Mirum Pharmaceuticals, Inc. announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has recommended public reimbursement for LIVMARLI® (maralixibat oral solution) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS), with certain conditions. This announcement follows Health Canada?s authorization of LIVMARLI in this indication in 2023. The CADTH recommendation is based on data from the pivotal ICONIC study including six years of data across the LIVMARLI clinical program resulting in a robust body of evidence in patients with cholestatic pruritus in ALGS.

Data from ICONIC demonstrated statistically significant and clinically meaningful reductions in pruritus compared to placebo, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment. LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. and Europe, and for Progressive Familial Intrahepatic Cholestasis (PFIC) in the U.S. and in Europe. ALGS is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease.

Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly. Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS. LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and progressive familial intrahepatic cholestasis (PFIC) five years of age and older.

LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.