuria Clinical Trials & Pipeline Outlook 2025: Approvals, Therapies, Treatment, Companies by DelveInsight

(Albany, United States) DelveInsight's "Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight, 2025" report offers a detailed overview of the current clinical development landscape and future growth opportunities within the Paroxysmal Nocturnal Hemoglobinuria (PNH) market.

According to DelveInsight's analysis, over 20 leading companies worldwide are actively developing more than 25 therapeutic candidates for PNH. The report provides an in-depth examination of clinical trials, therapies in development, mechanisms of action, routes of administration, and recent advancements.

It includes comprehensive commercial and clinical evaluations of pipeline products, spanning from preclinical to marketed stages. Each drug profile features its mechanism of action, clinical trial data, regulatory approvals (if applicable), and associated development activities, including technology platforms, collaborations, mergers and acquisitions, funding, designations, and other relevant product details.

Download sample report to know in detail about Paroxysmal Nocturnal Hemoglobinuria therapies @ Paroxysmal Nocturnal Hemoglobinuria Pipeline Outlook - https://www.delveinsight.com/sample-request/paroxysmal-nocturnal-hemoglobinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Some of the key takeaways from the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report:
• 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 June 2025, Regeneron Pharmaceuticals has announced results of an Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
• In May 2025, Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced that Chugai Pharma Taiwan Ltd. (hereafter, CPT), a wholly-owned subsidiary of Chugai, obtained an orphan drug import drug license from the Taiwan Food and Drug Administration (TFDA) for Chugai's PiaSky® for "for the treatment of the patients 13 years and older with paroxysmal nocturnal hemoglobinuria (PNH) and body weight of at least 40 kg," on May 19, 2025. CPT is responsible for the development, regulatory submission, import, and sales of Chugai-originated products in Taiwan.
• 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.
• Key Paroxysmal Nocturnal Hemoglobinuria companies in the treatment market include AstraZeneca (Alexion Pharmaceuticals), Novartis, Regeneron Pharmaceuticals, Apellis Pharmaceuticals, Omeros Corporation, Swedish Orphan Biovitrum, BioCryst Pharmaceuticals, Alnylam Pharmaceuticals, Kira Pharma, NovelMed Therapeutics, Hoffmann-La Roche (Chugai Pharmaceutical), Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, and others-each actively engaged in advancing therapies for Paroxysmal Nocturnal Hemoglobinuria.
• 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 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, life-threatening blood disorder characterized by the destruction of red blood cells (hemolysis), bone marrow failure, and a higher risk of blood clots (thrombosis). It is caused by a mutation in the PIGA gene in hematopoietic stem cells, leading to the absence of protective surface proteins (CD55 and CD59) on blood cells. This deficiency makes red blood cells vulnerable to attack by the body's complement system, resulting in intravascular hemolysis.

Symptoms of Paroxysmal Nocturnal Hemoglobinuria include fatigue, abdominal pain, dark-colored urine (especially in the morning), difficulty swallowing, and shortness of breath. The disease can also lead to complications such as pulmonary hypertension, kidney damage, and stroke. PNH can occur on its own or alongside other bone marrow disorders like aplastic anemia or myelodysplastic syndromes.

Paroxysmal Nocturnal Hemoglobinuria Diagnosis typically involves flow cytometry to detect the absence of GPI-anchored proteins on blood cells. Paroxysmal Nocturnal Hemoglobinuria Treatment options have significantly advanced with the development of complement inhibitors like eculizumab and ravulizumab, which reduce hemolysis and thrombosis risk. Ongoing research focuses on novel complement pathway inhibitors and potential curative approaches, including bone marrow transplantation. Early diagnosis and appropriate treatment are essential for improving outcomes and quality of life in Paroxysmal Nocturnal Hemoglobinuria patients.

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

Paroxysmal Nocturnal Hemoglobinuria ROA
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

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

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 Clinical Trials 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 - https://www.delveinsight.com/sample-request/paroxysmal-nocturnal-hemoglobinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Some of the key companies in the Paroxysmal Nocturnal Hemoglobinuria Therapeutics Market include:
Key companies developing therapies for Paroxysmal Nocturnal Hemoglobinuria are AstraZeneca (Alexion Pharmaceuticals), Novartis, Regeneron Pharmaceuticals, Apellis Pharmaceuticals, Omeros Corporation, Swedish Orphan Biovitrum, BioCryst Pharmaceuticals, Alnylam Pharmaceuticals, Kira Pharma, NovelMed Therapeutics, Hoffmann-La Roche (Chugai Pharmaceutical), Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, and others

Download Sample PDF Report to know more about Paroxysmal Nocturnal Hemoglobinuria drugs and therapies - https://www.delveinsight.com/sample-request/paroxysmal-nocturnal-hemoglobinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

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

Contact Us:
Ankit Nigam
Manager Marketing
info@delveinsight.com
+14699457679

About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

This release was published on openPR.