FOSTER CITY, Calif., November 7, 2025 — Mirum Pharmaceuticals, Inc., a biopharmaceutical company dedicated to transforming the lives of patients with rare and chronic liver diseases, showcased new data from its LIVMARLI® (maralixibat) and volixibat clinical programs at the American Association for the Study of Liver Diseases (AASLD) “The Liver Meeting” 2025. The presentations featured real-world experience with LIVMARLI and detailed results from the VANTAGE Phase 2b trial of volixibat in adults with Primary Biliary Cholangitis (PBC). These new findings reinforce Mirum’s leadership in the development of ileal bile-acid transporter (IBAT) inhibitors and demonstrate continued innovation in the management of cholestatic liver diseases.
Science Significance
The new analyses highlight how IBAT inhibition represents a powerful, disease-modifying approach for cholestatic conditions characterized by bile-acid accumulation. In the VANTAGE study, volixibat significantly improved biochemical markers of cholestasis, including reductions in alkaline phosphatase (ALP) and total bile acids, while improving patient-reported outcomes such as fatigue and pruritus scores. These results are consistent with Mirum’s prior data showing that interrupting enterohepatic circulation can normalize bile-acid levels and reduce liver injury. Parallel presentations demonstrated real-world effectiveness of LIVMARLI, already approved in pediatric patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis (PFIC), confirming its safety and efficacy in broader patient populations. Together, the findings establish Mirum’s IBAT-inhibitor portfolio as an integrated scientific platform addressing both genetic and acquired cholestatic liver diseases through a shared molecular pathway.
Regulatory Significance
Mirum’s presentation at AASLD reflects its active engagement with global regulators as it advances label expansions and new indications. LIVMARLI, approved in over 40 countries, is currently under review for additional pediatric and adult cholestatic disorders, while volixibat’s Phase 2b success supports advancement toward Phase 3 registration trials in PBC. All programs are conducted under Good Clinical Practice (GCP) and follow FDA and EMA rare-disease development guidance, emphasizing endpoints that correlate with both biochemical improvement and clinical benefit. The use of real-world evidence (RWE) from post-marketing studies also enhances regulatory confidence in long-term safety. By combining traditional clinical-trial evidence with RWE analytics, Mirum is helping shape regulatory standards for the approval of innovative, mechanism-based therapies in rare liver diseases.
Business Significance
Advertisement
From a corporate perspective, Mirum’s scientific updates underscore its dominant position in the rare-liver-disease market, a therapeutic area valued at over $5 billion globally. The company’s dual-asset strategy—commercializing LIVMARLI while developing volixibat—creates a sustainable revenue and innovation model. Revenue growth from LIVMARLI supports volixibat’s late-stage development, minimizing dependence on external financing. In parallel, Mirum continues to strengthen partnerships with key hepatology centers and patient-advocacy groups to broaden access. Presenting at AASLD elevates the company’s visibility among investors and clinicians, potentially stimulating new collaboration and licensing opportunities. The combined pipeline positions Mirum as one of the few biopharma companies with commercialized and clinical-stage IBAT inhibitors, reinforcing its long-term strategic value.
Patients’ Significance
For patients, the data presented at AASLD signify meaningful progress in conditions long associated with severe pruritus, fatigue, and progressive liver damage. LIVMARLI has already transformed the standard of care for children suffering from rare cholestatic syndromes, improving sleep, itch control, and overall quality of life. Extending these benefits to additional disorders could profoundly reduce the physical and emotional toll on patients and families. Meanwhile, volixibat’s clinical benefits in adults with PBC address a population that has few effective options beyond ursodeoxycholic acid and obeticholic acid, both of which have limitations. By reducing bile-acid toxicity and improving symptom burden, Mirum’s therapies represent a new therapeutic era focused on mechanism-based relief rather than symptomatic management.
Policy Significance
The progress of Mirum’s pipeline aligns closely with international policy frameworks supporting rare-disease innovation. Global agencies such as the FDA, EMA, and Japan’s PMDA continue to prioritize expedited review pathways for therapies addressing unmet medical needs. Mirum’s success exemplifies how targeted mechanisms and RWE integration can accelerate approvals and expand access to orphan drugs. Additionally, by addressing pediatric and adult populations alike, Mirum contributes to the WHO’s global liver-health agenda, which emphasizes early intervention and equitable treatment access. The company’s efforts also highlight the value of public–private collaboration in developing therapies for ultra-rare conditions, aligning with orphan-drug legislation and rare-disease research funding initiatives worldwide.
By unveiling new data from LIVMARLI and volixibat at the AASLD Liver Meeting 2025, Mirum Pharmaceuticals reaffirms its position as a leader in rare-liver-disease therapeutics. Through a deep understanding of bile-acid physiology, a robust regulatory strategy, and a focus on measurable patient benefit, Mirum is translating scientific insight into transformative clinical solutions. As it prepares for global Phase 3 development and broader commercialization, the company continues to set a benchmark for clinical excellence, regulatory innovation, and patient-centric progress in hepatology. For patients worldwide, Mirum’s growing evidence base signals more than scientific advancement—it represents the arrival of new hope in the management of chronic and rare liver disease.
Source: Mirum Pharmaceuticals, Inc. press release
