Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The new Prescription Drug User Fee Act (PDUFA) date is March 13, 2024. The FDA extended the PDUFA date to allow time for a full review of a submission provided in response to an FDA information request, which the FDA deemed a major amendment.

The submission comprised further analysis of certain clinical information that was previously provided to the FDA by the company. No additional data or studies have been requested. LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholESTatic pruritus in patients With Alagille syndrome (ALGS) three months of age and older.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and an designation for ALGS, PFIC and biliary atresia.LIVMARLI can cause side effects, including: Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can shorten during treatment with LIVMARLI.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of cholastatic pruritus in patients of Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic prur features in patients with Alagille Syndrome (PFIC). LIVMARLI can cause side effect, including: Changes in certain liver tests are Common in patients with Alagille and Phase 2b VANTAGE study for primary biliary cholangitis.

Lastly, Chenodal, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX.