LIVMARLI Efficacy in PFIC Patients Confirmed in Lancet Phase 3 Study

Mirum Pharmaceuticals recently reported the outcomes of the Phase 3 MARCH-PFIC study, which evaluated the efficacy and safety of LIVMARLI® (maralixibat) oral solution in treating Progressive Familial Intrahepatic Cholestasis (PFIC). The study, published in The Lancet Gastroenterology and Hepatology, stands out as the largest of its kind, involving a randomized, double-blind, placebo-controlled approach.

The research focused on two primary groups: those with BSEP deficiency and a broader All-PFIC cohort, which encompassed a range of genetic deficiencies associated with PFIC. LIVMARLI, an Ileal Bile Acid Transporter (IBAT) inhibitor, demonstrated significant improvements across various health parameters, including pruritus, serum bile acids, growth, and bilirubin levels. These improvements were statistically significant and clinically meaningful, with benefits observed as early as week 2 and sustained throughout the 26-week study duration.

The study's success in meeting its primary and secondary endpoints highlights the potential of LIVMARLI as a non-surgical, pharmacological treatment for PFIC. Notably, the drug showed a positive impact on serum bile acids and bilirubin, which are indicators of transplant-free survival. This advancement is crucial for the long-term care and health of patients with PFIC.

Dr. Alexander Miethke, from Cincinnati Children’s Hospital and the lead author of the publication, emphasized the significance of these findings. He noted that LIVMARLI could provide substantial improvements in pruritus and other parameters that affect the quality of life for patients with various PFIC types. Furthermore, the drug's ability to reduce serum bile acid and bilirubin levels suggests a potential for enhancing native liver survival in PFIC.

Pam Vig, PhD, Chief Scientific Officer at Mirum, expressed satisfaction with the publication of the study, highlighting the significant impact LIVMARLI could have on PFIC patients suffering from cholestasis, which often leads to severe pruritus and poor growth, along with progressive liver disease.

LIVMARLI is an orally administered, once-daily IBAT inhibitor that has been approved by the U.S. Food and Drug Administration for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) and PFIC. It is also the only approved IBAT inhibitor in Europe for treating cholestatic pruritus in patients with ALGS and has been submitted for approval in Europe for PFIC patients.

Mirum Pharmaceuticals is a biopharmaceutical company committed to improving treatments for rare diseases. In addition to LIVMARLI, the company has two other approved medications: CHOLBAM® (cholic acid) capsules and CHENODAL® (chenodiol) tablets. Mirum continues to develop its pipeline with investigational treatments for debilitating liver diseases, such as Volixibat, which is being evaluated in studies for primary sclerosing cholangitis and primary biliary cholangitis.

The company's dedication to advancing the understanding and treatment of rare liver diseases is evident through its ongoing clinical trials and research initiatives. Mirum's mission is to transform the lives of children and adults affected by these rare conditions through innovative and effective therapies.