EU approves Livmarli for cholestatic pruritus in patients with Alagille syndrome

The approval follows a positive opinion granted by the European Committee for Medicinal Products for Human Use (CHMP) in September 2022. Livmarli is the first and only approved treatment in both the European Union and the United States to treat this rare liver disease affecting one out of 30,000 people globally.

The European Commission’s approval 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 with a mean difference of -1.4 points between groups, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment. The label also includes reductions in xanthoma severity and cholesterol, and the submission was supported by data showing an improvement in event-free survival compared to a natural history cohort.