EC Approves Mirum Pharmaceuticals’ Livmarli for Pruritus in Patients with Alagille Syndrome

The European Commission has granted marketing authorization to Mirum Pharmaceuticals’ Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome 2 months of age and older, the first and only approved therapy for the rare liver condition in the European Union or United States.

Photo: Chris Peetz, president and CEO at Mirum

Alagille syndrome is a rare genetic disorder in which bile ducts are abnormally narrow, malformed, and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people. In patients with the condition, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys, and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60 percent to 75 percent of patients with Alagille syndrome have a liver transplant before reaching adulthood.

Signs and symptoms arising from liver damage in ALGS may include jaundice, disfiguring cholesterol deposits under the skin, and intense itching known as pruritus. The pruritus experienced by patients with Alagille syndrome is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.

Livmarli is an orally administered, once-daily, ileal bile acid transporter. It 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 Alagille syndrome 1 year of age and older. Regulatory reviews are also underway in China, Taiwan, South Korea, and Great Britain.

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.

“With more than six years of data and real-world evidence showing Livmarli’s impact, we believe patients and their families will have a chance at experiencing a new normal with relief from the burden caused by cholestasis,” said Chris Peetz, president and CEO at Mirum. “We look forward to advancing Livmarli’s availability throughout Europe and getting this important treatment option into the hands of patients.”

Author: Rare Daily Staff