The Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation for the public reimbursement of LIVMARLI® (maralixibat oral solution) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS). This follows Health Canada’s authorization of LIVMARLI for this indication in 2023, marking an essential milestone for patients suffering from this rare liver disorder. LIVMARLI stands as the first and only approved medication in Canada for this condition, offering hope to those affected, many of whom are children, by alleviating the debilitating effects of cholestatic pruritus associated with ALGS.
This CADTH recommendation is grounded on data from the pivotal ICONIC study and six years of data across the LIVMARLI clinical program. The study showcased statistically significant and clinically meaningful reductions in pruritus and serum bile acids, with benefits sustained over several years of treatment. This recommendation not only emphasizes the treatment’s clinical efficacy but also its potential to significantly improve patients’ quality of life.
The approval of LIVMARLI in Canada, coupled with existing approvals in the U.S. and Europe for ALGS and Progressive Familial Intrahepatic Cholestasis (PFIC), reinforces Mirum Pharmaceuticals’ commitment to addressing the needs of patients with rare liver diseases. ALGS, characterized by bile duct abnormalities leading to progressive liver disease, has long been a challenging condition with limited treatment options. The disorder’s hallmark, cholestasis, results in bile acid accumulation, causing severe pruritus and contributing to liver injury. This condition frequently necessitates liver transplantation, highlighting the critical need for effective treatments like LIVMARLI.
The CADTH’s move to recommend LIVMARLI for reimbursement is a step forward in making this vital treatment more accessible to Canadian patients, potentially alleviating the physical and emotional burden of ALGS and offering a new beacon of hope for affected families.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.