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4 Diseases |  |
6 Trials |
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6 Trials |  |
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- || Bylvay (odevixibat) / Ipsen
Review, Journal: Exploring Odevixibat's efficacy in alagille syndrome: insights from recent clinical trials and IBAT Inhibitor Experiences. (Pubmed Central) - Sep 5, 2024
IBAT inhibition has been shown to reduce serum bile acids and pruritus in trials of cholestatic liver diseases in children including ALGS. Odevixibat or IBAT inhibitor should be considered as a first-line treatment for ALGS to improve pruritis, quality of life and liver-related outcomes including absence of liver transplant, surgical biliary diversion, hepatic decompensation, and death.
- | Bylvay (odevixibat) / Ipsen
Journal: Odevixibat as an adjunctive treatment for refractory pruritus in rare variants of cholestatic liver disease. (Pubmed Central) - Aug 16, 2024
There were no side effects reported and it was well tolerated in all. We suggest that Odevixibat may be used as an adjunctive drug in refractory pruritus and could be started early in the course of disease if clinically and phenotypically indicated.
- | Bylvay (odevixibat) / Ipsen
Trial completion date, Trial primary completion date: BOLD-EXT: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (clinicaltrials.gov) - Aug 6, 2024
P3, N=180, Enrolling by invitation, Sponsor: Albireo, an Ipsen Company
We suggest that Odevixibat may be used as an adjunctive drug in refractory pruritus and could be started early in the course of disease if clinically and phenotypically indicated. Trial completion date: Dec 2026 --> Aug 2028 | Trial primary completion date: Dec 2024 --> Jul 2028
- || Review, Journal: Transporter Proteins as Therapeutic Drug Targets-With a Focus on SGLT2 Inhibitors. (Pubmed Central) - Jul 13, 2024
Probenecid can be considered to increase uric acid excretion in the urine mainly via the inhibition of urate transporter 1 (URAT1), and due to pharmacokinetic interactions involving organic anion transporters 1 and 3 (OAT1 and OAT3), it modifies renal excretion of penicillins or ciprofloxacin as well as nephrotoxicity of cidofovir. This review discusses clinically approved drugs that affect membrane/drug transporter function.
- | Bylvay (odevixibat) / Ipsen
Enrollment closed: Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) (clinicaltrials.gov) - Jul 11, 2024
P3, N=254, Active, not recruiting, Sponsor: Albireo, an Ipsen Company
This review discusses clinically approved drugs that affect membrane/drug transporter function. Recruiting --> Active, not recruiting
- | Bylvay (odevixibat) / Ipsen
Enrollment open, Enrollment change, Trial completion date, Trial primary completion date: ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Jun 23, 2024
P3, N=70, Recruiting, Sponsor: Albireo, an Ipsen Company
Recruiting --> Active, not recruiting Active, not recruiting --> Recruiting | N=50 --> 70 | Trial completion date: Mar 2025 --> Apr 2027 | Trial primary completion date: Feb 2024 --> Oct 2026
- ||| Bylvay (odevixibat) / Ipsen
P3 data, Journal: Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. (Pubmed Central) - Jun 13, 2024
P3
Odevixibat could be an efficacious non-surgical intervention to improve pruritus, reduce serum bile acids, and enhance the standard of care in patients with Alagille syndrome. Longer-term safety and efficacy data of odevixibat in this population are awaited from the ongoing, open-label ASSERT-EXT study.
- Bylvay (odevixibat) / Ipsen
Efficacy and safety of odevixibat in a subgroup of adult patients with progressive familial intrahepatic cholestasis in the PEDFIC 2 study (Track Hub: General Hepatology) - May 14, 2024 - Abstract #EASLILC2024EASL_ILC_2787;
P3
Most TEAEs with odevixibat in adults with PFIC were mild or moderate in severity, and none were considered to be drug related. These findings suggest that odevixibat treatment in adult patients with PFIC warrants further clinical investigation.
- |||| Bylvay (odevixibat) / Ipsen
Trial primary completion date, Adverse events: Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation (clinicaltrials.gov) - Apr 4, 2024
P=N/A, N=20, Recruiting, Sponsor: Ipsen
These findings suggest that odevixibat treatment in adult patients with PFIC warrants further clinical investigation. Trial primary completion date: Sep 2032 --> Sep 2031
- Bylvay (odevixibat) / Ipsen
Efficacy and safety of odevixibat in a subgroup of adult patients with progressive familial intrahepatic cholestasis in the PEDFIC 2 study (Poster Area) - Apr 2, 2024 - Abstract #EASLILC2024EASL_ILC_761;
P3
Most TEAEs with odevixibat in adults with PFIC were mild or moderate in severity, and none were considered to be drug related. These findings suggest that odevixibat treatment in adult patients with PFIC warrants further clinical investigation.
- | Bylvay (odevixibat) / Ipsen
Journal, Metastases: Odevixibat treatment in an adult patient with advanced icteric progressive cholestatic liver disease. (Pubmed Central) - Feb 20, 2024
These findings suggest that odevixibat treatment in adult patients with PFIC warrants further clinical investigation. No abstract available
- |||||| Bylvay (odevixibat) / Ipsen
New trial, Adverse events: Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation (clinicaltrials.gov) - Feb 15, 2024
P=N/A, N=20, Recruiting, Sponsor: Ipsen
- || Bylvay (odevixibat) / Ipsen
Trial completion date: PEDFIC 2: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (clinicaltrials.gov) - Jan 17, 2024
P3, N=116, Active, not recruiting, Sponsor: Albireo
No abstract available Trial completion date: May 2024 --> Mar 2025
- | Bylvay (odevixibat) / Ipsen
Journal: Loss of SLC27A5 Activates Hepatic Stellate Cells and Promotes Liver Fibrosis via Unconjugated Cholic Acid. (Pubmed Central) - Jan 15, 2024
The re-expression of hepatic SLC27A5 by an adeno-associated virus or the reduction of CA levels in the liver using A4250, an apical sodium-dependent bile acid transporter (ASBT) inhibitor, ameliorates liver fibrosis in Slc27a5 mice. In conclusion, SLC27A5 deficiency in mice drives hepatic fibrosis through CA-induced activation of HSCs, highlighting its significant implications for liver fibrosis treatment.
- || Bylvay (odevixibat) / Ipsen
Trial completion date: ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Dec 10, 2023
P3, N=63, Active, not recruiting, Sponsor: Albireo
In conclusion, SLC27A5 deficiency in mice drives hepatic fibrosis through CA-induced activation of HSCs, highlighting its significant implications for liver fibrosis treatment. Trial completion date: Mar 2024 --> Mar 2025
- |||||| Bylvay (odevixibat) / Ipsen
Enrollment open, Trial completion date, Trial primary completion date: Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) (clinicaltrials.gov) - Oct 19, 2023
P3, N=245, Recruiting, Sponsor: Albireo
Trial completion date: Mar 2024 --> Mar 2025 Active, not recruiting --> Recruiting | Trial completion date: Jun 2024 --> Jul 2026 | Trial primary completion date: May 2024 --> Jun 2026
- Bylvay (odevixibat) / Ipsen
INDIVIDUAL PRURITUS AND BILE ACID RESPONSES OVER TIME WITH ODEVIXIBAT TREATMENT: POOLED DATA FROM THE PHASE 3 ASSERT AND ASSERT-EXT STUDIES IN PATIENTS WITH ALAGILLE SYNDROME (Room 304/306) - Oct 11, 2023 - Abstract #AASLD2023AASLD_947;
P3
In patients with ALGS, odevixibat treatment for up to 36 weeks resulted in improvements in pruritus and reduced BA levels; these changes occurred rapidly, with effects sustained over time. Most TEAEs with odevixibat were related to diarrhea and were mild or moderate in severity.
- | Bylvay (odevixibat) / Ipsen
Preclinical, Journal: Intestinal in vitro transport assay combined with physiologically based kinetic modeling as a tool to predict bile acid levels in vivo. (Pubmed Central) - Aug 2, 2023
For this proof-of-principle study we used the selective ASBT-inhibitor odevixibat (ODE) as a model compound...Our simulations match in vivo data and provide a first proof-of-principle for the incorporation of active intestinal bile acid uptake in a bile acid PBK model. This approach could in future be of use to predict the effects of other ASBT-inhibitors on plasma and intestinal bile acid levels.
- Livmarli (maralixibat) / Mirum Pharma, Takeda, Bylvay (odevixibat) / Ipsen
Ileal Bile Acid Transporter Inhibition for Treatment of Pruritis in Progressive Familial Intrahepatic Cholestasis (Exhibit Hall) - Jul 29, 2023 - Abstract #ACG2023ACG_3066;
Additionally, novel therapies like IBAT inhibitors, specifically Odevixibat and Maralixibat, which received FDA approval in 2021, should be considered. Physicians caring for patients with this condition should be aware of these novel drug classes' benefits and side effects.
- | Bylvay (odevixibat) / Ipsen
Journal: Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis. (Pubmed Central) - Jul 17, 2023
P3
Odevixibat is an orally administered medication that shunts bile acids away from the liver. The current study, called PEDFIC 2, suggested that odevixibat can improve the problematic signs and symptoms of progressive familial intrahepatic cholestasis and was generally safe for patients.
- || Bylvay (odevixibat) / Ipsen
Review, Journal: Surgical versus Medical Management of Progressive Familial Intrahepatic Cholestasis-Case Compilation and Review of the Literature. (Pubmed Central) - Jun 28, 2023
(4) Limited data on IBAT in selected patients with PFIC show safety and effectiveness, although surgical methods should still be considered as a successful bridging procedure. Further studies to evaluate a possible combination of IBAT and SBD in PFIC are warranted and treatment decision should be discussed in an interdisciplinary board.
- |||| Bylvay (odevixibat) / Ipsen
Clinical: Breaking news this afternoon from @HCPLiveNews: Odevixibat has been approved for pediatric patients with #Alagille syndrome! Hear from experts including @Dr_NOvchinsky and @KowdleyMd on what this means for the rare liver disease: https://t.co/eHP0eMJDuH (Twitter) - Jun 13, 2023
- Bylvay (odevixibat) / Ipsen
Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with 96 (General hepatology) - Jun 7, 2023 - Abstract #EASLILC2023EASL_ILC_2813;
- Bylvay (odevixibat) / Ipsen
Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with ?96 weeks of treatment (Hall 3A) - May 30, 2023 - Abstract #BSG2023BSG_77;
P3
Odevixibat treatment for ?96 weeks was associated with improvements in sBAs, transaminase levels, and growth in patients with PFIC. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Ipsen
Analysis of long-term treatment effects of odevixibat on clinical outcomes in children with progressive familial intrahepatic cholestasis in odevixibat clinical studies vs external controls from the NAPPED database (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_873;
Odevixibat was generally well tolerated. Odevixibat treatment is associated with higher EFS in patients with PFIC without prior SBD, upon comparison to matched, non
- Bylvay (odevixibat) / Ipsen
Odevixibat therapy in patients with MYO5B mutations: a retrospective case series (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_864;
Odevixibat treatment in patients with PFIC10 led to a rapid and sustained resolution of pruritus and a significant decrease in or normalisation of sBA levels, supporting the effectiveness of odevixibat. Treatment with odevixibat should be further evaluated in patients with PFIC associated with MYO5B gene mutations.
- Bylvay (odevixibat) / Ipsen
Real-world experience of Odevixibat in adults with genetic disorders of cholestasis (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_855;
Treatment with odevixibat should be further evaluated in patients with PFIC associated with MYO5B gene mutations. In adults with genetic disorders of cholestasis, odevixibat treatment appears to be safe, well tolerated, and associated with a reduction in pruritus intensity.
- Bylvay (odevixibat) / Ipsen
Outcomes in adult patients with progressive familial intrahepatic cholestasis treated with odevixibat: subgroup analysis from the PEDFIC 2 study (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_854;
Several adult patients with PFIC enrolled in the ongoing PEDFIC 2 study have experienced clinical benefits with odevixibat, including reductions in sBAs, pruritus, and/or improvements in hepatic parameters. Odevixibat was generally well tolerated in this adult population.
- Bylvay (odevixibat) / Ipsen
Odevixibat therapy following liver transplantation in patients with FIC1-deficient progressive familial intrahepatic cholestasis: a retrospective case series (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_853;
The real-world data presented here indicate that odevixibat can improve diarrhoea and QoL after LT in patients with PFIC1. In addition, 2 of 4 patients where biopsy was performed following odevixibat initiation had improved steatosis (odevixibat duration: 11 and 17 months, respectively, at last data collection).
- Bylvay (odevixibat) / Ipsen
Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with 96 (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_852;
Odevixibat was generally well tolerated. Table:
- Bylvay (odevixibat) / Ipsen
Efficacy and safety outcomes with odevixibat treatment: Pooled data from the phase 3 ASSERT and ASSERT-EXT studies in patients with Alagille syndrome (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_848;
Consistent with effects on pruritus and BA levels, there were significant changes in autotaxin and p-C4 levels with odevixibat. Odevixibat treatment was generally well tolerated in patients with ALGS.
- ||||| Bylvay (odevixibat) / Ipsen
Trial completion: ASSERT: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Apr 10, 2023
P3, N=52, Completed, Sponsor: Albireo
Odevixibat treatment was generally well tolerated in patients with ALGS. Active, not recruiting --> Completed
- Bylvay (odevixibat) / Albireo
Odevixibat therapy in patients with FIC1-deficient progressive familial intrahepatic cholestasis and severe diarrhoea following liver transplantation: A retrospective case series () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_141;
Active, not recruiting --> Completed Attempts to treat the diarrhoea with cholestyramine (patients 2 and 3) and Oralp
- Bylvay (odevixibat) / Albireo
Odevixibat treatment in a patient with undefined cholestasis and no unified genetic diagnosis: A case report () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_140;
Table. Hepatic Parameters Over Time in a Patient With Undefined Cholestasis and No Unifying Genetic Diagnosis ALT, IU/L AST, IU/L GGT, IU/L Total Bilirubin, ?mol/L Within months of birth 307 274 158 120 Age 5 (before treatment) 219 154 230 70 Time Frame ALT, alanine aminotransferase; AST, aspartate aminotransferase; GGT, gamma-glutamyl transferase.
- Bylvay (odevixibat) / Albireo
Native liver survival in odevixibat serum bile acid responders: Data from PEDFIC studies in patients with progressive familial intrahepatic cholestasis () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_139;
Figure. Native Liver Survival in Serum Bile Acid Responders, Partial Responders, and Nonresponders to Odevixibat in the PEDFIC Studies.
- Bylvay (odevixibat) / Albireo
Odevixibat: Are we changing the status quo in cholestatic liver disease? (Hanover 3) - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_81;
Native Liver Survival in Serum Bile Acid Responders, Partial Responders, and Nonresponders to Odevixibat in the PEDFIC Studies. Sponsored by Albireo
- Bylvay (odevixibat) / Albireo
Long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: results with ?96 weeks of treatment () - Mar 27, 2023 - Abstract #BSPGHAN2023BSPGHAN_71;
P3
Inferential statistics were not calculated for hepatic parameters or growth. ALT, alanine aminotransferase; AST, aspartate aminotransferase; PFIC, progressive familial intrahepatic cholestasis; sBA, serum bile acid.
- Economic Modeling Insights for Ultra Orphan Drugs Based on Review of NICE Highly Specialized Technology (HST) Assessment Reports () - Mar 9, 2023 - Abstract #ISPOR2023ISPOR_1247;
Previous HST submissions provide valuable insights and lessons for future ultra orphan economic modeling. In many instances NICE mentioned benefits that were not captured by the sponsor and also allowed or suggested methods to address lack of evidence in a rare disease setting.
- |||| Bylvay (odevixibat) / Albireo
FDA event, PDUFA date: $ALBO FDA Grants June 15, 2023 PDUFA Date to Albireo for Bylvay® in Alagille Syndrome https://t.co/iEGxUIcJvI (Twitter) - Feb 13, 2023
- || Livmarli (maralixibat) / Mirum Pharma, Bylvay (odevixibat) / Albireo
Retrospective data, Review, Journal: Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis. (Pubmed Central) - Dec 24, 2022
The efficacy of IBAT inhibitors across current trials was noted. Future trials may focus on the optimization of dosing regimens, considering gastrointestinal side effects and drug-induced ALT elevation in AGLS patients.
- ||| Bylvay (odevixibat) / Albireo
$ALBO Albireo Submits Bylvay® Alagille Syndrome Regulatory Filings to FDA and EMA https://t.co/EVpGOyTFNb (Twitter) - Dec 19, 2022
- | Bylvay (odevixibat) / Albireo
Journal: Odevixibat: an investigational inhibitor of the ileal bile acid transporter (IBAT) for the treatment of biliary atresia. (Pubmed Central) - Nov 29, 2022
In principle, however, the time until diagnosis and surgical treatment of BA should still be kept as short as possible in order to minimize liver remodeling before medical intervention can be initiated. The new group of IBAT inhibitors may add to the medical options in limiting disease progression in BA.
- ||||| Bylvay (odevixibat) / Ipsen
Enrollment closed: Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) (clinicaltrials.gov) - Nov 25, 2022
P3, N=205, Active, not recruiting, Sponsor: Albireo
The new group of IBAT inhibitors may add to the medical options in limiting disease progression in BA. Recruiting --> Active, not recruiting
- | Bylvay (odevixibat) / Albireo
Journal: Odevixibat: a promising new treatment for progressive familial intrahepatic cholestasis. (Pubmed Central) - Nov 22, 2022
IBAT inhibition with therapeutics such as odevibixat have shown that it is well-tolerated and efficacious in mitigating itch and reducing serum bile acid levels. While the few early published trials with odevixibat have shown good efficacy, what remains to be seen is long-term, sustainable improvement and if or how these medications will supplement or replace the current medical and surgical therapies available for managing PFIC disorders.
- | Bylvay (odevixibat) / Albireo
Odevixibat is approved for PFIC on basis of the PFIC1 pivotal trial https://t.co/hPLmPM4lRB #TLM22 (Twitter) - Nov 7, 2022
- Bylvay (odevixibat) / Albireo
EFFICACY AND SAFETY OF ODEVIXIBAT IN PATIENTS WITH ALAGILLE SYNDROME: TOP-LINE RESULTS FROM ASSERT, A PHASE 3, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY (Ballroom ABC) - Nov 2, 2022 - Abstract #AASLD2022AASLD_2393;
P3
ALGS is a disease with high unmet needs, and more noninvasive treatment options that address the potentially serious signs and symptoms of ALGS would be valuable. Top-line, unblinded efficacy and safety data from the ASSERT study, including the effects of odevixibat versus placebo on pruritus, serum bile acids, and TEAEs will be presented.
- Bylvay (odevixibat) / Albireo
ODEVIXIBAT TREATMENT IN RESPONSIVE PATIENTS WITH BILE SALT EXPORT PUMP DEFICIENCY RESTORES BILIARY BILE ACID SECRETION, AS INDICATED BY SERUM BILE ACID COMPOSITION (Ballroom ABC) - Nov 2, 2022 - Abstract #AASLD2022AASLD_2392;
P3
Exclusively in Rs, odevixibat treatment was associated with a strongly increased relative contribution of unconjugated BAs. Since unconjugated BAs originate from deconjugation of biliary BAs by the intestinal microbiome, our data indicate that odevixibat restored biliary BA secretion in treatment-responsive patients with BSEP deficiency.
- Bylvay (odevixibat) / Albireo
Product Theater: Relief Made Possible with Bylvay® (odevixibat) (Exhibit Hall A; Product Theater 2) - Nov 2, 2022 - Abstract #AASLD2022AASLD_2384;
This presentation will be followed by an interview with Dr. Alisha Mavis, Pediatric Hepatologist at Duke University, on her experience with prescribing Bylvay.
- Bylvay (odevixibat) / Albireo
LONG-TERM EFFICACY AND SAFETY OF ODEVIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: RESULTS WITH 96 WEEKS OR MORE OF TREATMENT () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2344;
Odevixibat treatment for ≥96 weeks was associated with durable clinical benefits in patients with PFIC, including improvements in sBA and transaminase levels and growth. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Albireo
ODEVIXIBAT THERAPY IN PATIENTS WITH FIC1-DEFICIENT PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS AND DIARRHEA FOLLOWING LIVER TRANSPLANTATION THAT IMPACTED DAILY ACTIVITIES: A RETROSPECTIVE CASE SERIES () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2340;
In patients with FIC1 deficiency, chologenic diarrhea after LT, which may be due to physiologic bile acid levels in the FIC1-deficient bowel, can be a frequent and severe symptom. The real-world data presented here indicate that odevixibat can improve diarrhea and quality of life in patients with PFIC1 and severe post-LT diarrhea.