Maralixibat (Livmarli)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Livmarli be reimbursed by public drug plans for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) if certain conditions are met.
• The Canadian Drug Expert Committee (CDEC) determined that Livmarli demonstrates acceptable clinical value versus vehicle placebo and the currently appropriate comparator, odevixibat, in patients with PFIC. This determination was sufficient for CDEC to recommend that Livmarli be reimbursed. Given that Livmarli is expected to be an alternative to odevixibat, acceptable clinical value refers to at least comparable value versus odevixibat.
• Evidence from one randomized trial showed that treatment with Livmarli for 26 weeks resulted in a clinically meaningful improvement in cholestatic pruritus (itching) compared with inactive foam in patients with PFIC. Improvements were observed across multiple pruritus-related outcomes as well as supportive blood markers. These outcomes aligned with patient-identified priorities and were considered clinically meaningful. Evidence from indirect treatment comparisons did not demonstrate a clear difference in effectiveness between Livmarli and odevixibat. Overall, the available evidence was considered consistent with similar clinical effects between treatments, although with residual uncertainty.
• PFIC is a group of rare, inherited liver diseases that typically begins in infancy or early childhood. Patients and caregivers identified pruritus as the most burdensome symptom of the disease, with substantial impacts on their quality of life. Available treatment options often provide limited or inconsistent relief of pruritus, and some patients require invasive surgical interventions or liver transplantation.
• CDEC concluded that maralixibat addresses an important unmet clinical need by providing a nonsurgical, mechanism-based treatment option that improves pruritus and may reduce disease burden. The committee considered limitations in the clinical evidence in the context of the rarity of PFIC and the challenges of conducting large trials in this population.
• Livmarli should only be reimbursed for the treatment of cholestatic pruritus in patients with PFIC. Eligible patients are those with a diagnosis of PFIC who have clinically significant cholestatic pruritus and elevated serum bile acid levels. Treatment with Livmarli should not be initiated in patients with a history of liver transplantation or biliary surgery and should be discontinued if either procedure occurs during therapy.
• Livmarli should only be reimbursed if there is meaningful clinical benefit following an adequate trial of therapy, there is ongoing assessment by a specialist experienced in the management of PFIC, and the cost of Livmarli is reduced.