The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for odevixibat (Albireo Pharma) for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
Odevixibat is a non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine. The Company believes that inhibition of IBAT could lead to a reduction in bile acids returning to the liver.
The NDA submission is supported by efficacy and safety data from two phase 3 studies (PEDFIC 1 and PEDFIC 2). PEDFIC 1 is a double-blind, placebo-controlled study that included 62 patients aged 6 months to 18 years with PFIC. Patients were randomized to receive odevixibat 40mcg/kg or 120mcg/kg orally once daily for 24 weeks or placebo. The coprimary end points were change in pruritus and bile acid reduction.
Results from PEDFIC 1 showed that both doses of odevixibat were associated with statistically significant reductions in pruritus symptoms (P =.004) and serum bile acids (P =.003) over 24 weeks compared with placebo. Odevixibat was found to be well tolerated with no drug-related serious adverse events reported. The most common treatment-related adverse events were diarrhea/frequent bowel movements (9.5% vs 5.0% for placebo).
Additionally, the long-term, open-label extension PEDFIC 2 study is currently evaluating the efficacy and safety of odevixibat in patients with PFIC who successfully completed the PEDFIC 1 study. Interim results demonstrated sustained reductions in pruritus and serum bile acid over 48 weeks.
“With regulatory submissions completed in record time and now accepted for review by both the FDA and EMA, odevixibat has the potential to become the first approved drug treatment for patients with PFIC,” said Ron Cooper, President and CEO of Albireo. A Prescription Drug User Fee Act (PDUFA) target date of July 20, 2021 has been set for this application.
The Company is also investigating odevixibat for the treatment of patients with biliary atresia in the phase 3 BOLD study as well as for the treatment of Alagille Syndrome in the phase 3 ASSERT study.
1. Albireo announces U.S. FDA acceptance of New Drug Application for odevixibat. [press release]. Boston, MA: Albireo Pharma, Inc; January 25, 2021.
2. New phase 3 data at AASLD show durable response to odevixibat in a rare pediatric liver disease. [press release]. Boston, MA: Albireo Pharma, Inc; November 13, 2020.