Children with Alagille syndrome can experience itching severe enough to disrupt sleep and development, driven by bile acids the liver cannot clear. Livmarli (maralixibat) was the first therapy approved for it.
By blocking bile acid reabsorption in the distal intestine, it causes bile acids to be excreted rather than recirculated, lowering the levels that drive itch. The once-daily oral solution treats cholestatic pruritus in patients with Alagille syndrome.
Livmarli has since expanded into progressive familial intrahepatic cholestasis and holds orphan designation with no generic competition. It competes head-to-head with odevixibat across the same cholestatic indications, making age-range breadth and formulation the key differentiators.