Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a leader in developing innovative treatments for rare liver diseases, has proudly announced the U.S. Food and Drug Administration (FDA) approval of LIVMARLI® (maralixibat) oral solution. This pivotal approval makes LIVMARLI available for prescribing in the United States for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC). This milestone is supported by the compelling results of the MARCH Phase 3 study, which demonstrated a highly statistically significant reduction in pruritus severity between patients treated with LIVMARLI and those given a placebo (p<0.0001).

Mirum's commitment to expanding treatment options for this patient population is further underscored by its submission of a supplemental new drug application (sNDA) for a higher concentration formulation of LIVMARLI. This formulation, utilized in the MARCH study, aims to enable label expansion for younger patients with PFIC later this year.

LIVMARLI has already been approved for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) across multiple age groups in the U.S., Europe, Canada, and other global regions. Its approval for PFIC marks a significant advancement, offering new hope to patients suffering from this rare and debilitating condition, including those with the rarest subtypes of PFIC.

Chris Peetz, CEO of Mirum Pharmaceuticals, expressed gratitude towards the patients, families, and clinicians whose participation in the study was crucial for achieving this approval. The MARCH study, the largest randomized trial conducted in PFIC, encompassed 93 patients across various genetic types of PFIC, showcasing LIVMARLI's potential to transform the lives of those affected by cholestatic pruritus associated with PFIC.

Richard Thompson, Professor of Molecular Hepatology at King's College London and an investigator in the MARCH study, highlighted the significance of LIVMARLI's approval, noting the meaningful improvements observed across important parameters, including pruritus. Emily Ventura, Executive Director of the PFIC Network and a mother of a daughter with PFIC, shared the profound impact of PFIC on patients and their families, expressing hope that LIVMARLI will bring measurable relief and improve the quality of life for those affected by cholestatic pruritus.