Odevixibat shows promise in ALGS in pooled data analysis
The Phase 3 ASSERT and ASSERT-EXT trials demonstrated that odevixibat led to rapid and significant improvements in pruritus and reductions in bile acid levels by week 4 of treatment in patients with Alagille syndrome (ALGS). In addition, patients experienced significant enhancements in sleep parameters.
In the completed 24-week ASSERT study, patients with ALGS exhibiting significant pruritus and elevated serum bile acids were randomly assigned to receive either odevixibat (at a dosage of 120 µg/kg/day) or a placebo in a 2:1 ratio. Patients who completed the ASSERT trial were given the option to enroll in the ASSERT-EXT, an ongoing 72-week open-label extension study where all participants receive odevixibat at the same dosage.
Pooled data from these trials, which included all patients who received odevixibat, were analyzed, demonstrating that odevixibat treatment led to rapid and significant improvements in pruritus and reductions in bile acid levels by week 4. Moreover, patients experienced substantial enhancements in sleep parameters, including a reduction in the percentage of days experiencing blood due to scratching, decreased need for assistance falling asleep, less need for soothing, and a decrease in sleeping with a caregiver.
No patients exhibited concurrent elevations in alanine aminotransferase or total bilirubin indicative of drug-induced liver injury. Treatment-emergent adverse events were reported in 83% of odevixibat-treated patients, with diarrhea being the most common drug-related side effect (reported in 12% of patients).
Ovchinsky N, 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 NASPGHAN 2023.
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