Odevixibat effectively reduces pruritus, serum bile acids in children with PFIC

Odevixibat administered as once a day oral capsules, effectively reduced pruritus and serum bile acids compared with placebo in children with progressive familial intrahepatic cholestasis (PFIC), according to results of a phase 3 trial.

Patients were randomly assigned to placebo (n = 20), odevixibat 40 μg/kg per day (n = 23), or odevixibat 120 μg/kg per day (n = 19).

Mean proportion of positive pruritus assessments was 55% in the combined odevixibat group (58% in the 40 μg/kg per day group and 52% in the 120 μg/kg per day group) versus 30% in the placebo group. Serum bile acid response was also significantly higher with odevixibat versus placebo 14 of 42 patients in the combined odevixibat group (10 in the 40 μg/kg per day group and 4 in the 120 μg/kg per day group) versus none of 20 in the placebo group.

Treatment was generally well tolerated, with diarrhea or frequent bowel movements and fever the most common treatment-emergent adverse events (TAEs). Serious TEAEs occurred in 7% of odevixibat-treated patients and 25% placebo-treated patients.

Reference
Thompson RJ, Arnell H, Artan R, et al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Sep;7(9):830-842. doi: 10.1016/S2468-1253(22)00093-0. Epub 2022 Jul 1. PMID: 35780807.