News
Positive results from PEDFIC 1 trial of odevixibat to treat progressive familial intrahepatic cholestasis.- Albireo Pharma Inc.
Albireo Pharma, Inc. announced positive topline results from PEDFIC 1, a global Phase III clinical trial evaluating the efficacy and safety of odevixibat and the largest study ever conducted in PFIC1 and PFIC2.
PEDFIC 1 met its two primary endpoints, demonstrating that odevixibat reduced serum bile acid responses (sBAs) (p=0.003) and improved pruritus assessments (p=0.004) with a single digit diarrhea rate. Odevixibat is a highly potent, non-systemic ileal bile acid transport inhibitor (IBATi), for the treatment of progressive familial intrahepatic cholestasis (PFIC) patients.
In the primary analysis , the study met the U.S. regulatory primary endpoint with the proportion of positive pruritus assessments being 53.5% in the odevixibat arms compared to 28.7% in the placebo arm (p=0.004).As a secondary endpoint 42.9% of patients in the odevixibat arms had a clinically meaningful improvement in the pruritus score, defined as a drop from baseline of 1.0 point or more on the 0-4 point scale, at week 24 compared to 10.5% in the placebo arm (p=0.018). The study also met the EU regulatory primary endpoint with 33.3% of subjects in the odevixibat arms experiencing either a 70% reduction in sBAs or reaching a level of 70 ?mol/L compared to no patients in the placebo arm (p=0.003). As a secondary endpoint, mean reduction of bile acids was 114.3 µmol/L in the odevixibat arms compared to an increase of 13.1 µmol/L in the placebo arm (p=0.002). Both doses of odevixibat were statistically significant for each of the endpoints.
Odevixibat was well tolerated, with an overall adverse event incidence similar to placebo. There were no drug-related serious adverse events (SAEs) reported during the study. Diarrhea/frequent bowel movements were the most common treatment-related gastrointestinal adverse events which occurred in 9.5% of odevixibat treated patients vs. 5.0% of placebo patients. Full results from the Phase III clinical trial will be presented at a future scientific meeting.
Condition: Familial Intrahepatic Cholestasis
Type: drug