odevixibat (Bylvay)

following a reassessment through the ultra-orphan framework:

odevixibat (Bylvay®) is accepted for use within NHSScotland.

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

In a phase III study, odevixibat significantly improved serum bile acid response and reduced pruritus symptoms at 24 weeks compared with placebo in paediatric patients with PFIC.

Reductions in serum bile acid concentration and pruritus symptoms were maintained for at least 72 weeks in an open-label extension study.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.