Iptacopan

How it works

It selectively inhibits factor B in the alternative complement pathway, reducing both intravascular and extravascular haemolysis of complement-sensitised red cells.

Prescribing in practice

• Because complement inhibition raises the risk of serious infection by encapsulated organisms, meningococcal vaccination is required before starting and antibiotic prophylaxis may be needed.
• Vaccination against pneumococcal and Haemophilus influenzae type b infection is also advised in line with national guidance.
• Counsel patients to seek urgent care for symptoms suggesting meningococcal infection given the risk of rapid deterioration.

Monitoring

Monitor haemoglobin and markers of haemolysis to assess response, and remain vigilant for signs of serious infection throughout treatment.

Counselling the patient

• Carry your patient safety card and seek urgent medical help for fever, severe headache or neck stiffness.
• Ensure your meningococcal and other recommended vaccinations are up to date.
• Do not stop treatment suddenly without discussing it with your team.

Evidence & guidelines

Iptacopan is recommended within its licensed use for paroxysmal nocturnal haemoglobinuria, supported by the APPLY-PNH and APPOINT-PNH trials.