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FDA approves new treatment for rare liver disorder

EditorLina Guerrero
Published 03/13/2024, 06:06 PM
© Reuters.

FOSTER CITY, Calif. - The U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients aged five years and older with progressive familial intrahepatic cholestasis (PFIC), Mirum Pharmaceuticals (NASDAQ:MIRM) announced today. The approval, based on the Phase 3 MARCH study, signifies a statistically significant reduction in pruritus severity compared to a placebo and covers a wide range of PFIC subtypes.

LIVMARLI is also approved for cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. and other regions, with Mirum planning to introduce a higher concentration formulation for younger PFIC patients later this year.

The MARCH study is the largest randomized trial in PFIC, involving 93 patients with various genetic types of the disease. The approval follows years of clinical research demonstrating improvements in key symptoms, including pruritus, which profoundly affects children with PFIC.

PFIC is a rare genetic disorder leading to progressive liver disease and potential liver failure. The disease impedes bile secretion from liver cells, causing severe itching, jaundice, and growth failure in patients, with an estimated incidence of one in every 50,000 to 100,000 births in the United States and Europe.

LIVMARLI, a once-daily oral solution, is the only FDA-approved medication for cholestatic pruritus in patients with ALGS and PFIC. The drug has received Breakthrough Therapy designation for ALGS and PFIC type 2, as well as orphan designation for both conditions.

Mirum Pharmaceuticals focuses on rare diseases, with three approved medications including LIVMARLI. The company's pipeline includes investigational treatments for liver diseases, with ongoing clinical trials for LIVMARLI and other potential therapies.

The approval of LIVMARLI offers new hope for patients and families affected by PFIC, providing an option beyond liver transplant to manage severe itching and improve quality of life. This information is based on a press release statement from Mirum Pharmaceuticals.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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