Mirum Pharmaceuticals has announced that Health Canada has authorised the tablet formulation of Livmarli (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS). The approval adds to the previously authorised 9.5 mg/mL oral solution available in Canada.

Livmarli was earlier approved in Canada as an oral solution for patients aged 12 months and older with ALGS. The newly authorised tablet formulation is designed for patients weighing 22 kg or more who are able to swallow tablets, offering an additional dosing option and greater flexibility as patients grow older.

Jamie Twiselton, General Manager, Mirum Pharmaceuticals Canada, said the tablet formulation enhances treatment flexibility by providing a convenient one tablet per dose option for older patients while maintaining the liquid formulation for younger children, supporting continuity of care.

Alagille syndrome is a rare genetic disorder affecting multiple organ systems, particularly the liver, where bile duct abnormalities lead to bile accumulation (cholestasis) and severe itching (pruritus). The chronic pruritus associated with ALGS can significantly disrupt sleep, growth and daily functioning, with many patients requiring liver transplantation before adulthood.

Dr. Or Steg Saban of The Hospital for Sick Children, University of Toronto, noted that as children with ALGS grow, their treatment needs evolve, and the availability of a tablet formulation may improve adherence and continuity of care.

Mirum also highlighted that Health Canada has authorised Livmarli for cholestatic pruritus in patients aged 12 months and older with Progressive Familial Intrahepatic Cholestasis (PFIC), including both oral solution and tablet formulations.

Livmarli is an orally administered ileal bile acid transporter (IBAT) inhibitor approved in multiple regions for paediatric cholestatic liver diseases. The therapy is currently being evaluated in the Phase III EXPAND study for additional cholestatic pruritus indications.