Ultra Orphan Assessment

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework:

Indication under review: For the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.

Key points:

  • Progressive familial intrahepatic cholestasis (PFIC) is a rare heterogeneous group of life-threatening diseases characterised by the hepatotoxic accumulation of bile acids which causes severe pruritus. Many patients progress to cirrhosis, hepatocellular cancer, end-stage liver failure and require liver transplant. Odevixibat is the first medicine to receive a marketing authorisation for this condition.

  • Severe pruritus has a significant impact on quality of life and can cause cutaneous mutilation, sleep deprivation, irritability, poor attention, and impaired school performance. Caring for a child with PFIC has a significant impact on families and caregivers.

  • In the randomised, double-blind, phase III study PEDFIC1, treatment with odevixibat resulted in clinically relevant reductions in serum bile acids and pruritus compared with placebo.

  • There was a general improvement in quality of life in the odevixibat group compared with placebo in PEDFIC1.

  • There is currently no evidence that treatment with odevixibat reduces or delays biliary diversion surgery or liver transplant. Long-term efficacy and safety data are awaited.

  • Despite a Patient Access Scheme that improves the cost-effectiveness of odevixibat, the treatment’s cost in relation to its health benefits remains high.


From 11 July 2022 odevixibat can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated.  After 3 years the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.

Medicine details

Medicine name:
odevixibat (Bylvay)

For treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older

Pharmaceutical company
Albireo Pharma
BNF chapter
Gastro-intestinal system
Submission type
Ultra-orphan initial assessment
Date Published
11 July 2022
Additional notes

A new approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan webpage and in the Scottish Government’s guidance.