Odevixibat in progressive familial intrahepatic cholestasis: a profile of its use
Funding The preparation of this review was not supported by any external funding.
Authorship and Conflict of interest Young-A Heo, a salaried employee of Adis International Ltd/Springer Nature and an editor of Drugs & Therapy Perspectives, was not involved in any publishing decisions for the manuscript and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.
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Odevixibat (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 parameters 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 being mild or moderate in severity. Longer-term safety data (up to 72 weeks of treatment) revealed no new safety concerns.
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