Las Vegas – September 19, 2026 -- The paroxysmal nocturnal hemoglobinuria (PNH) market across the seven major markets was valued at approximately $1.5 billion in 2025 and is projected to grow at a compound annual growth rate of 7.1% through 2036, according to DelveInsight.
Regeneron's pozelimab-cemdisiran combination leads next wave of PNH therapies
Pozelimab, a fully human anti-C5 monoclonal antibody developed on Regeneron's VelocImmune platform, is expected to generate the highest revenue among emerging PNH therapies in the seven major markets. The drug is being tested in combination with Alnylam's siRNA therapy cemdisiran; Regeneron has said Phase III results in PNH are anticipated in the fourth quarter of 2026 or the first quarter of 2027.
Novo Nordisk's zaltenibart shows efficacy in ravulizumab-refractory patients
Zaltenibart (OMS906), a MASP-3 inhibitor, ranks second in projected revenue and demonstrated strong efficacy in a Phase II study among PNH patients with suboptimal responses to ravulizumab. Novo Nordisk acquired exclusive worldwide rights to the antibody from Omeros Corporation in November 2025 under an asset purchase and license agreement, and the therapy holds FDA Orphan Drug Designation.
NovelMed advances two complement-pathway candidates toward Phase II
Ruxoprubart (NM8074), an anti-Bb monoclonal antibody that selectively blocks the alternative complement pathway, completed Phase I testing in 40 healthy volunteers with dose-dependent, complete pathway inhibition and is now in Phase II trials among treatment-naive PNH patients. NM5072, a properdin inhibitor from the same company, has finished Phase I testing, received Orphan Drug Designation, and secured Phase II IND approvals for both hematological and renal indications.
Kira Pharmaceuticals' dual-pathway KP104 moves into global Phase II studies
KP104, a bifunctional biologic that inhibits both the alternative pathway via Factor H and the terminal pathway via C5, has received FDA Orphan Drug Designation for PNH and is entering Phase II proof-of-concept trials in the United States, China and Australia. The compound is formulated for both intravenous and subcutaneous administration and has not yet been approved by any regulator.
Standard of care shifts beyond terminal complement inhibition
Soliris (eculizumab), Ultomiris (ravulizumab) and Empaveli (pegcetacoplan) established complement inhibition as the PNH standard of care after eculizumab's 2007 introduction reduced hemolysis and thrombosis risk. Newer proximal inhibitors -- Fabhalta (iptacopan), Voydeya (danicopan) and Piasky (crovalimab) -- have since expanded treatment options to address limitations tied to breakthrough hemolysis and transfusion dependence.