The PNH treatment landscape, historically limited, has evolved significantly since the 2007 introduction of eculizumab. While current standard-of-care therapies like Soliris, Ultomiris, and Empaveli rely on complement inhibition, safety concerns and the need for more effective disease control have spurred innovation. Recent approvals, including iptacopan, danicopan, and crovalimab, represent the first wave of this transition toward proximal complement inhibition.

DelveInsight forecasts a 7.1% compound annual growth rate for the PNH market through 2036. The next phase of development focuses on five key candidates: Regeneron’s Pozelimab combined with Cemdisiran, Novo Nordisk’s Zaltenibart (formerly OMS906), NovelMed’s Ruxoprubart (NM8074), Kira Pharmaceuticals’ KP104, and NovelMed’s NM5072. Among these, Pozelimab is currently projected to capture the highest revenue share in major markets. These candidates aim to address persistent challenges such as breakthrough hemolysis and transfusion dependence, offering diverse mechanisms ranging from MASP-3 inhibition to bifunctional Factor H and C5 targeting.