Alagille Syndrome
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Odevixibat shows promise in alleviating symptoms of Alagille syndrome: Phase 3 ASSERT study results

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The phase 3 ASSERT study has demonstrated the effectiveness and safety of odevixibat in treating Alagille syndrome (ALGS), according to a poster presented at The Liver Meeting 2023.

Patients who responded positively to odevixibat showed significant reductions in bile acids, improved pruritus symptoms, enhanced sleep parameters, and better quality of life compared to baseline.

The study, which included 52 patients with a confirmed diagnosis of ALGS, history of significant pruritus, and elevated serum bile acids, randomized participants 2:1 to receive odevixibat (n = 35) or placebo (n = 17). Caregivers utilized the PRUCISION observer-reported outcome (ObsRO) tool to assess patient pruritus and daytime tiredness.

Notably, 80% of patients treated with odevixibat were classified as pruritus responders, defined as those achieving a ≥1-point decrease in ObsRO scratching score from baseline to weeks 21–24. This group exhibited remarkable improvements in pruritus, with mean scratching scores decreasing from 2.8 at baseline to 0.9 at weeks 21–24.

Alongside the alleviation of pruritus, odevixibat-treated pruritus responders showed clinically meaningful reductions in bile acids, improved sleep parameters, and enhanced Pediatric Quality of Life Inventory (PedsQL) scores compared to baseline.

Among the odevixibat-treated pruritus responders, 75% reported treatment-emergent adverse events (TEAEs), the majority of which were mild or moderate in severity. Importantly, no TEAEs led to study discontinuation. One patient with enteroviral gastroenteritis experienced serious, drug-related TEAEs (hematemesis and international normalized ratio increased), which resolved after 2 days without necessitating a change in odevixibat dosage.

Wehrman A, et al. Outcomes with odevixibat treatment in patients with Alagille syndrome: Analysis of pruritus responders from the phase 3 ASSERT study. Presented at: The Liver Meeting 2023.

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).