According to a recent press release from Globe Newswire, Albireo Pharma announced that Lancet Gastroenterology published results of the PEDFIC 1 trial evaluating Bylvay® in the treatment of progressive familial intrahepatic cholestasis (PFIC). It is now the largest trial ever held evaluating PFIC.
PFIC causes impaired bile flow (cholestasis) which, when it accumulates in the liver cells, results in liver disease. The symptoms may include intense itching (pruritus), delayed growth, sleep problems, and poor quality of life.
About the Trial
The study, phase 3 PEDFIC 1, is a global study, double-blind, randomized, and placebo-controlled.
The study involved sixty-two evaluable patients ages six months to fifteen years. The patients had PFIC type I or type II. PEDFIC 1 took place from May 2018 through July 2020.
The U.S. FDA has now approved Bylvay to treat pruritus in patients who are three months or older for all forms of PFIC. European guidelines are currently six months and older. These guidelines are based on the PEDFIC I study and open-label extension PEDFIC II study.
The primary endpoints were (a)proof that Bylvay significantly improved pruritus severity and (b) serum bile acid level reduction together with rapid and sustained consistent results.
The study met both endpoints. Notably, pruritus symptoms were improved by the fourth week of treatment. Bylvay’s adverse events (AEs) were in line with the placebo. No drug-related (AEs) were noted.
The PEDFIC 1 study may be accessed on The Lancet Gastroenterology & Hepatology website.
Bylvay (odevixibat) is an ileal bile acid transporter inhibitor. It is non-systemic, meaning it is not system-wide and is active locally in the small intestine..
Note that Blvay is sometimes not effective for use for PFIC type II patients who have ABCBII variants that result from the lack of bile salt export pump protein.
The list of the drug’s adverse reactions are abnormalities in liver tests, diarrhea, vomiting, deficiency in fat-soluble vitamins, and abdominal pain.
Bylvay is now available in the UK and Germany and after reimbursement and pricing approval other European countries are slated to follow.