Mirum Receives FDA Approval for Tablet Version of LIVMARLI
In a significant advancement for the rare liver disease community, Mirum Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted approval for a new tablet formulation of its drug LIVMARLI® (maralixibat). This latest development marks an important step forward in the treatment of cholestatic pruritus associated with two rare and debilitating liver disorders: Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, had previously been approved in an oral solution format. With this new regulatory milestone, Mirum has broadened the treatment options for patients by offering a tablet formulation, which is expected to offer increased convenience and flexibility for older pediatric and adolescent patients.
“The approval of LIVMARLI in tablet form provides a meaningful additional treatment option for patients with ALGS and PFIC,” said Peter Radovich, President and Chief Operating Officer of Mirum Pharmaceuticals. “It allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet-per-dose option for older patients. We have had tremendous success with LIVMARLI since its launch, and we hope that the availability of the tablet will provide convenience that positively impacts patients’ lives.”
Rare, Devastating Conditions with Few Options
Both ALGS and PFIC are rare pediatric liver diseases characterized by impaired bile flow, which can lead to severe complications including liver failure, growth impairment, and life-altering symptoms such as cholestatic pruritus—an intense, unrelenting itch that can drastically reduce quality of life. This itch can be so severe that it leads to sleep disturbances, behavioral issues, and even self-mutilation in young children.
Alagille syndrome is a multisystem disorder resulting from mutations in the JAG1 or NOTCH2 genes. It impacts the liver, heart, skeleton, and other organ systems, and is typically diagnosed in infancy or early childhood. Progressive Familial Intrahepatic Cholestasis, on the other hand, is a group of genetic disorders caused by mutations in genes such as ATP8B1, ABCB11, and others. PFIC patients can present symptoms ranging from early childhood jaundice to persistent itching and progressive liver damage.
Until recently, the treatment landscape for both conditions was limited to surgical interventions (like biliary diversion) or ultimately, liver transplantation in the most severe cases. The introduction of LIVMARLI has been a major therapeutic breakthrough in reducing the burden of disease.
The Science Behind LIVMARLI
LIVMARLI (maralixibat) is the first and only FDA-approved treatment targeting cholestatic pruritus in patients with ALGS and PFIC. The drug works by inhibiting the ileal bile acid transporter (IBAT), which plays a key role in the reabsorption of bile acids in the small intestine. By blocking this transporter, LIVMARLI helps reduce systemic bile acid levels, thereby addressing one of the main drivers of cholestatic itch and liver injury.
Maralixibat has demonstrated a favorable safety and efficacy profile in multiple clinical trials, including the pivotal ICONIC and MARCH studies. Patients treated with LIVMARLI showed statistically significant and clinically meaningful reductions in pruritus scores as well as improvements in quality of life metrics. Moreover, reductions in serum bile acids have been associated with delayed disease progression and improved liver function markers in these patient populations.
From Liquid to Tablet: A Strategic Evolution
The approval of the tablet formulation represents a deliberate and strategic move by Mirum Pharmaceuticals to enhance the patient experience. While the oral solution formulation of LIVMARLI has been instrumental in serving younger pediatric patients, the transition to a tablet format for older children and adolescents provides an easier, more discreet option that aligns with the lifestyle of maturing patients.
For physicians and caregivers, the tablet formulation simplifies dosing regimens and eliminates the need for precise volume measurements, making administration more convenient in both home and clinical settings. Additionally, tablets tend to have longer shelf stability and are easier to transport—critical considerations for families managing a chronic illness.
This formulation expansion also demonstrates Mirum’s ongoing commitment to lifecycle management of its therapeutic assets, ensuring that patients have access to treatment formats that evolve alongside their needs.
Commercial and Strategic Implications
Since its initial launch, LIVMARLI has rapidly established itself as a cornerstone therapy within the pediatric hepatology community. The commercial success of the drug has been a major driver of Mirum’s growth and positioning within the rare disease pharmaceutical market.
By introducing a tablet formulation, Mirum may be able to increase patient adherence and retention while also improving operational logistics across its global supply chain. Moreover, this move may facilitate market expansion into geographies or healthcare systems that favor solid oral dosage forms over liquids.
The new formulation could also have pricing and reimbursement implications. In some healthcare systems, tablet formulations may be viewed as more cost-effective due to their ease of use and reduced need for patient or caregiver support.
Future Outlook and Pipeline Momentum
Mirum’s announcement also reflects the company’s broader strategy of establishing a leading presence in rare liver disease therapeutics. Beyond ALGS and PFIC, the company is investigating maralixibat and its second IBAT inhibitor, volixibat, for other cholestatic liver diseases including Primary Sclerosing Cholangitis (PSC) and Intrahepatic Cholestasis of Pregnancy (ICP).
As the company builds out its franchise in liver health, the tablet approval for LIVMARLI marks an important credibility boost for both regulators and the investment community. It showcases Mirum’s ability to innovate within the lifecycle of an approved product and respond to real-world patient and clinician feedback.
Voices from the Community
The rare disease community has long been underserved, and news of the tablet approval is likely to resonate deeply with patients and caregivers who face daily challenges related to chronic liver disease management.
Patient advocacy groups have lauded the availability of the tablet as a quality-of-life enhancement. Many families with children on maralixibat have shared their experiences navigating dosing routines, particularly during travel or school hours, when liquid medications are less convenient.
“This is more than just a new formulation,” said one parent whose child is being treated with LIVMARLI. “It’s a sign that the company is listening to us and making life a little easier for families like ours.”
The FDA’s approval of LIVMARLI in tablet form marks a milestone moment not only for Mirum Pharmaceuticals but also for the rare liver disease community at large. It underscores the importance of therapeutic innovation that goes beyond efficacy—embracing convenience, patient experience, and real-world application.
As Mirum continues to expand its impact in pediatric hepatology and rare disease care, the tablet formulation of LIVMARLI stands as a testament to the company’s mission: to improve lives through thoughtful, patient-centric drug development.
