Odevixibat on the PBS

Odevixibat has recently been added to the Pharmaceutical Benefits Scheme (PBS) for the treatment of progressive familial intrahepatic cholestasis (PFIC). PFIC is a collection of rare genetic conditions of impaired bile formation and secretion. Signs and symptoms of cholestasis typically begin in infancy and can progress to liver failure.

Odevixibat acts locally in the distal ileum to inhibit the ileal bile acid transporter (IBAT). This reduces the reabsorption of bile acids, thereby increasing their clearance from the body.

The safety and efficacy of odevixibat was evaluated in a double-blind study of children with PFIC who had pruritus and elevated serum bile acids at baseline. Patients were randomly assigned to receive odevixibat (40 mcg/kg or 120 mcg/kg daily) or placebo for 24 weeks. Compared to placebo, odevixibat was associated with statistically significant improvements in pruritus. A serum bile acid response was observed in 33% of patients in the odevixibat group, while no patients met the response threshold in the placebo group. Data from the PEDFIC 2 extension study suggest these benefits are sustained with ongoing treatment.

Odevixibat is typically dosed once daily in the morning. The capsules may be swallowed whole, or the contents sprinkled on food. Odevixibat is minimally absorbed, and the most common adverse events are diarrhoea or frequent bowel movements.