The Food and Drug Administration (FDA) has approved odevixibat (odevixibat), the first drug approved for the treatment of progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease due to an inability to properly remove 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 40 years. Prior to the approval of odevixibat, no drug therapies were approved for PFIC.

The approval of odevixibat was supported by data from the phase 3 PEDFIC 1 and PEDFIC 2 trials. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, odevixibat met both its pruritus and serum bile acid primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label extension study, reaffirmed that odevixibat delivered sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth, and other markers of liver function in patients treated up to 48 weeks. Across both studies, odevixibat was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious treatment-related adverse events.

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