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

Indirect treatment comparison of odevixibat and maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome. (PubMed, J Comp Eff Res) - " In this first indirect treatment comparison of ileal bile acid transporter inhibitors in ALGS, ODX demonstrated a differentiated clinical profile versus MRX, with significant benefits in HRQoL and tolerability, and consistent trends favoring better efficacy. These results may support informed treatment decision-making for patients."

Journal • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Pain • Pruritus

OASIS: MRX-901: An Open-label Study to Assess Safety and Tolerability of Maralixibat in Participants With Intrahepatic Cholestasis of Pregnancy (ICP) (clinicaltrials.gov) - P2 | N=15 | Not yet recruiting | Sponsor: Mirum Pharmaceuticals, Inc.

New P2 trial • Cholestasis • Hepatology

Real-world use of maralixibat in biliary atresia: a case series. (PubMed, Front Pediatr) - "Pruritus was assessed using the Clinician Scratch Scale (CSS) at baseline and last clinical follow-up. All patients reported improved CSS with no increased usage of other antipruritic medications, and the medication was well tolerated."

Journal • Real-world evidence • Cholestasis • Dermatology • Hepatology • Pruritus • Transplantation

Post-Marketing Safety Signals of Ileal Bile Acid Transporter Inhibitors: A FAERS Pharmacovigilance Analysis of Maralixibat and Odevixibat (ACG 2026) - "Among 7,654,686 FAERS reports, 1,026 involved maralixibat and 352 involved odevixibat. Age data was available in 520 reports with a median age of 7 years (IQR 2â15). The most frequently reported indications were Alagille syndrome in 361 reports (26%), cholestasis in 339 (25%), PFIC in 270 (20%), and pruritus in 186 (14%)."

Adverse events • Clinical • P4 data • Cholestasis • Gastrointestinal Disorder • Hepatology • Liver Failure • Pruritus • ROR1

Comparative Efficacy and Safety of IBAT Inhibitors, Bezafibrate, Rifampin, and Naltrexone for Cholestatic Pruritus in Primary Biliary Cholangitis: A Systematic Review and Network Meta-Analysis (ACG 2026) - "Our network meta-analysis included 14 randomized controlled trials comprising 1,142 patients with primary biliary cholangitisâassociated cholestatic pruritus. The cohort was predominantly female (94.2%), with a mean age of 52.8 years and severe baseline pruritus (mean WI-NRS 7.2±1.1). Treatment allocation included Linerixibat (n=238), Maralixibat (n=96), Odevixibat (n=82), Bezafibrate (n=184), Rifampin (n=112), Naltrexone (n=98), and placebo (n=332)."

Retrospective data • Review • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

Evolving Treatment Strategies in Alagille Syndrome (ACG 2026) - "She was started on fat-soluble vitamins, ursodiol, hydroxyzine, and antihistamines for pruritus...After transferring care, maralixibat was recommended for continued pruritus, but the patient initially declined...Figure: Figure 2: Liver enzymes with persistently elevated AST, ALT, and GGT levels over time, consistent with ongoing cholestatic liver disease in the setting of Alagille syndrome. Liver enzymes have not been collected after initiation of odevixibat."

Cholestasis • Fibrosis • Hepatology • Immunology • Inflammation • Primary Biliary Cholangitis • Pruritus • JAG1 • NOTCH2

Therapeutic Efficacy and Safety Profile of Ileal Bile Acid Transporter Inhibitors in Pruritus Associated With Primary Biliary Cholangitis: A Systematic Review and Meta-Analysis of Randomized Controlled Trial (ACG 2026) - "Across the four included trials, a total population of 406 patients was evaluated, with an estimated overall mean age of 55.5 ± 11.1 years. IBAT inhibitors significantly reduced pruritus severity as measured by the 5-D Itch score compared with placebo (MD= -0.57,95% CI:-0.87 to -0.28,P = 0.0001,I² = 0%) with significant reduction with linerixibat (MD= -0.59,95% CI: -0.89 to -0.29) with no difference in maralixibat group (MD= -0.30,95% CI:-1.54 to 0.94). No significant improvement was observed in the PBC-40 itch domain score (MD= -1.26,95% CI: -3.17 to 0.65,P = 0.20,I² = 66%)."

Retrospective data • Review • Dermatology • Hepatology • Immunology • Primary Biliary Cholangitis • Pruritus

Ileal Bile Acid Transporter Inhibitors in Severe Refractory Cholestatic Pruritus in Adults: A Case Series Exploring Real-World Application Beyond Confirmed Progressive Familial Intrahepatic Cholestasis (ACG 2026) - "Odevixibat and maralixibat are two FDA-approved ileal bile acid transporter inhibitors (IBATi) for PFIC SRCP, which interrupt enterohepatic bile acid (BA) recirculation...Medications included mycophenolate mofetil, prednisone, ursodiol, hydroxyzine, and previous trial of seladelpar...Our case findings pose IBATi as promising therapy for adults with suspected or uncharacterized variants of PFIC with SRCP. Real-world data characterizing treatment response predictors in adults remain limited, and our cases further highlight the importance of genotypic associations, BA burden, and overlapping disease presentations as potential determinants of response requiring further study."

Clinical • Real-world • Real-world evidence • Autoimmune Hepatitis • Cholestasis • Dermatology • Dyslipidemia • Hepatology • Immunology • Inflammation • Primary Biliary Cholangitis • Pruritus • ABCB1 • ABCB4

Progressive familial intrahepatic cholestasis disease burden and clinical approaches: a systematic review. (PubMed, J Comp Eff Res) - "Many patients treated with maralixibat and odevixibat demonstrated improvements in pruritus, serum bile acid concentrations, quality of life and markers of liver health, particularly those with PFIC2/BSEP deficiency. PFIC is associated with significant clinical and psychosocial burden. Ileal bile acid transporter inhibitors provide a novel, targeted, nonsurgical treatment option for many patients: current evidence supports improvements in pruritus and serum bile acid control, particularly in PFIC2/BSEP deficiency; however, treatment responses are heterogeneous and additional long-term clinical and economic evidence is needed."

Journal • Review • Cholestasis • Dermatology • Hepatology • Pruritus

Maralixibat for PFIC. (PubMed, Can Liver J) - No abstract available

Journal

A REAL-WORLD, PROSPECTIVE EVALUATION OF MARALIXIBAT THERAPY IN PEDIATRIC ALAGILLE SYNDROME: CLINICAL AND LIVER ELASTOGRAPHY OUTCOMES (ESPGHAN 2026) - "Treatment with ursodeoxycholic acid, rifampicin, hydroxyzine, and vitamins were maintained. Larger cohorts with longer observation are needed to clarify the long-term influence of Maralixibat on liver stiffness trajectories in ALGS. Contact e-mail address [email protected]"

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

EVENT-FREE SURVIVAL OF MARALIXIBAT-TREATED PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS COMPARED WITH A NATURAL HISTORY COHORT FROM NAPPED (ESPGHAN 2026) - "Conclusions Maralixibat treatment was associated with improved event-free survival in nt-BSEP and FIC1 patients. Contact e-mail address [email protected]"

Clinical • Cholestasis • Hepatology • Liver Failure • Pruritus

LONGITUDINAL EVOLUTION OF BILE ACID PROFILES AFTER IBAT THERAPY IN PEDIATRIC CHOLESTATIC DISEASES (ESPGHAN 2026) - "Stratified by therapy, both maralixibat and odevixibat reduced total bile acids over time; maralixibat produced a faster early decline, while odevixibat showed a gradual decrease, although differences between agents were not statistically significant. Larger, disease-stratified longitudinal studies are needed to confirm these exploratory observations. Contact e-mail address [email protected]"

Clinical • Cholestasis • Hepatology • Pediatrics

CILIOPATHIES AS A CAUSE OF CHOLESTASIS AND A POTENTIAL ROLE FOR MARALIXIBAT, AN ILEAL BILE ACID TRANSPORTER INHBITOR: A CASE SERIES AND LITERATURE REVIEW (ESPGHAN 2026) - "Pruritus and sBA levels were infrequently reported in the literature, potentially indicating a lack of awareness about the frequency of cholestatic pruritus and hypercholanemia in these patients. Contact e-mail address [email protected]"

Clinical • Review • Cholestasis • Genetic Disorders • Hepatology • Pruritus

IMPACT OF MARALIXIBAT ON CAREGIVER BURDEN FOR PATIENTS WITH ALAGILLE SYNDROME AND PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: BASELINE AND 6-MONTH FOLLOW-UP (ESPGHAN 2026) - "ZBI-12 trends support reduced burden, though HADS results were mixed. Longer-term follow-up is needed to fully understand the broader caregiver benefits of maralixibat therapy."

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

EFFICACY AND TOLERABILITY OF MARALIXIBAT IN PATIENTS WITH ALGS OR PFIC AFTER CONVERTING FROM A LIQUID TO A TABLET FORMULATION (ESPGHAN 2026) - "Availability of both formulations provides valuable flexibility, supporting patient preference and adherence, particularly as patients age. Contact e-mail address [email protected]"

Clinical • Cholestasis • Hepatology • Pruritus

REAL-WORLD USE OF MARALIXIBAT IN BILIARY ATRESIA: A CASE SERIES (ESPGHAN 2026) - "All patients were receiving ursodiol and rifampin. Conclusions Maralixibat may be effective in patients with BA for the management of CP. These data support the need for larger studies to systematically evaluate the potential benefit of maralixibat for the treatment of CP in BA."

Clinical • Real-world • Real-world evidence • Cholestasis • Hepatology • Pruritus

Benefits Beyond Itch Reduction with Maralixibat: Emerging Clinical Evidence in PFIC and ALGS (ESPGHAN 2026) - No abstract available

Clinical

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU) (clinicaltrials.gov) - P4 | N=230 | Recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | N=100 ➔ 230

Enrollment change • Cholestasis • Hepatology

Event-free survival of maralixibat-treated patients with progressive familial intrahepatic cholestasis compared with aligned real-world data of untreated patients from NAPPED (EASL 2026) - "Maralixibat treatment was associated with improved event free survival in nt-BSEP and FIC1 patients."

Clinical • Real-world • Real-world evidence • Cholestasis • Hepatology • Liver Failure • Pruritus

Utilization of maralixibat, an ileal bile acid transporter inhibitor, for the treatment of intrahepatic cholestasis of pregnancy: a case series (EASL 2026) - " Retrospective analysis of four cases of recurrent, severe ICP with marked hypercholanemia and pruritus with insufficient ursodeoxycholic acid response treated with MRX through a compassionate use program was conducted. These findings suggest that maralixibat may effectively manage ICP by lowering TBA and improving pruritus."

Clinical • Cholestasis • Hepatology • Pruritus • ABCB1

Use of maralixibat for the treatment of cholestatic pruritus in late-onset PFIC (EASL 2026) - "MRX led to significant improvements in pruritus and sBA in patients with late-onset PFIC, suggesting IBATi may offer benefit in this group. Additional studies evaluating longer follow-up in a larger cohort would allow for better characterization of the benefits of IBATi in late-onset PFIC."

Cholestasis • Dermatology • Hepatology • Pruritus • ABCB1 • ABCB4

INDIRECT TREATMENT COMPARISON OF ODEVIXIBAT AND MARALIXIBAT FOR THE TREATMENT OF CHOLESTATIC PRURITUS IN PATIENTS WITH ALAGILLE SYNDROME (ALGS) (ISPOR 2026) - P2, P3 | "In patients with ALGS, odevixibat demonstrated numerically greater improvements in pruritus and sBA, and significantly enhanced HRQoL versus maralixibat. Odevixibat exhibited superior tolerability for gastrointestinal events and abdominal pain. This is the first analysis applying regression-adjusted matching methodology to robustly and indirectly compare efficacy and safety between odevixibat and maralixibat in ALGS."

Clinical • Dermatology • Hepatology • Pruritus

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU) (clinicaltrials.gov) - P4 | N=100 | Recruiting | Sponsor: Mirum Pharmaceuticals, Inc. | N=223 ➔ 100

Enrollment change • Trial initiation date

Maralixibat in Patients With Cystic Fibrosis and Constipation (clinicaltrials.gov) - P2/3 | N=20 | Recruiting | Sponsor: Children's Hospital Los Angeles | Not yet recruiting ➔ Recruiting | Trial primary completion date: Jun 2026 ➔ Jun 2027

Enrollment open • Trial primary completion date • Constipation • Cystic Fibrosis • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases