Скачать или смотреть Pivotal PFIC Clinical Trials Meet Primary Endpoints: FDA Submission Expected in Early 2021

Ron Cooper, President and CEO of Albireo Pharma discusses the results from two clinical trials assessing odevixibat to treat progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease due to an inability to properly bile acids from the liver. The most common manifestation of PFIC is pruritus, which often results in a severely diminished quality of life. In many cases, this disease leads to cirrhosis and liver failure within the first 10 years of life, and nearly all PFIC patients require treatment before the age of 30 years.

Mr. Cooper discusses the full results from the PEDFIC1 clinical trial as well as the interim results from the PEDFIC2 trial which were presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting held November 13-16, 2020.

PEDFIC1 is a global phase 3 clinical trial evaluating the efficacy and safety of odevixibat and is the largest study ever conducted in PFIC1 and PFIC2. PEDFIC1 met both of its primary endpoints, demonstrating that odevixibat improved pruritus assessments and reduced serum bile acid responses. Additionally, the results showed a high level of tolerability in patients receiving odevixibat.

PEDFIC2 is an open label extension study of PEDFIC1. The interim data for PEDFIC2 reaffirm odevixibat’s efficacy in improved pruritus assessments and reduced serum bile acid responses. Interim data also reaffirm the high tolerability in participants and sustained effect of the drug.

Based on these results, Albireo hopes to get all regulatory filings for FDA approval submitted by early 2021.

For more information about PFIC and other rare metabolic disorders, visit checkrare.com/diseases/metabolic-disorders/