Which medications in the drug class Complement Inhibitors are used in the treatment of Paroxysmal Nocturnal Hemoglobinuria?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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Complement Inhibitors

Pegcetacoplan binds to complement protein C3 and its activation fragment C3b, thereby regulating C3 cleavage and generation of downstream effector of complement activation.

Two other monoclonal antibodies (ravulizumab and eculizumab) bind downstream in the complement cascade to C5 with high affinity inhibit cleavage to C5a (proinflammatory anaphylatoxin) and C5b (initiating subunit of the terminal complement complex [C5b-9]). The prevents generation of the terminal complement complex C5b9. This action inhibits terminal complement-mediated intravascular hemolysis in patients with PNH.

All three of these agents carry Black Box warnings regarding increased risk of meningococcal infection. Because of that risk, they are available only through a restricted program, under a Risk Evaluation and Mitigation Strategy, which providers must enroll in.

Pegcetacoplan (Empaveli)

Indicated for paroxysmal nocturnal hemoglobinuria (PNH). Administered as a twice weekly SC infusion. After proper training in SC infusion, the patient/care-giver may administer, if the healthcare provider deems appropriate. 

Ravulizumab (Ultomiris)

Indicated for paroxysmal nocturnal hemoglobinuria (PNH). Long-acting IV product that allows for every 8 week dosing starting 2 weeks after a single loading dose.

Eculizumab (Soliris)

Indicated for treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. After weekly IV loading doses over 5 weeks, maintenance doses are administered every 2 weeks.

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