A phase 3 clinical trial and its open-label extension study have shown that the bile acid inhibitor odevixibat can decrease serum bile acid levels and improve pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The study found that odevixibat provided significant benefits across various measures of PFIC, including patient sleep and quality of life. The analysis included data from pediatric patients with different subtypes of PFIC who were treated with odevixibat. Researchers observed a reduction in serum bile acid levels and improvement in pruritus scores in all patient populations. Patients with different subtypes of PFIC experienced varying levels of treatment efficacy, but overall, odevixibat was associated with improved sleep parameters and quality of life. The most common treatment-emergent adverse events reported were pyrexia and diarrhea.
This study contributes to the growing evidence of the clinical benefits of odevixibat in the treatment of cholestatic conditions. The findings suggest that odevixibat may be effective in managing PFIC symptoms, including elevated serum bile acids, liver damage, and intractable pruritus. Odevixibat treatment was associated with decreased serum bile acid levels, improved pruritus scores, and enhanced sleep and quality of life measures in patients with PFIC. However, the magnitude of the efficacy response varied across different subgroups of patients with PFIC. Overall, the results support the potential use of odevixibat as a treatment option for patients with PFIC.