Bylvay (odevixibat) / Ipsen - LARVOL DELTA

Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation (clinicaltrials.gov) - P=N/A | N=20 | Recruiting | Sponsor: Ipsen | Trial completion date: Sep 2032 ➔ May 2032 | Trial primary completion date: Sep 2031 ➔ May 2032

Adverse events • Trial completion date • Trial primary completion date

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea (clinicaltrials.gov) - P=N/A | N=10 | Recruiting | Sponsor: Ipsen | Not yet recruiting ➔ Recruiting | Trial completion date: Sep 2031 ➔ Sep 2032 | Trial primary completion date: Sep 2031 ➔ Sep 2032

Enrollment open • Trial completion date • Trial primary completion date • Cholestasis • Hepatology

Enantiomeric Resolution of Odevixibat via Immobilized Polysaccharide Columns: Impact of NP, RP, and PO Modes on LC-MS-Compatible Method Design. (PubMed, Chirality) - "The method proved robust under deliberate variations in column temperature (±1°C), mobile phase composition (±2%), and flow rate (1.0 ± 0.1 mL min-1), showing no significant change in enantioresolution. These complementary approaches are suitable for routine quality control (CHIRALPAK IA), purification (CHIRALPAK IM) process monitoring and LC-MS impurity profiling (CHIRALPAK ID-3), and regulatory submissions, supporting current good manufacturing practices (cGMP) in the development of Odevixibat."

Journal • Cholestasis • Dermatology • Hepatology • Pruritus

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

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

Extraordinary Response to Odevixibat in an Adult Patient With Progressive Familial Intrahepatic Cholestasis Type 1 and Intractable Pruritus: A Case Report (ACG 2025) - "He had poor response to prior therapeutic measures including cholestyramine, ursodiol, sertraline, hydroxyzine, rifampin, and topical steroids. Hence, odevixibat should be considered as an option for refractory pruritus in adult patients with PFIC type 1.Figure: Table 1. Liver tests before (blue) and after (green) initiation of Bylvay (odevixibat)."

Case report • Clinical • Cholestasis • Dermatology • Fibrosis • Hepatology • Immunology • Liver Failure • Pruritus

Novel Use of Ileal Bile Acid Transport Inhibitor in Benign Recurrent Intrahepatic Cholestasis Refractory to Conventional Therapy (ACG 2025) - "Even though he responded well initially to pharmacotherapy including cholestyramine, ursodeoxycholic acid and rifampin, he subsequently required placement of nasobiliary drain during these episodes for symptom control...He was then treated off-label with IBAT inhibitor Odevixibat at a dose of 2400 mcg daily starting at onset of pruritic episodes with prompt and complete symptom relief and normalization of liver biochemical tests and serum bile acid levels...IBAT inhibitors are a novel class of drugs approved recently for the treatment of pruritus in patients 3 months of age and older with PFIC and Alagille's syndrome. Our case illustrates the potential role of IBAT inhibitors as a safe, effective and well-tolerated treatment option in aborting pruritic episodes in patients with BRIC unresponsive to conventional therapy, highlighting the potential applicability of this class of medications in a wider spectrum of genetic cholestatic disorders."

Cholestasis • Dermatology • Hepatology • Pruritus

Scratching the Surface: Successful Treatment of Severe Intrahepatic Cholestasis of Pregnancy With an Ileal Bile Acid Transporter Inhibitor (IBAT) (ACG 2025) - "Current standard of care include treatment with ursodeoxycholic acid (UDCA) which may be insufficient in severe cases...She was started on UDCA, cholestyramine and hydroxyzine...Despite maximal doses of UDCA, she continued to have worsening pruritus and rising BA with peak of 364 µmol/L in her third trimester.After discussion with maternal fetal medicine, she received Odevixibat though expanded access...While further study is needed, our case report demonstrated improvement in pruritus and serum BA in our patient with refractory ICP. This supports the need for potential for IBAT inhibitors as adjunctive therapy in select, high-risk ICP patient unresponsive to conventional therapy."

Cholestasis • Dermatology • Fibrosis • Gynecology • Hepatology • Immunology • Infectious Disease • Inflammation • Pruritus • ABCB1

ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - P3 | N=70 | Recruiting | Sponsor: Albireo, an Ipsen Company | Trial completion date: Apr 2026 ➔ Dec 2026 | Trial primary completion date: Apr 2026 ➔ Dec 2026

Trial completion date • Trial primary completion date

An update on novel investigational agents for the treatment of primary biliary cholangitis. (PubMed, Expert Opin Investig Drugs) - "While ursodeoxycholic acid (UDCA) remains the first-line treatment, up to 40% of patients show an inadequate response...Obeticholic acid (OCA), a farnesoid X receptor agonist, was initially approved but recently lost its marketing authorization in the EU due to an unfavorable risk-benefit balance. Fibrates, particularly bezafibrate and fenofibrate, have shown promising results in improving biochemical markers and reducing pruritus, although they remain off-label. We here focus on new FDA- and EMA-approved therapies, including the PPAR agonists elafibranor and seladelpar, which demonstrate improved biochemical response and, in the case of seladelpar, a significant reduction in pruritus. Additional investigational agents include NOX inhibitors such as setanaxib, IBAT inhibitors like linerixibat and odevixibat, and golexanolone, targeting fatigue through modulation of GABAergic neurotransmission. Despite advances, challenges remain in treatment personalization, access to..."

Journal • Review • Dermatology • Fatigue • Fibrosis • Gastroenterology • Hepatology • Immunology • Liver Cirrhosis • Liver Failure • Primary Biliary Cholangitis • Pruritus

BOLD AND BOLD-EXT, THE FIRST GLOBAL PHASE III CLINICAL PROGRAM TO EVALUATE THE LONG-TERM EFFICACY AND SAFETY OF ODEVIXIBAT IN INFANTS WITH BILIARY ATRESIA AFTER KASAI HEPATOPORTOENTEROSTOMY – STUDY DESIGN AND CURRENT STATUS (AASLD 2025) - P3 | "BOLD and BOLD-EXT will provide the first data on the use of odevixibat to target disease pathogenesis of biliary atresia in infants and children post-Kasai HPE. Results from BOLD-EXT will help to determine whether odevixibat is efficacious and protects the native liver in the long term. Reference: 1."

Clinical • P3 data • Hepatology • Liver Failure • Pediatrics

PREDICTING RESPONSE TO ODEVIXIBAT TREATMENT AND COMPARISON BETWEEN CLASSICAL TYPES AND RARER FORMS OF CHILDREN WITH PFIC (AASLD 2025) - "In our cohort, the majority of patients responded well to odevixibat treatment; higher percentages of response were recorded among children with rarer forms. Reduction of sBA levels of around 50% from baseline value at 1 and 3 months is associated with a full response to OT at 6 months. This information could be useful to plan prosecution or prompt discontinuation of treatment after 6 months of odevixibat."

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

ODEVIXIBAT RAPIDLY REDUCES PRURITUS AND SERUM BILE ACIDS IN CHILDREN WITH BILIARY ATRESIA AND CHRONIC CHOLESTASIS FOLLOWING KASAI PORTOENTEROSTOMY (AASLD 2025) - "This is the first report of Odevixibat in children with BAtr. Odevixibat is highly effective and safe in cholestatic pruritus in children with BAtr and residual cholestasis after KPE."

Clinical • Cardiovascular • Cholestasis • Dermatology • Fibrosis • Hepatology • Immunology • Liver Cirrhosis • Pediatrics • Portal Hypertension • Pruritus

EFFICACY AND CLINICAL OUTCOMES WITH ODEVIXIBAT IN PATIENTS WITH MDR3 DEFICIENCY: RESULTS: FROM THE PEDFIC 2 PHASE III, OPEN-LABEL EXTENSION STUDY (AASLD 2025) - P3 | "At BL: 5 patients were receiving ursodeoxycholic acid (UDCA), and 1 rifampicin, 3 patients had cirrhosis; all patients had abnormal liver tests. These data suggest ODX, in addition to UDCA, may have clinical benefit, providing symptomatic relief in patients with MDR3 deficiency; further investigation is warranted."

Clinical • Clinical data • P3 data • Cardiovascular • Cholestasis • Dermatology • Fibrosis • Hematological Disorders • Hepatology • Immunology • Portal Hypertension • Pruritus • Thrombocytopenia • ABCB4

ODEVIXIBAT TREATMENT IN PATIENTS WITH ALAGILLE SYNDROME: CHARACTERIZATION OF OUTCOMES BY SERUM BILE ACID LEVELS AFTER 24 WEEKS OF TREATMENT IN AN INTEGRATED ANALYSIS FROM ASSERT AND ASSERT-EXT (AASLD 2025) - P3 | "Patients with ALGS treated with ODX achieved clinically meaningful improvements in pruritus by Week 24 that were sustained long-term, independent of sBA level at Week 24. Higher bilirubin and sBA levels at BL, indicating more advanced disease, were seen in patients who did not achieve sBA ≤102 µmol/L after 24 weeks of treatment with ODX. Further study is warranted to explore sBA thresholds predictive of clinical outcomes in patients with ALGS receiving ODX."

Clinical • Dermatology • Hepatology • 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

Odevixibat after liver transplant in patients with progressive familial intrahepatic cholestasis type 1: A case series. (PubMed, J Pediatr Gastroenterol Nutr) - "Overall, the majority of patients with PFIC1 post-LT complications in this case series experienced improvements in diarrhea and daily activities with odevixibat. Treatment with odevixibat following LT also appeared to reduce steatosis in some patients. Further studies, particularly those with a prospective design, are needed to confirm these findings."

Journal • Cholestasis • Hepatology • Transplantation

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

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: Ipsen

New trial • Cholestasis • Hepatology

Odevixibat improves pruritus and bile acid level in Alagille syndrome: A case report. (PubMed, World J Gastrointest Pharmacol Ther) - "This case demonstrates the use of ileal bile acid transporter inhibitors treating pruritus and improving patient QoL. It has potential to delay the need for liver transplantation. However more long-term studies are needed to help clinicians better understand the benefit of this new treatment."

Journal • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Pediatrics • Pruritus • Transplantation • JAG1

Health Canada Approves Bylvay (odevixibat) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (PRNewswire) - "Health Canada's approval of Bylvay for ALGS was supported by data from the global Phase III ASSERT study, which demonstrated the efficacy of Bylvay in reducing cholestatic pruritus associated with ALGS within weeks of treatment."

Canada approval • Genetic Disorders

Odevixibat treatment reverses severe phenotype of PFIC in a young adult: A real-life experience beyond the genetic diagnosis. (PubMed, JHEP Rep) - No abstract available

Journal

PEDFIC 2: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (clinicaltrials.gov) - P3 | N=116 | Active, not recruiting | Sponsor: Albireo, an Ipsen Company | Trial completion date: Mar 2025 ➔ Aug 2025

Trial completion date • Cholestasis • Hepatology