Mirum Pharmaceuticals Inc. has received Health Canada authorization for the tablet formulation of Livmarli (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).
The approval expands Livmarli’s existing use in Canada, where the drug was previously authorized only as an oral solution.
The tablet formulation complements Livmarli’s 9.5 mg/mL oral solution and is indicated for:
This creates a dual-formulation approach, supporting patients as they age and their dosing needs evolve.
“With a liquid formulation for younger patients and a convenient one-tablet-per-dose option for older patients, Livmarli now provides greater dosing flexibility,”
ALGS-associated pruritus is severe, chronic, and life-disrupting, often affecting:
As paediatric patients grow older, treatment adherence becomes increasingly important.
“The availability of a tablet formulation may support adherence and continuity of care as children with ALGS grow,”
Earlier, Health Canada also authorized Livmarli for PFIC-related cholestatic pruritus, including:
This positions Livmarli as one of the few therapies in Canada offering formulation flexibility across two rare paediatric cholestatic liver diseases.
ALGS is a rare genetic, multisystem disorder with an estimated incidence of 1 in 30,000 people. Key disease characteristics include:
Clinical burden is significant:
Livmarli (maralixibat) is an oral IBAT inhibitor with approvals across major markets:
The drug is currently being evaluated in the Phase 3 EXPAND study for additional cholestatic pruritus indications.
This tablet approval isn’t about market expansion—it’s about lifecycle management done right:
For rare paediatric liver diseases, formulation flexibility is not cosmetic—it’s clinical.