Odevixibat, or A4250, is an ileal sodium/bile acid cotransporter inhibitor indicated for the treatment of pruritus in patients older than 3 months, with progressive familiar intrahepatic cholestasis (PFIC). Odevixibat is the first approved non-surgical treatment option for PFIC. Previous therapies for PFIC included a bile acid sequestrant such as ursodeoxycholic acid.
Odevixibat was granted FDA approval on 20 July 2021.
Odevixibat is indicated for the treatment of pruritus in patients older than 3 months with progressive familiar intrahepatic cholestasis (PFIC) and cholestatic pruritus in patients 12 months of age and older with Alagille Syndrome. It may not be effective in patients with PFIC type 2 with ABCB11 variants since these patients lack a functional bile salt export pump.
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