Mirum’s Oral Drug Becomes First U.S. Approved Alagille Syndrome Therapy

Mirum Pharmaceuticals’ Livmarli (maralixibat) has become the first FDA-approved drug for treating itching in children suffering from Alagille syndrome, a rare liver disorder.

The approval was supported by positive results from a pivotal study, plus five years of supportive data from other trials, showing the drug significantly reduced itching, a common symptom associated with the disease.

Alagille syndrome is caused by malformed bile ducts that over time can lead to a progressive liver disease that causes inflammation and injury to the liver.

Mirum also received a rare pediatric disease priority review voucher for the treatment, which it can either use for a future application or sell to another drug sponsor.