Mirum Pharmaceuticals announced the European Commission’s marketing authorization for maralixibat (Livmarli®) oral solution for patients aged 2 months and older with Alagille syndrome (ALGS). The medication treats cholestatic pruritus and is the only therapy for ALGS approved in both the European Union and the United States.

“It is encouraging to know that physicians will now have a medication to reduce cholestatic pruritus and to possibly decrease the need for liver transplantation when pruritus is the indication for liver transplantation,” professor Emmanuel Jacquemin, MD, PhD, head of the pediatric hepatology and liver transplantation unit at Bicêtre Hospital, AP-HP, Paris-Saclay University in Le Kremlin-Bicêtre, France, said.

“The data collected in the clinical studies demonstrate significant potential for Livmarli to reduce serum bile acids and pruritus, which could significantly improve the quality of life for patients and their families.”


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The European Commission approved the treatment based on 6 years of data from the maralixibat program, specifically from the ICONIC study, which demonstrated significant and meaningful reductions in pruritus in patients taking the medication vs placebo.

Read more about ALGS therapies

ALGS is a rare hereditary disorder characterized by abnormally narrow and malformed bile ducts, resulting in bile accumulation in the liver and progressive liver disease. The disease occurs in 1 in 30,000 people and can affect multiple organ systems in addition to the liver, including the heart, kidneys, and central nervous system.

Pruritus, jaundice, and xanthomas are common symptoms arising from liver damage in patients with ALGS. Maralixibat was also shown to significantly and durably reduce bile acids, xanthoma severity, and cholesterol levels, and the data also showed improvements in event-free survival compared to that in a natural history cohort.

Reference

Mirum Pharmaceuticals’ Livmarli (maralixibat) approved by the European Commission for the treatment of cholestatic pruritus in patients with Alagille syndrome two months and older. News release. Mirum Pharmaceuticals; December 13, 2022.