Bylvay (odevixibat) / Ipsen - LARVOL DELTA

Early predictors of pruritus response in children with PFIC treated with odevixibat. (PubMed, Dig Liver Dis) - "reduction of sBA levels of around 50% from baseline value at 1 and 3 months is associated with a pruritus response to OT at 6 months. This information could be useful to the management of children with PFIC."

Journal • Cholestasis • Dermatology • Hepatology • Pruritus

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

BOLD-EXT: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (clinicaltrials.gov) - P3 | N=180 | Active, not recruiting | Sponsor: Albireo, an Ipsen Company | Trial completion date: Jun 2028 ➔ Oct 2026 | Trial primary completion date: Jun 2028 ➔ Oct 2026 | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Trial completion date • Trial primary completion date • Hepatology

Pruritus in Progressive Familial Intrahepatic Cholestasis on Resmetirom for MASLD Treated With Ileal Bile Acid Transporter Inhibitor (ACG 2026) - "Subsequently, patient was started on IBATi odevixibat at 40μg/day. Figure: Figure 1A: Change in Fibrosis Score and Serum Liver Enzymes (alanine transaminase, aspartate transaminase) before resmetirom treatment and four months after starting remetirom treatment. Figure 1B: Change in Serum Bile Acids and 5-D Itch Score before IBATi (Ileal Bile Acid Transport Inhibitor) treatment and two months after starting IBATi treatment."

Cardiovascular • Cholestasis • Dermatology • Diabetes • Fibrosis • Genetic Disorders • Hepatology • Hypertension • Immunology • Metabolic Disorders • Metabolic Dysfunction-Associated Steatotic Liver Disease • Pruritus • ABCB4

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

Progressive Intrafamilial Hepatic Cholestasis: An Atypical and Delayed Presentation (ACG 2026) - "Labs showed partial response with short course of empirical prednisone...The patient was started on Odevixibat with improvement in symptoms and ALP...Most commonly manifesting PFIC are homozygous genotypes, our case involved a compound heterozygous manifesting disease. This case highlights the importance of keeping PFIC as part of the differential and including cholestatic genetic panels when evaluating adult-onset prolonged cholestatic diseases."

Alpha-1 Antitrypsin Deficiency • Cholestasis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Pruritus • Respiratory Diseases

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

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

Odevixibat-Induced Remission in Benign Recurrent Intrahepatic Cholestasis Type 1 Complicated by Bile Cast Nephropathy and Hemolytic Anemia (ACG 2026) - "Case Description/ A 22-year-old man presented with one week of severe pruritus, jaundice, scleral icterus, clay-colored stools, vomiting, and weight loss that worsened after doxycycline treatment for presumed chlamydia...Extensive evaluation was unrevealing, and symptoms initially improved with cholestyramine...Endoscopic nasobiliary drainage and biliary diversion are more effective but invasive. Odevixibat, an ileal bile acid transporter inhibitor approved for PFIC, offers a noninvasive alternative by reducing enterohepatic bile acid circulation and hepatic bile acid burden."

Clinical • Anemia • Cholestasis • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Liver Failure • Pruritus • Renal Disease

Biliary atresia-related liver fibrosis. (PubMed, Front Cell Dev Biol) - "In therapeutics, we assess the enduring role of Kasai portoenterostomy (KPE), the limitations of current adjunctive therapies, and the evolving landscape of pharmacological interventions-including ileal bile acid transporter inhibitors (odevixibat), farnesoid X receptor agonists (obeticholic acid), eicosapentaenoic acid, and N-acetylcysteine-alongside ongoing clinical trials. We further highlight how single-cell and spatial transcriptomic technologies are revolutionizing our comprehension of cellular heterogeneity and intercellular crosstalk within the fibrotic niche, offering unprecedented opportunities for molecular subtyping and precision medicine. By bridging mechanistic insights with clinical translation, this review provides a conceptual framework to guide future research, improve diagnostic precision, and develop rationally targeted combination therapies for BA-associated liver fibrosis."

Journal • Review • Cholestasis • Fibrosis • Hepatology • Immunology • Liver Cirrhosis • Transplantation • GPC1 • MMP7 • TGFB1

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

Odevixibat for progressive familial intrahepatic cholestasis and Alagille syndrome-associated cholestatic pruritus. (PubMed, Aust Prescr) - No abstract available

Journal • Review • Cholestasis • Dermatology • Hepatology • Pruritus

A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment (clinicaltrials.gov) - P=N/A | N=30 | Recruiting | Sponsor: Ipsen | Not yet recruiting ➔ Recruiting

Enrollment open

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment (clinicaltrials.gov) - P=N/A | N=30 | Recruiting | Sponsor: Ipsen | Trial completion date: Nov 2029 ➔ Nov 2030 | Trial primary completion date: Nov 2029 ➔ Nov 2030

Trial completion date • Trial primary completion date

Parvovirus B19 infection as a trigger of acute cholestasis in heterozygous genetic BSEP deficiency: a case report and review of the literature of acute parvovirus B19-related hepatitis. (PubMed, Intern Emerg Med) - "Despite treatment with ursodeoxycholic acid and cholestyramine, the symptoms persisted, leading to the introduction of the ileal bile acid transporter (IBAT) inhibitor, odevixibat, which resulted in rapid relief of pruritus and near-complete biochemical normalization within 2 months. In more than half of the patients, predisposing factors such as hematologic disorders or viral co-infections were identified. This review highlights the importance of considering parvovirus B19 infection in the differential diagnosis of acute hepatitis in adults and of investigating potential underlying predisposing factors when the clinical course cannot be fully explained by the viral infection alone."

Journal • Review • Cholestasis • Dermatology • Genetic Disorders • Hematological Disorders • Hepatology • Infectious Disease • Inflammation • Liver Failure • Pruritus • Transplantation • ABCB1

ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - P3 | N=62 | Active, not recruiting | Sponsor: Albireo, an Ipsen Company | Recruiting ➔ Active, not recruiting

Enrollment closed

Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) (clinicaltrials.gov) - P3 | N=254 | Completed | Sponsor: Albireo, an Ipsen Company | Active, not recruiting ➔ Completed

Trial completion • Hepatology

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China (clinicaltrials.gov) - P=N/A | N=20 | Recruiting | Sponsor: Ipsen | -> Recruiting

Enrollment open • Cholestasis • Hepatology

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China (clinicaltrials.gov) - P=N/A | N=20 | Sponsor: Ipsen

Trial initiation date • Cholestasis • Hepatology

REDUCTIONS IN SERUM BILE ACIDS WITH ODEVIXIBAT ARE POSITIVELY CORRELATED WITH LIVER PARAMETERS IN PATIENTS WITH BILE SALT EXPORT PUMP DEFICIENCY (ESPGHAN 2026) - P3 | "Thompson RJ, et al. JPGN Reports 2025; 6:S910−S911 (Poster presentation 1347) Contact e-mail address [email protected]"

Clinical • Cholestasis • Hepatology • 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

SAFETY AND TOLERABILITY OF ODEVIXIBAT IN INFANTS WITH ALAGILLE SYNDROME AGED (ESPGHAN 2026) - P3 | "Conclusions ODX was well tolerated, decreased sBA, and pruritus improved/remained stable (assessed by CSS) in infants aged <12 months with ALGS. Contact e-mail address [email protected]"

Clinical • Hepatology • Pruritus

PRURITUS IMPROVEMENTS WITH ODEVIXIBAT ARE CORRELATED WITH REDUCTIONS IN SERUM BILE ACIDS AND AUTOTAXIN IN PATIENTS WITH BILE SALT EXPORT PUMP DEFICIENCY (ESPGHAN 2026) - P3 | "Thompson RJ, et al. JHEP Rep 2023; 5:100782 Contact e-mail address [email protected]"

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

MHRA grants marketing authorisation for Ipsen’s Bylvay (odevixibat) for cholestatic pruritus in Alagille syndrome (Firstwordpharma Press Release) - "The marketing authorisation of odevixibat was based on the ASSERT Phase III clinical trial data."

MHRA approval • Cholestasis • Genetic Disorders • Pruritus