Odevixibat Improves Long-Term Quality of Life, Sleep in patients with Alagille Syndrome

Pooled long-term data from the phase 3 ASSERT (NCT04674761) and ASSERT-EXT (NCT05035030) studies showed that treatment with odevixibat led to sustained improvements in sleep and caregiver-reported quality of life (QoL) in patients with Alagille syndrome (ALGS). The results were presented at the 2025 North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) and authored by Nadia Ovchinsky, MD, Pediatric Gastroenterology and Hepatology, Hassenfeld Children's Hospital, NYU Langone, New York, NY, and colleagues.1

Odevixibat on Long-Term Improvements in Sleep and Quality of Life

Patients with Alagille syndrome frequently experience severe pruritus, disrupted sleep, and reduced quality of life. Odevixibat, a selective ileal bile acid transport inhibitor, has shown efficacy in reducing cholestatic pruritus in this population. In this pooled analysis, long-term outcomes from up to 96 weeks of treatment were evaluated for sleep parameters and caregiver-assessed QoL measures.

In the ASSERT trial, patients received 24 weeks of placebo or odevixibat 120 µg/kg/day; study completers were eligible for ASSERT-EXT, during which patients received odevixibat for an additional 72 weeks. Endpoints included observer-reported sleep parameters and caregiver-assessed Pediatric Quality of Life Inventory (PedsQL) scores.

A total of 52 patients with a median age of 5.7 years (1.0-15.5) received odevixibat across both studies.

“Patients had significant improvements in 6 of 7 Observer-Reported Outcome (ObsRO) sleep parameters from baseline (BL) up to weeks 93−96 and a numerical reduction in days taking medication to induce sleep," the study authors wrote. Caregiver-reported PedsQL scores “also showed numerical improvements from BL up to week 96 with odevixibat.”

READ MORE: Odevixibat Shows Sustained Pruritus and Bile Acid Improvements in FIC1 Deficiency

According to the authors, “Long-term odevixibat given for up to 96 weeks improved multiple sleep parameters and caregiver-reported QoL in patients with ALGS; data support long-term improvements in pruritus seen with odevixibat.”

Specifically, the following improvements in Mean ObsRo sleep parameters were demonstrated from baseline (n = 50) to week 93-96 (n = 31).

• Days seeing blood due to scratching: 35.2% at baseline vs 4.7% at week 93-96
• Days needing help falling asleep: 56.5% at baseline vs 8.8% at week 93-96
• Days with soothing: 64.4% at baseline vs 14.5% at week 93-96
• Days sleeping with caregiver: 43.2% vs 4.6%
• Number of awakenings: 3.5 at baseline vs 0.6 at week 93-96
• Days taking medication to sleep: 29.7% vs 18.1%
• Daytime tiredness (score): 2.1 at baseline vs 0.9 at week 93-966

Odevixibat received FDA approval to treat cholestatic pruritus in patients aged 12 months and older on June 13, 2023. Approval was based on the ASSERT data through 24 weeks, where more than 90% of patients were responders to pruritus. Additionally, no patients discontinued the study, and 96% of patients were rolled into the open-label extension.2

References:

1. Ovchinsky N, Aumar M, Baker A, et al. LONG-TERM QUALITY OF LIFE OUTCOMES FOR PATIENTS WITH ALAGILLE SYNDROME TREATED WITH ODEVIXIBAT: POOLED RESULTS FROM PHASE III, RANDOMIZED, DOUBLE-BLIND ASSERT AND OPEN-LABEL ASSERT-EXT STUDIES. Abstract. Presented at: 2025 NASPGHAN Annual Meeting. November 5-9, 2025. Chicago, Illinois.