FDA Approvals, Highlights, and Summaries: Gastroenterology

February 17, 2022

Bylvay (odevixibat)

Odevixibat is a first-in-class ileal bile acid transport inhibitor (IBATi). It acts locally in the distal ileum to decrease reuptake of bile acids and increase clearance of bile acids through the colon, thereby reducing bile acid serum concentration. It is indicated for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 3 months and older.

Approval was supported by data from the Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis (PEDFIC) types 1 and 2 phase 3 trials. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, odevixibat showed improvement of pruritus and serum bile acid compared with placebo. PEDFIC 2, a long-term, open-label phase 3 extension study, showed 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. AASLD The Liver Meeting 2021. 2021 Nov 12-15

Livmarli (maralixibat)

Maralixibat is another IBATi that gained approval for cholestatic pruritus in adults and children aged 1 year and older with Alagille syndrome. Approval was based on the ICONIC study and 5 years of data from supportive studies in 86 patients with Alagille syndrome. On average, patients who received maralixibat for 22 weeks maintained pruritus reduction, whereas those in the placebo group who were withdrawn from maralixibat after Week 18 returned to baseline pruritus scores by Week 22. Lancet. 2021 Oct 30;398(10311):1581-1592

Other gastroenterology approvals

Dartisla ODT (glycopyrrolate) - New oral disintegrating tablet for adults to reduce symptoms of peptic ulcer as an adjunct to treatment.

Zeposia (ozanimod) - New indication approved for adults with moderately to severely active ulcerative colitis.


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