Pegcetacoplan

How it works

It binds complement protein C3 and its activation fragment C3b, providing broad control of the complement cascade and limiting both intravascular and extravascular haemolysis of PNH red cells.

Prescribing in practice

• Because complement inhibition increases susceptibility to encapsulated organisms, vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type b is required before starting, with antibiotic cover if treatment cannot await vaccination.
• On switching from a C5 inhibitor, regimens overlap to reduce the risk of haemolysis, and abrupt discontinuation can precipitate haemolysis requiring close monitoring.
• It is given by subcutaneous infusion, typically via a pump, in accordance with the SPC.

Monitoring

Monitor haemoglobin, lactate dehydrogenase, reticulocytes and signs of haemolysis, and remain vigilant for symptoms of serious infection both during treatment and after any interruption.

Counselling the patient

• Stress the importance of staying up to date with the required vaccinations and carrying a patient safety card.
• Advise seeking urgent medical attention for fever or other features suggesting serious infection, and not stopping treatment without specialist advice.

Evidence & guidelines

The PEGASUS trial showed pegcetacoplan improved haemoglobin compared with eculizumab in paroxysmal nocturnal haemoglobinuria.