Odevixibat is effective in reducing symptoms typically associated with Alagille syndrome (ALGS), such as intractable pruritus and quality of sleep; in addition, it is generally well-tolerated, according to a study published in the Journal of Hepatology.

One of the characteristic features of ALGS is the accumulation of bile acids in the liver, which then seeps into the systemic circulation. The most common symptoms of excessive bile acid storage is pruritus that is often described as severe, as well as sleep impairment. 

Intractable pruritus and insomnia are 2 medical complaints that can cause significant distress. Some patients with severe pruritus scratch their skin so hard that it bleeds. Difficulties in sleeping can reduce the quality of social interactions, as well as performance at work. These issues, if left unresolved, can significantly affect a patient’s health-related quality of life. 

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Odevixibat is an ileal bile acid transporter inhibitor that was developed to primarily help with 3 issues associated with ALGS: pruritus, sleeping difficulties, and abnormally raised bile acid levels. The use of odevixibat relative to its performance in these 3 parameters, as well as its overall safety and tolerability, were the study goals of the phase 3 ASSERT and ASSERT-EXT trials. 

Read more about ALGS etiology 

Ovchinsky and colleagues sought to use pooled data from the studies to investigate the efficacy and safety of odevixibat in achieving its treatment goals. The research team focused on a 24-week study that recruited patients with ALGS and a history of significant pruritus and high serum bile acid levels. These patients were then randomized to receive 120 mcg/kg of odevixibat per day or placebo in a 2:1 manner. Patients completing this phase of the study had the option of being recruited to the 72-week, open-label extension portion of the study, during which all patients received 120 mcg/kg of odevixibat per day. 

The results indicate that patients treated with odevixibat experienced a “rapid and significant” mean improvement in pruritus and a decrease in bile acid levels. Patients also demonstrated improvement across a number of sleep parameters, such as ease of falling asleep and whether the help of a caregiver was needed. Odevixibat was generally safe and well-tolerated in this patient population. 

If further studies confirm these findings, odevixibat may be useful in treating some of the more severe symptoms of ALGS. 


Ovchinsky N, Aumar M, Baker A, et al. Efficacy and safety outcomes with odevixibat treatment: pooled data from the phase 3 ASSERT and ASSERT-EXT studies in patients with Alagille syndrome. Presented at: European Association for the Study of the Liver (EASL) Congress 2023, Vienna, Austria; June 21-24. Abstract THU-284.