Topline results of the phase 3 ASSERT study evaluating the safety and efficacy of odevixibat (Bylvay®) in patients with Alagille syndrome (ALGS) aged 0 to 17 years confirmed reductions in the primary endpoint of pruritus and the secondary endpoint of serum bile acid levels. Complete results will be presented at an upcoming scientific meeting.
“The robust results from the ASSERT phase 3 trial are important because physicians urgently need more options in the treatment of ALGS. Bylvay reduced the devastating pruritus, which is so common among this patient population and critical to helping us improve sleep, among other burdens of the disease,” explained the principal investigator of the study, Nadia Ovchinsky, MD. “The study also showed Bylvay reduced serum bile acid levels, which could indicate that Bylvay may diminish the severity of liver disease over time, an important consideration for me as a treating physician.”
The treatment arm showed statistically significant reductions in pruritus from baseline at month 6 (weeks 21-24) and in serum bile acid concentration from baseline to the average of weeks 20 and 24 when compared to the placebo arm. Moreover, patients on odevixibat experienced improvements in multiple sleep parameters as early as weeks 1 to 4, with continued improvement through week 24.
The incidence of overall adverse events was similar between the treatment and placebo arms, suggesting that odevixibat was well tolerated, with low rates of drug-related diarrhea (11.4% vs 5.9% with placebo). No patient discontinued the study.
Read more about ALGS clinical trials
Given the positive results, the company will work on submitting regulatory filings in the US and EU.
Odevixibat is a nonsystemic ileal bile acid transport inhibitor that acts locally in the small intestine. Systemic exposure is residual. It is currently approved in the US to treat pruritus in patients aged 3 months and older and in Europe in patients aged 6 months and older for all types of progressive familial intrahepatic cholestasis.
Albireo reports positive topline data from phase 3 trial of Bylvay® (odevixibat) in Alagille syndrome. News release. Albireo Pharma, Inc,; October 11, 2022.