Which treatments are under investigation for paroxysmal nocturnal hemoglobinuria (PNH)?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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A variety of agents that inhibit complement are under development for treatment of PNH. Novel anti-C5 agents include monoclonal antibodies (eg, crovalimab [32] ) and an anti-C5 small interfering RNA. [33] Because clinically relevant C3-mediated extravascular hemolysis can occur in PNH, [34]  (the C5 inhibitors eculizumab and ravulizumab reduce intravascular hemolysis only), the anti-C3 small peptide compstatin and its derivatives are being investigated. 

Inhibitors of complement Factor D or B are also being studied. [33]  Yuan et al reported that two novel small-molecule inhibitors of Factor D, which is a component of the alternative complement pathway, show potential as oral agents for treating PNH. In the Ham test, using cells from PNH patients, the Factor D inhibitors significantly reduced complement-mediated hemolysis at concentrations as low as 0.01 μM. In an animal model, the compound ACH-4471 blocked alternative pathway activity. [35]

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