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Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":" FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today 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.\n\n\nThe 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.\n\n\nAbout LIVMARLI® (maralixibat) oral solution\n\n\nLIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older.\n\n\nLIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.\n\n\nMirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older, and in Europe, in PFIC for patients two months of age and older.\n\n\nLIVMARLI 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 orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.\n\n\nIMPORTANT SAFETY INFORMATION\n\n\nLIVMARLI can cause side effects, including:\n\n\nChanges in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of live...