Paroxysmal nocturnal hemoglobinuria

Active Ingredient: Iptacopan

Indication for Iptacopan

Population group: only adults (18 years old or older)

Therapeutic intent: Curative procedure

Iptacopan is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

For this indication, competent medicine agencies globally authorize below treatments:

200 mg twice daily

For:

Dosage regimens

Oral, 200 milligrams iptacopan, 2 times daily.

Detailed description

The recommended dose is 200 mg taken orally twice daily.

Healthcare professionals should advise patients with PNH about the importance of adherence to the dosing schedule in order to minimise the risk of haemolysis.

If a dose or doses are missed, the patient should be advised to take one dose as soon as possible (even if it is shortly before the next scheduled dose) and then to resume the regular dosing schedule. Patients with several consecutive missed doses should be monitored for potential signs and symptoms of haemolysis.

PNH is a disease that requires chronic treatment. Discontinuation of this medicinal product is not recommended unless clinically indicated.

Patients switching from anti-C5 (eculizumab, ravulizumab) or other PNH therapies to iptacopan

To reduce the potential risk of haemolysis with abrupt treatment discontinuation:

For patients switching from eculizumab, iptacopan should be initiated no later than 1 week after the last dose of eculizumab.
For patients switching from ravulizumab, iptacopan should be initiated no later than 6 weeks after the last dose of ravulizumab.

Switches from complement inhibitors other than eculizumab and ravulizumab have not been studied.

Dosage considerations

It may be taken with or without food.

Active ingredient

Iptacopan
Iptacopan is a proximal complement inhibitor that targets Factor B (FB) to selectively inhibit the alternative pathway. Inhibition of FB in the alternative pathway of the complement cascade prevents the activation of C3 convertase and the subsequent formation of C5 convertase to control both C3-mediated extravascular haemolysis (EVH) and terminal complement-mediated intravascular haemolysis (IVH). Read more about Iptacopan

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