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

Letter to The Editor, Regarding "Indirect Comparison of Maralixibat and Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis" Recently Published by Lacey and Colleagues. (PubMed, Clin Ther) - No abstract available

Journal • Cholestasis • Hepatology

Exploring Maralixibat for Treatment-Resistant Pruritus in Intrahepatic Cholestasis of Pregnancy and Primary Biliary Cholangitis: A Case Report. (PubMed, Am J Case Rep) - "Ursodeoxycholic acid remains the only validated treatment for ICP but shows limited efficacy in alleviating pruritus. However, prior treatments may have contributed to symptom improvement. Although the findings are encouraging, the use of ileal bile acid transporter inhibitors during pregnancy warrants further evaluation in controlled studies before definitive conclusions can be established."

Journal • Cholestasis • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

Treatment of intractable pruritus with maralixibat in patients with Alagille syndrome before and after reversal of biliary diversion. (PubMed, JPGN Rep) - "In both cases, treatment with maralixibat was well-tolerated and demonstrated marked improvements in cholestatic pruritus. This suggests that maralixibat is a pharmacological alternative for patients who would like to pursue reversal of SBD."

Journal • Cholestasis • Dermatology • Hepatology • Pediatrics • Pruritus • Transplantation

Efficacy of ileal bile acid transport inhibitors in children with Alagille syndrome: a meta-analysis. (PubMed, Eur J Pediatr) - "IBATIs are effective and well tolerated in pediatric ALGS, providing meaningful improvements in pruritus and quality of life. Longer trials are needed to confirm durability, safety, and transplant-free survival impact."

Clinical • Journal • Retrospective data • Review • Dermatology • Pediatrics • Pruritus • Transplantation

EFFICACY AND SAFETY OF ILEAL BILE ACID TRANSPORTER INHIBITORS IN AUTOIMMUNE CHOLESTATIC LIVER DISEASES: A META-ANALYSIS (UEGW 2025) - "Ileal bile acid transporter (IBAT) inhibitors, such as maralixibat and linerixibat, offer a novel approach by reducing bile acid reabsorption. IBAT inhibitors are associated with clinically meaningful improvements in pruritus and sleep quality in adults with ACLD. Although adverse events are common, serious events are infrequent, supporting a favorable safety profile. These findings highlight the promise of IBAT inhibitors as an emerging therapeutic option for cholestatic pruritus, though further high-quality studies are needed to confirm long-term benefits and safety across diverse patient populations."

Retrospective data • Cholestasis • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus • FGF19

IMPACT OF MARALIXIBAT ON CAREGIVER BURDEN FOR PATIENTS WITH ALAGILLE SYNDROME AND PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: BASELINE AND 6-MONTH FOLLOW-UP (AASLD 2025) - "These results suggest 6 months of MRX treatment may reduce caregiver sleep disruption and burden, likely due to improved pruritus in patients. ZBI-12 trends support reduced burden, though HADS results were mixed. While encouraging, these findings are preliminary."

Clinical • Cholestasis • CNS Disorders • Depression • Dermatology • Hepatology • Mood Disorders • Pruritus • Psychiatry • Sleep Disorder

LIVER ELASTOGRAPHY IN PEDIATRIC PATIENTS WITH ALAGILLE SYNDROME RECEIVING MARALIXIBAT THERAPY: A REAL-WORLD SINGLE-CENTER STUDY (AASLD 2025) - "All patients had previously been treated with ursodeoxycholic acid, rifampicin, hydroxyzine, and fat-soluble vitamins, which were continued during the study. In this real-world cohort study, children with ALGS who responded to Maralixibat and improved pruritus and serum bile acid levels, showed a favorable trend of decreasing values in liver elastography over time. Although the sample size is small, the effect of Maralixibat seems promising on mitigating disease progression and merits further investigation with recruitment of more patients and a longer follow-up period."

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

MARALIXIBAT, AN ILEAL BILE ACID TRANSPORTER INHIBITOR, REDUCES CHOLESTATIC PRURITUS IN PSC: REAL-WORLD EXPERIENCE (AASLD 2025) - "A clinically meaningful reduction in pruritus was observed in all MRX-treated patients with PSC, 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 • Real-world • Real-world evidence • Cholestasis • Dermatology • Hepatology • Immunology • Pruritus

ILEAL BILE ACID TRANSPORTER INHIBITORS FOR REFRACTORY PRURITUS IN LIVER TRANSPLANT RECIPIENTS: A CASE SERIES (AASLD 2025) - "Ileal bile acid transporter (IBAT) inhibitors, such as odevixibat and maralixibat, have demonstrated efficacy in treating cholestatic pruritus in other contexts but remain underexplored in the post-LT setting...She continued hydroxyzine and doxepin as needed but reports improved quality of life... IBAT inhibitors may represent a safe and effective option for managing refractory pruritus in liver transplant recipients, with potential benefits extending beyond symptom control to meaningful improvements in quality of life. Importantly, these agents were well-tolerated and did not disrupt immunosuppressive regimens or graft function. Given the significant burden of post-LT pruritus and the limited efficacy of existing treatments, further prospective studies are warranted to evaluate long-term safety, efficacy, and optimal clinical use of IBAT inhibitors in this population."

Clinical • Dermatology • Pruritus • Transplant Rejection • Transplantation

REAL-WORLD INSIGHTS INTO PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: FIRST UPDATE FROM THE PROSPECTIVE TREATFIC REGISTRY (AASLD 2025) - "Forty-six individuals are using odevixibat, 8 individuals used odevixibat in the past, 11 individuals are using maralixibat, 2 individuals used maralixibat in the past and 48 individuals are not treated with an IBATi. TreatFIC is rapidly expanding and provides prospective data on the treatments of individuals with PFIC and their 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 • Real-world • Real-world evidence • Cholestasis • Hepatology • Pediatrics

Ileal bile acid transporter inhibitors in Alagille syndrome and Progressive Familial Intrahepatic Cholestasis: A systematic review into dose-response. (PubMed, Br J Clin Pharmacol) - "Ileal bile acid transporter inhibitors (IBATi), including maralixibat and odevixibat, are a novel approach to the treatment of paediatric cholestatic liver diseases, such as Alagille syndrome (ALGS) and different forms of progressive familial intrahepatic cholestasis (PFIC). In the case of odevixibat, outcomes measures for patients with ALGS have only been reported for a dosage of 120 μg/kg/day. In conclusion, the variability in therapeutic outcomes of different doses for all IBATi underscores the necessity for personalization of dosing, which may include decreasing the dosage for responders to IBATi."

Journal • Review • Cholestasis • Dermatology • Hepatology • Pediatrics • Pruritus

Carrier cross-reactivities of the bile acid reabsorption inhibitors elobixibat, linerixibat, maralixibat, and odevixibat. (PubMed, J Lipid Res) - "In contrast, this mutation had no significant effect on the ASBT inhibition by elobixibat, maralixibat, and odevixibat, indicating distinct binding sites. In conclusion, the analyzed BARIs revealed carrier cross-reactivities with NTCP, SOAT, and members of the OATP family, but behaved differently regarding their time-dependent inhibition and potential inhibitor binding sites."

Journal • Hepatitis B • Infectious Disease • Inflammation • SLCO2B1

Treatment of Pruritus With Maralixibat in Early-Stage Chronic Graft Dysfunction of a Child With Alagille Syndrome. (PubMed, Cureus) - "No adverse events were reported. This case offers real-world evidence of the effectiveness of maralixibat in managing cholestatic pruritus in a patient with ALGS and early-stage chronic graft dysfunction, highlighting its potential as a therapeutic option in this challenging patient population."

Journal • Cholestasis • Dermatology • Hepatology • Pruritus • Transplantation

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (clinicaltrials.gov) - P3 | N=7 | Completed | Sponsor: Takeda | Active, not recruiting ➔ Completed

Trial completion

A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - P3 | N=5 | Completed | Sponsor: Takeda | Active, not recruiting ➔ Completed

Trial completion • Cholestasis • Hepatology • ABCB1 • ABCB4

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