ODEVIXIBAT

The PBAC recommended the PBS listing of odevixibat (Bylvay®), for the treatment of cholestatic pruritus (severe itching caused by bile acid build up) in people with Alagille syndrome (ALGS). 

The PBAC acknowledged input from consumers, healthcare professionals and organisations, describing the substantial burden of cholestatic pruritus in ALGS in this vulnerable population. The PBAC noted that ALGS is a rare condition and cholestatic pruritus can have severe impacts on quality of life, including sleep, comfort and wellbeing. 

The PBAC recalled that it previously noted there was evidence showing odevixibat can reduce itching for some patients with ALGS, however it does not treat the underlying disease, and the long-term benefits remain uncertain. The PBAC considered that a higher price than previously recommended could be supported; however, a price reduction from that proposed in the resubmission would still be required to ensure odevixibat was cost effective in this population. The PBAC considered the estimated number of patients likely to be treated was reasonable however a risk sharing arrangement would be required to manage uncertainty regarding the average cost per patient per year (as each dose of odevixibat should be administered according to body weight which will impact the average cost per patient per year).