Maralixibat-to-Odevixibat Switch Linked to Pruritus Improvement in ALGS

Pruritus improved in 3 adolescents with Alagille syndrome (ALGS) after switching from maralixibat to odevixibat, according to preliminary findings from a retrospective chart review presented at The Liver Meeting 2026 in Denver. Gastrointestinal adverse events reported during odevixibat treatment were transient and resolved without discontinuation by the data cutoff.¹

Nadia Ovchinsky, MD, director of the Division of Pediatric Gastroenterology and Hepatology in the Department of Pediatrics at NYU Grossman School of Medicine, discussed the findings in an interview with HCP Live. She emphasized that intolerance or inadequate response to one ileal bile acid transporter (IBAT) inhibitor may not rule out benefit from another in selected patients.

The multicenter review in France and the US had enrolled 5 patients by March 25, 2026; this interim analysis focused on 3 adolescents. Eligible patients had genetically confirmed ALGS and received each medication for ≥4 weeks.¹

Two adolescents discontinued maralixibat because of gastrointestinal adverse events, and 1 because of poor adherence with uncontrolled pruritus. After switching, all 3 experienced itch improvement. Diarrhea and abdominal pain each occurred in 2 patients receiving odevixibat, but these events resolved without treatment discontinuation.¹

The findings add preliminary switching experience to randomized evidence from the ASSERT clinical trial (NCT04674761), which demonstrated improvements in scratching scores and serum bile acids with odevixibat versus placebo. ASSERT did not evaluate switching from maralixibat.²

Ovchinsky highlighted adolescence as a vulnerable period, when chronic symptoms can compound school and social pressures. She said adult hepatologists assuming care should understand IBAT inhibitor adverse effects, dose optimization, and the potential role of switching in selected cases.

However, this uncontrolled, retrospective analysis of 3 adolescents cannot establish comparative efficacy or tolerability. Ovchinsky called for individualized treatment strategies and longer follow-up to determine whether targeted therapies affect liver disease prognosis beyond symptom control, alongside defining their long-term safety profiles.

Ovchinsky had no relevant financial disclosures of note.

References

1. Ovchinsky N, Ruiz M, Tiret A, et al. Real-world experience with switching to odevixibat from maralixibat in patients with Alagille syndrome: a retrospective chart review. Presented at: The Liver Meeting 2026; Denver, CO.
2. Ovchinsky N, Aumar M, Baker A, et al. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024;9(7):632-645. doi:10.1016/S2468-1253(24)00074-8