Odevixibat for treating progressive familial intrahepatic cholestasis

Odevixibat (Bylvay, Albireo Pharma) is a selective inhibitor of the ileal bile acid transporter (IBAT). IBAT is involved in the absorption of bile acids in the small intestine for circulation back to the liver. Odevixibat stops the recycling of bile acids, increasing their excretion through the colon and lowering hepatic and serum bile acid levels. It has a marketing authorisation under 'exceptional circumstances' for 'the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older'.

Odevixibat is administered daily as a capsule or sprinkled on food. The starting dose is 40 micrograms/kg/day. After 3 months of continuous therapy, the dose may be escalated to 120 micrograms/kg/day if there has not been an adequate clinical response.

The adverse reactions listed in the summary of product characteristics for odevixibat include: diarrhoea, abdominal pain, soft stools and hepatomegaly (an enlarged liver). For full details of adverse reactions and contraindications, see the summary of product characteristics for odevixibat.

Odevixibat is available as a pack of 30 capsules. The cost per pack of 200 microgram capsules is £3,085, per pack of 400 microgram capsules is £6,170, per pack of 600 microgram capsules is £9,255 and per pack of 1,200 microgram capsules is £18,510 (excluding VAT; company's evidence submission). The company has a commercial arrangement. This makes odevixibat available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.