Bylvay (odevixibat) / Ipsen - 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

Pretreatment serum bile acid composition and predictability of subsequent response to odevixibat in patients with bile salt export pump (BSEP) deficiency. (PubMed, Hepatology) - "Higher pretreatment serum levels of unconjugated CA and CDCA are associated with subsequent response to odevixibat in patients with BSEP deficiency. Response to odevixibat may be related to residual biliary bile acid secretion capacity in patients with BSEP deficiency."

Journal • Cholestasis • Hepatology

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

Odevixibat for Episodic Intrahepatic Cholestasis due to Biallelic Mutations in ATP8B1: A Case Series. (PubMed, Liver Int) - "In this case series, most patients with episodic IC associated with biallelic mutations in ATP8B1 treated with odevixibat during a relapse of cholestasis had clinical improvement, including reductions in serum bile acids and positive impacts on pruritus and quality of life. Treatment did not appear to have a preventive effect on future episodes. More studies are needed to confirm these findings."

Journal • Cholestasis • CNS Disorders • Dermatology • Fatigue • Hepatology • Pain • Pruritus • Rare Diseases

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

Food and Drug Administration Approval Summary: Odevixibat (Bylvay) for the Treatment of Pruritus With Progressive Familial Intrahepatic Cholestasis. (PubMed, Gastro Hep Adv) - "The most common adverse reactions included diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The benefit-risk assessment for odevixibat for the treatment of pruritus in the labeled population was considered favorable."

Journal • Review • Cholestasis • Dermatology • Hepatology • Liver Failure • Pain • Pediatrics • Pruritus • Rare Diseases

FAT-SOLUBLE VITAMIN DEFICIENCY IN PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS PATIENTS WHO ARE TREATED WITH ODEVIXIBAT (ESPGHAN 2025) - "Conclusions This study shows no significant association between odevixibat treatment and development of fat-soluble vitamin deficiency in PFIC patients, while vitamin supplementation doses were on average increased after the start of odevixibat treatment. It remains therefore relevant to monitor fat-soluble vitamin levels in PFIC patients to maintain adequate plasma levels."

Clinical • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Pruritus

LONG-TERM EFFICACY AND SAFETY OF ODEVIXIBAT IN PATIENTS WITH ALAGILLE SYNDROME: POOLED RESULTS FROM PHASE III RANDOMIZED DOUBLE-BLIND ASSERT AND OPEN-LABEL ASSERT-EXT STUDIES (ESPGHAN 2025) - P3 | "AASLD 2024. Abstract 50 (Oral)"

Clinical • P3 data • Dermatology • Hepatology • Pruritus

PREDICTING RESPONSE TO ODEVIXIBAT TREATMENT IN CHILDREN WITH DIFFERENT TYPES OF PFIC (ESPGHAN 2025) - "Conclusions In our cohort, 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 can be useful to plan prosecution or prompt discontinuation of treatment after 6 months of odevixibat."

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

LONG-TERM QUALITY OF LIFE OUTCOMES FOR PATIENTS WITH ALAGILLE SYNDROME TREATED WITH ODEVIXIBAT: POOLED RESULTS FROM PHASE III RANDOMIZED DOUBLE-BLIND ASSERT AND OPEN-LABEL ASSERT-EXT STUDIES (ESPGHAN 2025) - P3 | "AASLD 2024. Abstract 50 (Oral)"

Clinical • HEOR • P3 data • CNS Disorders • Dermatology • Hepatology • Pediatrics • Pruritus • Sleep Disorder

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

ODEVIXIBAT TREATMENT IN PATIENTS WITH BILE SALT EXPORT PUMP DEFICIENCY: SUSTAINED EFFICACY IN AN INTEGRATED ANALYSIS OF RESPONDERS (PEDFIC 1 AND 2) (ESPGHAN 2025) - P3 | "sBA responders had early and sustained reductions in sBA and pruritus. Patient-level data and additional endpoints (including liver function tests and safety) will be presented."

Clinical • Cholestasis • Dermatology • Hepatology • Pruritus

PRELIMINAR EXPERIENCE WITH ODEVIXIBAT – SIMILAR PATIENTS WITH DIFFERENT RESPONSES IN PATIENTS WITH PFIC1 (ESPGHAN 2025) - "All children experienced refractory jaundice and pruritus with chronic lichenification lesions, unresponsive to ursodeoxycholic acid, cholestyramine, rifampicin, and hydroxyzine. Conclusions These cases align with the 44% improvement in pruritus and serum bilirubin levels reported in phase 3 study 2 . However, the dichotomous response to ODX observed in this small series underscore the need for further investigation."

Clinical • Cardiovascular • Celiac Disease • Cholestasis • Dermatology • Heart Failure • Hepatology • Immunology • Pruritus • ABCB1

THE BIOCHEMICAL AND NUTRITIONAL EFFECTS OF ODEVIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC) AND ALAGILLE SYNDROME (ALGS) (ESPGHAN 2025) - "One child has been listed for liver transplant with improved MUAC. In this cohort, we have identified that biochemical parameters do not necessarily improve after odevixibat, however nutritional status does improve which can be associated with better long-term outcomes."

Clinical • Cholestasis • Hepatology • ABCB1

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

ODEVIXIBAT THERAPY ALLEVIATES REFRACTORY PRURITUS IN PFIC 7 CAUSED BY USP53: A CASE REPORT (ESPGHAN 2025) - "The patient was initially managed with UDCA, cholestyramine, rifampin. Conclusions This report documents the first case of PFIC Type 7 managed with odevixibat described in the literature. It highlights the remarkable efficacy of odevixibat in alleviating refractory pruritus and lowering bile acid levels in PFIC 7, while underscoring the critical need for uninterrupted access to therapy."

Case report • Clinical • Cholestasis • Dermatology • Hepatology • Pruritus • TP53

SUCCESSFUL TREATMENT OF CHOLESTATIC PRURITUS WITH ODEVIXIBAT IN VANISHING BILE DUCT SYNDROME: A NOVEL APPROACH (ESPGHAN 2025) - "An increased dose of ursodeoxycholic acid (UDCA) resulted in clinical worsening and higher sBA levels. This case highlights the potential for IBAT inhibitors in addressing unmet needs in the management of rare cholestatic disorders. Further research is essential to evaluate its broader application and long-term outcomes in cholestatic conditions."

Cholestasis • Dermatology • Hematological Malignancies • Hepatology • Lymphoma • Oncology • Pediatrics • Pruritus

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

ODEVIXIBAT RAPIDLY REDUCES PRURITUS AND SERUM BILE ACIDS IN CHILDREN WITH BILIARY ATRESIA AND RESIDUAL CHOLESTASIS FOLLOWING KASAI PORTOENTEROSTOMY (ESPGHAN 2025) - "Conclusions 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

ODEVIXIBAT TREATMENT IN PATIENTS WITH FIC1 DEFICIENCY: SUSTAINED EFFICACY, PARTICULARLY IN PRURITUS, IN AN INTEGRATED ANALYSIS OF RESPONDERS (PEDFIC 1 AND PEDFIC 2) (ESPGHAN 2025) - No abstract available

Clinical • Dermatology • Pruritus

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

Odevixibat therapy in progressive familial intrahepatic cholestasis with MYO5B variants: a retrospective case series. (PubMed, Orphanet J Rare Dis) - "This case series indicates that treatment with odevixibat is effective in children with myosin 5B-related PFIC and encourages further research into the utility of this medication in rare forms of PFIC."

Journal • Retrospective data • Cholestasis • CNS Disorders • Dermatology • Gastroenterology • Gastrointestinal Disorder • Hepatology • Pruritus • Sleep Disorder

Ipsen to present new data across four rare liver diseases at EASL, including late-breaking data in PBC and PSC (Ipsen Press Release) - "Today, Ipsen...announced that seven abstracts with new data from its rare liver disease portfolio will be presented at the European Association for the Study of the Liver (EASL) congress...These include two late-breaking abstracts selected for poster presentation from Ipsen’s IQIRVO (elafibranor) in primary biliary cholangitis (PBC) and one late-breaking abstract selected for oral presentation and simultaneous publication in the Journal of Hepatology: Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized controlled trial, on elafibranor in primary sclerosing cholangitis (PSC). Two other abstracts describing the effect of IQIRVO non-invasive markers of fibrosis and bone health in PBC were also selected for poster presentation; In addition, two abstracts with data on odevixibat known in the EU as Bylvay for PFIC and Kayfanda for ALGS, will be shared as poster presentations."

Clinical data • Late-breaking abstract • Hepatology • Primary Biliary Cholangitis

Odevixibat in the Treatment of Progressive Familial Intrahepatic Cholestasis: A Systematic Literature Review for Brazilian's Health Technology Assessment (ISPOR 2025) - "One RCT demonstrated that ODX was an effective and well tolerated treatment in patients with PFIC, reducing pruritus and sBA over 24 weeks. Additional non-RCT evidence of long-term efficacy of ODX have also been published but are not included in this SLR, namely the results of the extension trial PEDFIC2, an open label, single-arm, 72-week study in patients with PFIC, showing sustained efficacy over time."

Review • Cholestasis • Dermatology • Hepatology • Pediatrics • Pruritus