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Mirum Pharmaceuticals’ LIVMARLI (maralixibat) Approved by the European Commission for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome Two Months and Older
- First and only medication approved in both the U.S. and Europe to treat cholestatic pruritus in patients with Alagille syndrome. - Approval supported by
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- First and only medication approved in both the U.S. and Europe to treat cholestatic pruritus in patients with Alagille syndrome.\n\n- Approval supported by more than six years of LIVMARLI data demonstrating improvements in transplant-free survival, serum bile acids, growth, xanthomas, and quality of life.\n\n- Expected to launch first in Germany in early 2023.\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. 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.\n\nLIVMARLI is also approved by the U.S. Food and Drug Administration and the Israeli Ministry of Health for the treatment of cholestatic pruritus in patients with ALGS one year of age and older. Regulatory reviews are also underway in China, Taiwan, South Korea, and Great Britain.\n\nThe 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.\n\n“We are delighted that the European Commission has approved LIVMARLI and that patients and physicians in Europe will have a new standard of care for this challenging disease,” said Chris Peetz, president and chief executive officer at Mirum. “With more than six years of data and real-world evidence showing LIVMARLI’s impact, we believe pat...