Mirum Pharmaceuticals’ LIVMARLI Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC). Mirum has also submitted an additional supplemental new drug application (sNDA) to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC, later this year.

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LIVMARLI (maralixibat) oral solution is now available for prescribing in the US for cholestatic pruritus in patients with PFIC. (Photo: Business Wire)

LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. (three months and older), Europe (two months and older), in Canada and other regions globally.

“LIVMARLI has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, chief executive officer at Mirum. “Thank you to the patients, families, and clinicians whose study participation made this approval possible.”

The 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’s approval in cholestatic pruritus for patients with PFIC is a result of years of investigation and a collection of a strong body of clinical evidence showing meaningful improvements across a number of important parameters, including pruritus, affecting children with PFIC,” said Richard Thompson, professor of molecular hepatology at King’s College London and an investigator in the MARCH study. “I am pleased that we will have a well-studied and efficacious option to offer patients whose life has been disrupted by itch.”

“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.”