Odevixibat in progressive familial intrahepatic cholestasis: a profile of its use

AbstractOdevixibat (Bylvay™; Bylvay®) has been approved for progressive familial intrahepatic cholestasis (PFIC) in patients aged ≥ 3 months in the USA and those aged ≥ 6 months in the EU and UK. In the pivotal double-blind placebo-controlled phase 3 PEDFIC 1 study, odevixibat 40 or 120 μg/kg once daily significantly reduced serum bile acids and pruritus. Odevixibat also improved growth, sleep and quality of life para meters compared with placebo in patients with PFIC. The clinical benefits of odevixibat were maintained with continued treatment for up to 72 weeks, according to an interim analysis of the ongoing open-label phase 3 PEDFIC 2 study. Odevixibat is generally well tolerated, with most adverse events bei ng mild or moderate in severity. Longer-term safety data (up to 72 weeks of treatment) revealed no new safety concerns.
Source: Drugs and Therapy Perspectives - Category: Drugs & Pharmacology Source Type: research