New anti-complement drugs: not so far away

Blood. 2014 Mar 27;123(13):1975-6. doi: 10.1182/blood-2014-02-555805.


In this issue of Blood, Risitano et al demonstrate that small-molecule inhibitors of C3 cleavage prevent complement activation on erythrocytes from patients with paroxysmal nocturnal hemoglobinuria (PNH); the authors demonstrate that these agents reach therapeutic concentrations after subcutaneous injection in nonhuman primates.

Publication types

  • Comment

MeSH terms

  • Animals
  • Complement Activation / drug effects*
  • Complement C3 / antagonists & inhibitors*
  • Hemoglobinuria, Paroxysmal / drug therapy*
  • Humans
  • Peptides, Cyclic / therapeutic use*


  • Complement C3
  • Peptides, Cyclic
  • compstatin