Livmarli (maralixibat) / Mirum Pharma, Takeda, CANbridge Pharma - LARVOL DELTA

New hope in treating progressive familial intrahepatic cholestasis in children. (PubMed, World J Hepatol) - "IBAT inhibition has demonstrated efficacy in reducing serum bile acids and pruritus. We aim to present the 13 types of PFIC and the current evidence on the use of IBAT inhibitors in treating children with PFIC."

Journal • Review • Cholestasis • Dermatology • Hepatology • Inflammation • Liver Failure • Pruritus

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - P3 | N=90 | Completed | Sponsor: Mirum Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

Trial completion • Cholestasis • Hepatology

Indirect Comparison of Maralixibat and Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis. (PubMed, Clin Ther) - "These findings suggest that maralixibat provides additional clinical benefit for children with PFIC compared with odevixibat in terms of increasing the proportion of sBA responders. Further studies are needed to compare the ability of maralixibat and odevixibat to reduce pruritus and improve long-term outcomes, including patients' quality of life."

Journal • Cholestasis • Dermatology • Hepatology • Pruritus

Use of maralixibat in severe and recurrent intrahepatic cholestasis of pregnancy with concurrent benign recurrent intrahepatic cholestasis (BRIC). (PubMed, J Hepatol) - No abstract available

Journal • Cholestasis • Hepatology

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) (clinicaltrials.gov) - P=N/A | N=70 | Recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | N=50 ➔ 70 | Trial completion date: Sep 2028 ➔ Sep 2030

Enrollment change • Trial completion date • Cholestasis • Hepatology

Optimising Maralixibat in ALGS Management: Key Considerations for Clinical Implementation. (PubMed, Liver Int) - No abstract available

Journal • Dermatology • Pruritus

ALAGILLE SYNDROME: A COMPREHENSIVE ANALYSIS OF CASES DIAGNOSED AND MANAGED OVER A DECADE AT A SINGLE CENTER (ESPGHAN 2025) - "Recently, one patient with ALGS was started on an IBAT inhibitor, Maralixibat. After a 6-month follow-up, this patient showed notable improvements, including reduced pruritus and lower serum bile acid concentrations. Conclusions The availability of IBAT inhibitors for cholestatic liver disease marks a new era in the treatment of ALGS, potentially reducing the need for liver transplantation and enhancing the quality of life."

Clinical • Cholestasis • Dermatology • Hepatology • Liver Failure • Pruritus

EFFECTS OF MARALIXIBAT IN CHILDREN WITH ALAGILLE SYNDROME: A FRENCH REAL-LIFE DATA ANALYSIS (ESPGHAN 2025) - "Diarrhea (n=19) and abdominal pain (n=20) were reported in 28 out of the 54 patients (52%) and led to definitive treatment discontinuation in 9 patients. Conclusions This real-life study confirms the efficacy of maralixibat to decrease pruritus and sBA in ALGS patients with acceptable tolerability."

Clinical • Cholestasis • Dermatology • Hepatology • Pain • Pruritus

EFFECTIVENESS OF ILEAL BILE ACID TRANSPORT INHIBITORS (IBATS) IN CHILDREN WITH ALAGILLE SYNDROME: A SYSTEMATIC REVIEW AND META-ANALYSIS OF RANDOMIZED CONTROLLED TRIALS (ESPGHAN 2025) - "Methods PubMed, EMBASE, and Cochrane databases were systematically searched for randomized controlled trials (RCTs) comparing IBATs (maralixibat and odevixibat) to placebo. This meta-analysis demonstrates that IBATs significantly improve pruritus and reduce serum bile acid levels in pediatric ALGS patients compared to placebo. These findings highlight IBATs as a promising treatment option with good tolerability."

Retrospective data • Review • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Pediatrics • Pruritus

UNDERSTANDING TREATMENT EFFICACIES OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS VIA A PROSPECTIVE WORLD-WIDE REGISTRY, TREATFIC (ESPGHAN 2025) - "Twenty-nine individuals are using odevixibat, 4 individuals used odevixibat in the past, 9 individuals are using maralixibat and 42 individuals are not treated with an IBATi. TreatFIC will allow to assess real-world data on the current treatments for PFIC diseases. To further increase the impact of the registry, centers that treat children or adults with PFIC are invited to participate (pfic@bkk.umcg.nl)."

Clinical • Cholestasis • Hepatology • Pediatrics

IMPACT OF LONG-TERM MARALIXIBAT TREATMENT ON CONCOMITANT MEDICATION USE FOR THE TREATMENT OF CHOLESTATIC PRURITUS IN ALAGILLE SYNDROME: REAL-WORLD EXPERIENCE IN THE UNITED STATES (ESPGHAN 2025) - "Median days on concomitant medication until discontinuation was 309 days. Conclusions Consistent with previous findings, these data demonstrate that maralixibat can reduce polypharmacy burden long-term in a real-world setting."

Clinical • Real-world • Real-world evidence • Dermatology • Hepatology • Immunology • Pruritus

CLINICAL BENEFITS OF MARALIXIBAT FOR PATIENTS WITH ALAGILLE SYNDROME ARE DURABLE THROUGH 7 YEARS OF TREATMENT: DATA FROM THE MERGE STUDY (ESPGHAN 2025) - "There were no new safety signals. Conclusions The benefit of maralixibat in ALGS patients, including both improvements in clinical outcomes and sBA, persist through 7 years of treatment with no new safety signals."

Clinical • Dermatology • Hepatology • Pruritus

GREATER IMPROVEMENTS IN BILIRUBIN WERE OBSERVED IN PRURITUS RESPONDERS AFTER MARALIXIBAT TREATMENT IN PATIENTS WITH PFIC: DATA FROM THE MARCH/MARCH-ON TRIALS (ESPGHAN 2025) - "Conclusions Changes in bilirubin and pruritus are linked in patients on MRX with greater reductions in bilirubin, an important marker of liver health, being observed in pruritus responders. People treated earlier in the course of disease (i.e., lower bilirubin) may have greater likelihood of improvement in pruritus."

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

IMPROVEMENTS IN PRURITUS AFTER MARALIXIBAT TREATMENT ARE ASSOCIATED WITH IMPROVED HEALTH-RELATED QUALITY OF LIFE FOR PATIENTS WITH CHOLESTATIC LIVER DISEASE (ESPGHAN 2025) - No abstract available

Clinical • HEOR • Cholestasis • Dermatology • Hepatology • Pruritus

BILE ACID SUBSPECIES ARE CORRELATED WITH PRURITUS AND BILIRUBIN IMPROVEMENT IN PFIC PATIENTS TREATED WITH MARALIXIBAT: DATA FROM MARCH AND MARCH-ON (ESPGHAN 2025) - No abstract available

Clinical • Dermatology • Pruritus

THE RELATIONSHIP BETWEEN SERUM BILE ACIDS AND EVENT-FREE SURVIVAL FOLLOWING THE USE OF MARALIXIBAT FOR PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC): DATA FROM THE MARCH/MARCH-ON TRIALS (ESPGHAN 2025) - No abstract available

Cholestasis • Hepatology

DOSE-RESPONSE RELATIONSHIPS OF MARALIXIBAT AND ODEVIXIBAT IN PATIENTS WITH ALAGILLE SYNDROME (ESPGHAN 2025) - "Conclusions Present reports on IBATi for patients with ALGS do not establish reliable dose-response relationships for pruritus or sBA. We advocate to address optimization of dose regimens to enhance clinical efficacy at the lowest possible doses."

Clinical • Dermatology • Hepatology • Pruritus

MARALIXIBAT IN CHILDREN WITH CHOLESTASIS DUE TO ALAGILLE SYNDROME: REAL WORLD DATA FROM AN ITALIAN MULTICENTER PROSPECTIVE STUDY (ESPGHAN 2025) - "The appropriate reduction in sBA values during treatment could have a favorable effect on the fibrotic evolution of the liver disease. Side effects are rare, transient or reversible upon discontinuation of therapy."

Clinical • Real-world • Real-world evidence • Cholestasis • Dermatology • Fibrosis • Hepatology • Pediatrics • Pruritus

MARALIXIBAT AND IMPROVING CLINICAL OUTCOMES IN PAEDIATRIC LIVER DISEASES (ESPGHAN 2025) - "Sponsored by Mirum Pharmaceuticals, Inc."

Clinical • Clinical data • Hepatology • Pediatrics

ILEAL BILE ACID TRANSPORTER INHIBITOR TREATMENT FOR BILE SALT EXPORT PUMP DEFICIENCY: TOWARDS UNDERSTANDING CLINICAL RESPONSIVENESS (ESPGHAN 2025) - "Conclusions Maralixibat and odevixibat are approved IBATi treatments for patients with nt-BSEP-deficiency, but published data indicate that a significant proportion (32-68%) of patients do not have a relevant response in terms of relevant sBA or pruritus decrease. More precise characterization of IBATi non-responders may help to develop treatment strategies that are as effective as IBATi in responding patients."

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

ABCB11 mRNA therapy for the treatment of Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2) (ASGCT 2025) - "Current treatment options for PFIC2, including the newly approved small molecule ileal bile acid transporter (IBAT) inhibitors such as Odevixibat and Maralixibat, have limited efficacy and are associated with numerous side effects. Furthermore, administration of ABCB11-mRNA-LNP also improved the conditions and extended the survival of the Abcb11-/- mice challenged by diet enriched in cholic acids. Collectively, our data provides strong preclinical proof-of-concept for systemic mRNA-LNP as a potential disease-modifying therapy for patients with PFIC2."

Late-breaking abstract • Cholestasis • Dermatology • Gene Therapies • Hepatology • Liver Failure • Pruritus • ABCB1

IMPROVEMENTS IN PRURITUS, SERUM BILE ACIDS, AND TOTAL BILIRUBIN FOLLOWING TREATMENT WITH MARALIXIBAT IN PATIENTS WITH PRIMARY SCLEROSING CHOLANGITIS (DDW 2025) - "A clinically meaningful reduction in pruritus was observed in 7 patients with PSC treated with MRX, and a substantial reduction in sBA levels was observed in the majority. These findings indicate a potential role for IBAT inhibitors in treating pruritus in PSC."

Clinical • Cholestasis • Dermatology • Hepatology • Immunology • Pediatrics • Pruritus

Mirum Pharmaceuticals to Present Data at Upcoming Medical Congresses (Businesswire) - "Mirum Pharmaceuticals, Inc...announced that it will present data at three upcoming medical congresses. Digestive Disease Week (DDW) will be held in San Diego, May 3-6, 2025. The European Association for the Study of the Liver (EASL) will take place May 7-10 in Amsterdam, the Netherlands. The 57th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting will follow May 14-17 in Helsinki, Finland. Data from Mirum’s LIVMARLI...will be highlighted in oral and poster presentations during the congresses."

Clinical data • Cholestasis • Gastroenterology • Genetic Disorders • Pruritus

Understanding treatment efficacies of progressive familial intrahepatic cholestasis via a prospective world-wide registry, TreatFIC (EASL 2025) - "Twenty nine individuals are using odevixibat, 4 individuals used odevixibat in the past, 9 individuals are using maralixibat and 42 individuals are not treated with an IBATi. TreatFIC is rapidly expanding and provides prospective data on the treatments of individuals with PFIC and its efficacy. The number of included individuals has grown rapidly since its initiation in 2023. TreatFIC will allow to assess real-world data on the current treatments for PFIC diseases."

Clinical • Cholestasis • Hepatology • Pediatrics

Mirum’s LIVMARLI Now FDA Approved in Tablet Formulation (Businesswire) - "Mirum Pharmaceuticals, Inc...announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)....LIVMARLI tablets are planned to be available in June through Mirum Access Plus."

FDA approval • Launch US • Cholestasis