NICE recommends odevixibat for treating progressive familial intrahepatic cholestasis.
Odevixibat from Albireo is NICE recommended, within its marketing authorisation, as an option for treating progressive familial intrahepatic cholestasis (PFIC) in people 6 months and older. It is recommended only if the company provides odevixibat according to the commercial arrangement.
PFIC is a rare and serious genetic condition that reduces or stops the flow of bile acids from the liver. This can cause severe pruritus (itching), poor growth and liver damage. PFIC severely affects the quality of life of people with the condition, and of their families and carers. It is fatal if untreated. Current treatment includes medicines not licensed for this condition (off label), then surgery such as an operation called partial external biliary diversion (PEBD) and, finally, a liver transplant.
Results from clinical trials suggest that, in people with the PFIC types 1 and 2, odevixibat reduces bile acid levels in the blood and pruritus compared with placebo (with or without off-label medicines). There is limited data for other types of PFIC. The clinical effectiveness of odevixibat when using the dose escalation schedule that would be used in NHS practice compared with PEBD is also uncertain.
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