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Home > Drugs > Treatments > Cholestatic Pruritus in Alagille Syndrome

Medications for Cholestatic Pruritus in Alagille Syndrome

Alagille syndrome (ALGS) is a rare genetic disorder characterized by abnormally narrow, malformed, or reduced numbers of bile ducts in the liver, which leads to the accumulation of bile acids. It is caused by mutations in the JAG1 and NOTCH2 genes.

When bile acids accumulate in the liver, the liver cannot work properly to eliminate waste from the bloodstream. This may eventually progress to liver disease serious enough to require a liver transplant. Alagille syndrome can also affect the heart, kidneys, and central nervous system.

Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and severe cholestatic pruritus (itch). 

Cholestatic pruritus associated with Alagille syndrome can be serious, unremitting, and debilitating. Patients can suffer from sleep disruption, and bleeding and scarring of the skin due to constant scratching.

Livmarli (maralixibat) and Bylvay (odevixibat) are FDA-approved medications to treat cholestatic pruritus associated with Alagille syndrome. They work by inhibiting ileal bile acid transporter (IBAT) to reduce serum bile acid levels.

Drugs used to treat Cholestatic Pruritus in Alagille Syndrome

Name Drug Class Updated
Maralixibat Miscellaneous GI agents 16-Aug-2023
Livmarli Miscellaneous GI agents 05-Aug-2023
Bylvay Miscellaneous GI agents 25-Aug-2023
Bylvay Miscellaneous GI agents 13-Jul-2023
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