RARE Daily

FDA Expands Use of Mirum’s Livmarli to Include PFIC

March 14, 2024

Rare Daily Staff

The U.S. Food and Drug Administration has expanded the approval of Mirum Pharmaceuticals’ Livmarli for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis.
Mirum said it has submitted an additional supplemental new drug application to introduce a higher concentration formulation of Livmarli to enable label expansion for younger patients with PFIC.

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate, and an increasing inability of the liver to function.
Livmarli is an orally administered, once-daily, ileal bile acid transporter inhibitor and the only approved medication in the United States for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older and PFIC five years of age and older. Livmarli is approved in Europe, Canada, and other regions for the treatment of cholestatic pruritus in ALGS.

The expanded approval is based on data from the phase 3 MARCH study, the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status. Livmarli has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC.

Mirum has also submitted Livmarli for approval in Europe in PFIC for patients two months of age and older.
“PFIC is a difficult disease for both the patient and family and significantly impedes the quality of life for all,” said Emily Ventura, executive director of the PFIC Network and mom to a daughter with PFIC. “These young and fragile patients endure an itch so severe that they experience deficits in their sleep, nutrition, growth, and, in the past, some have turned to transplant to resolve their itch. We are hopeful that LIVMARLI will have a measurable impact for patients, potentially offering a new normal and relief from the sleepless nights and disruption to their lives caused by cholestatic pruritus.”

Photo: Emily Ventura, executive director of the PFIC Network

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