Paroxysmal Nocturnal Hemoglobinuria Market Forecast Upto 2036: 5 Emerging Therapies Poised to Accelerate Market Growth | DelveInsight
The expected launch of potential therapies such as Pozelimab + Cemdisiran (Regeneron Pharmaceuticals), Zaltenibart (OMS906; Novo Nordisk), Ruxoprubart (NM8074; NovelMed Therapeutics), NM5072 (NovelMed Therapeutics), ADX-038 (ADARx Pharmaceuticals), KP104 (Kira Pharmaceuticals), and others may increase the paroxysmal nocturnal hemoglobinuria market size in the coming years, assisted by an increase in the diagnosed prevalent population of PNH.
LAS VEGAS, Sept. 17, 2026 /PRNewswire/ -- Until 2007, paroxysmal nocturnal hemoglobinuria (PNH) was a life-threatening disorder with limited therapeutic options for controlling hemolysis and thrombosis, the latter being a major contributor to mortality. The treatment landscape underwent a significant transformation with the introduction of the anti-C5 complement inhibitor eculizumab, which substantially reduced hemolysis, lowered transfusion requirements, and, importantly, decreased the risk of thrombosis.
The current disease-modifying treatment approach for PNH is centered on complement inhibition. FDA-approved therapies, including SOLIRIS (eculizumab), ULTOMIRIS (ravulizumab), and EMPAVELI (pegcetacoplan), have established complement inhibition as the standard of care. However, limitations and safety considerations associated with terminal complement inhibition have driven the development of alternative therapeutic approaches. More recent approvals of proximal complement inhibitors, including FABHALTA (iptacopan), VOYDEYA (danicopan), and PIASKY (crovalimab), are expanding treatment options and aim to provide more effective disease control while addressing limitations associated with existing therapies.
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The PNH pipeline continues to evolve, with emerging candidates such as Zaltenibart (OMS906), pozelimab, and ruxoprubart (NM8074), among others, highlighting ongoing innovation and the growing diversity of complement-targeted treatment strategies.
Aparna Thakur, Project Manager of Forecasting & Analytics at DelveInsight, said that among the emerging therapies, Pozelimab is expected to generate the highest revenue in the 7MM, followed by Zaltenibart. In a Phase II study, Zaltenibart demonstrated strong efficacy in PNH patients with suboptimal responses to ravulizumab.
Reflecting continued therapeutic innovation and significant unmet medical needs, DelveInsight estimates that the paroxysmal nocturnal hemoglobinuria market across the seven major markets, comprising the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, was valued at approximately USD 1.5 billion in 2025. The market is projected to expand at a CAGR of 7.1% during the forecast period through 2036, driven by the introduction of novel complement-targeted therapies, advances in disease management, increasing awareness and diagnosis of PNH, and ongoing efforts to address breakthrough hemolysis, transfusion dependence, and other limitations associated with existing treatments.
Below, we highlight 5 promising emerging therapies poised to reshape the future of the paroxysmal nocturnal hemoglobinuria market.
Regeneron Pharmaceuticals' Pozelimab + Cemdisiran
C5 complement inhibitor
Pozelimab is an investigational fully human monoclonal antibody engineered to inhibit complement component C5, thereby preventing the destruction of red blood cells associated with paroxysmal nocturnal hemoglobinuria (PNH) and other disorders driven by complement pathway activation. As an IgG4 antibody, pozelimab binds with high affinity to both wild-type and variant forms of human C5, blocking its biological activity.
Pozelimab was developed using Regeneron's proprietary VelocImmune® technology, which employs a genetically humanized mouse platform to generate optimized fully human antibodies. The technology enables the efficient and direct identification of multiple antibody drug candidates from immunized mice. By overcoming limitations associated with conventional antibody-generation approaches, VelocImmune facilitates the rapid development of fully human antibodies with strong target binding while minimizing the potential for immune reactions associated with antibodies containing nonhuman, typically murine, components.
As part of its broader clinical development program, pozelimab is also being investigated in combination with cemdisiran, Alnylam's small interfering RNA (siRNA) therapy targeting complement C5, for the treatment of additional complement-mediated disorders, including PNH and myasthenia gravis (MG). Regeneron Pharmaceuticals has reported that results from the Phase III study evaluating the pozelimab–cemdisiran combination in PNH are anticipated in the fourth quarter of 2026 or the first quarter of 2027.
Novo Nordisk's Zaltenibart (OMS906)
MASP-3 inhibitor
Zaltenibart (OMS906), a lead monoclonal antibody designed to target mannan-binding lectin-associated serine protease-3 (MASP-3), a key activator of the alternative complement pathway, is currently being evaluated in Phase II clinical programs for paroxysmal nocturnal hemoglobinuria and complement 3 glomerulopathy (C3G). In a single-ascending-dose Phase I study conducted in healthy participants, zaltenibart demonstrated a favorable safety and tolerability profile, with no significant safety concerns reported. The therapy has also received Orphan Drug Designation (ODD) from the FDA for the treatment of PNH.
In November 2025, Omeros Corporation announced the completion of an asset purchase and license agreement with Novo Nordisk, under which Novo Nordisk obtained exclusive worldwide rights to develop and commercialize zaltenibart, formerly known as OMS906, across all indications. The transaction transferred the global development and commercialization rights for the human monoclonal antibody targeting MASP-3 to Novo Nordisk.
Discover more about the new treatment options for paroxysmal nocturnal hemoglobinuria @ Paroxysmal Nocturnal Hemoglobinuria Treatment Market
NovelMed Therapeutics's Ruxoprubart (NM8074)
Complement C3–C5 convertases inhibitor
Ruxoprubart (NM8074) is an anti-Bb monoclonal antibody designed to selectively inhibit the complement alternative pathway while preserving the classical pathway, which remains essential for effective infection clearance. The therapy completed a Phase I clinical trial involving 40 healthy volunteers, demonstrating a favorable safety and tolerability profile across all tested doses. Complete inhibition of the alternative pathway was achieved, with the duration of inhibition increasing in a dose-dependent manner.
Ruxoprubart is currently being evaluated in a Phase II clinical trial in treatment-naive patients with paroxysmal nocturnal hemoglobinuria, where it has demonstrated encouraging results to date. Beyond PNH, the therapy may also have potential across other clinical indications associated with dysregulation of the complement alternative pathway.
Kira Pharmaceuticals' KP104
Complement C5 inhibitors; Complement factor H inhibitors
KP104 is a first-in-class bifunctional biologic engineered to simultaneously inhibit the alternative complement pathway through Factor H and the terminal pathway through C5. By targeting both pathways, KP104 is designed to deliver a potent and synergistic approach to addressing established drivers of complement-mediated diseases. This dual mechanism differentiates KP104 from single-target complement therapies and may offer broader therapeutic benefits across complement-related disorders.
KP104 has been engineered for an extended half-life and increased potency, with a formulation that supports both intravenous and subcutaneous administration. The therapy is advancing into Phase II proof-of-concept studies across multiple renal and hematologic indications. The U.S. FDA has also granted KP104 Orphan Drug Designation for the treatment of paroxysmal nocturnal hemoglobinuria. Phase II clinical development is planned globally, including in the United States, China, and Australia. KP104 remains an investigational therapy and has not yet received regulatory approval for any indication from any health authority.
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NovelMed Therapeutics's NM5072
Properdin inhibitors
NovelMed is developing NM5072, a monoclonal antibody that has completed a Phase I clinical trial for paroxysmal nocturnal hemoglobinuria. Preclinical studies in animal models have demonstrated the therapy's potential across hematological, inflammatory, and ocular disease indications, while extensive evaluations have also been conducted using serum and blood samples from patients with PNH. NM5072 has received Orphan Drug Designation (ODD), and Phase II INDs have been approved for both hematological and renal indications.
Download the report to understand the top emerging therapies in PNH treatment @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials
Source: Paroxysmal Nocturnal Hemoglobinuria Market Report
Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PNH companies, including Alexion Pharmaceuticals, AstraZeneca, Apellis Pharmaceuticals, Novartis, Novo Nordisk, Regeneron Pharmaceuticals, NovelMed Therapeutics, ADARx Pharmaceuticals, Kira Pharmaceuticals, and others.
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