[15] In the United States, odevixibat is indicated for the treatment of pruritus in people three months of age and older with progressive familial intrahepatic cholestasis.
[18] Odevixibat is metabolized through a process called mono-hydroxylation.The drug is primarily eliminated through the feces (97% unchanged), with a minimal amount excreted in the urine (0.002%).
[17] Common side effects of odevixibat include diarrhea, stomach pain, vomiting, liver test abnormalities, abnormal liquid function tests, and a deficiency in vitamins A, D, E and K.[21][17] There are no enough human data on odevixibat use during pregnancy to build a drug-associated risk of major birth defects, miscarriage, or adverse developmental outcomes.
A 24-week clinical trial, played a role in demonstrating the effectiveness and safety of odevixibat in treating pruritus in children with progressive familial intrahepatic cholestasis.
[21] Odevixibat was granted its initial approval in July 2021, in the European Union for the treatment of progressive familial intrahepatic cholestasis in people aged six months and older.
In July 2021, it received approval in the United States for the treatment of pruritus (itching) in people aged three months and older with progressive familial intrahepatic cholestasis.
[6][7] In July 2024, the CHMP adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Kayfanda, intended for the treatment of cholestatic pruritus in people with Alagille syndrome aged six months or older.
[8] A phase III randomized control trial showed odevixibat reduced pruritis and serum bile acids in children with progressive familial intrahepatic cholestasis.