Progressive familial intrahepatic cholestasis (PFIC)

Active Ingredient: Maralixibat

Indication for Maralixibat

Population group: only minors (0 - 18 years old) , adults (18 - 65 years old)
Therapeutic intent: Curative procedure

Maralixibat is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients 3 months of age and older.

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

285 ug/kg once daily that may be increased to 285 ug/kg 1-2 times daily after 1-2 weeks, and after 1-2 weeks the dose can be increased to 570 ug/kg twice daily

For:

Dosage regimens

In case that patient age in months is ≥ 3, oral, 285 micrograms maralixibat, once daily, over the duration of 1 to 2 weeks. Afterwards, in case that patient age in months is ≥ 3, oral, 285 micrograms maralixibat, 1 to 2 times daily, over the duration of 1 to 2 weeks. Afterwards, in case that patient age in months is ≥ 3, oral, between 285 micrograms maralixibat and 570 micrograms maralixibat, 1 to 2 times daily.

Detailed description

The starting dose is 285 mcg/kg once daily (QD) and may be increased after 1-2 weeks to 285 mcg/kg twice daily (BID, morning and evening). After 1-2 weeks, the dose can be increased to 570 mcg/kg twice daily if clinically indicated, as tolerated. In case of poor tolerability, dose reduction or treatment interruption should be considered. Renewed dose-escalation can be attempted as tolerated.

Alternative treatment should be considered in patients for whom no treatment benefit can be established following 3 months of continuous daily treatment with maralixibat.

Missed dose

If a dose is missed, the dose should be omitted, and the original dose schedule resumed with the next scheduled intake.

Dosage considerations

Maralixibat is administered orally via an oral syringe by a caregiver or the patient, before (up to 30 minutes) or with a meal, in the morning for once daily dosing, or in the morning and evening for twice daily dosing.

Active ingredient

Maralixibat

Maralixibat is a minimally absorbed, reversible, potent, selective inhibitor of the ileal bile acid transporter (IBAT). Maralixibat acts locally in the distal ileum to decrease the reuptake of bile acids and increase the clearance of bile acids through the colon, reducing the concentration of bile acids in the serum.

Read more about Maralixibat

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