 |
4 Diseases |  |
6 Trials |
|
6 Trials |  |
318 News |  |
| «123» |
- Bylvay (odevixibat) / Albireo
ODEVIXIBAT TREATMENT IN PATIENTS WITH RECURRENT EPISODIC CHOLESTASIS AND BIALLELIC MUTATIONS IN ATP8B1: A RETROSPECTIVE CASE SERIES () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2337;
In this case series, most patients with recurrent episodic cholestasis and ATP8B1 deficiency had clinical improvement on odevixibat during an episode, including improved sBA levels, pruritus, and impacts on daily life. In patients who continued on treatment, odevixibat failed to prevent recurrence of a cholestatic episode.
- Bylvay (odevixibat) / Albireo
EFFECT OF ODEVIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 WITH AT LEAST 1 SEVERE MUTATION (BSEP3 COMPOUND HETEROZYGOTES): POOLED DATA FROM THE PEDFIC 1 AND PEDFIC 2 STUDIES () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2334;
In patients who continued on treatment, odevixibat failed to prevent recurrence of a cholestatic episode. In the PEDFIC 1 and PEDFIC 2 studies, the majority of patients with PFIC2 with BSEP2/BSEP3 mutations responded to odevixibat treatment, as shown by substantial reductions in sBAs and/or pruritus severity.
- Bylvay (odevixibat) / Albireo
SERUM BILE ACID LEVELS, PRURITUS SCORES, AND GROWTH OVER TIME IN ODEVIXIBAT RESPONDERS: POOLED DATA FROM THE PEDFIC STUDIES IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2333;
P3
Mean improvements in growth were also observed. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Albireo
NATIVE LIVER SURVIVAL IN ODEVIXIBAT SBA RESPONDERS: DATA FROM THE PEDFIC STUDIES IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (Room 145) - Oct 23, 2022 - Abstract #AASLD2022AASLD_328;
P3
At 104 weeks after starting odevixibat treatment in PFIC patients, their survival with native liver appeared strongly associated with the decrease in sBA at 6 months. No patient in which odevixibat reduced sBA by at least 50% had been transplanted after 6 months of treatment.
- |||| Bylvay (odevixibat) / Ipsen
Enrollment closed, Trial completion date, Trial primary completion date: ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Oct 18, 2022
P3, N=63, Active, not recruiting, Sponsor: Albireo
No patient in which odevixibat reduced sBA by at least 50% had been transplanted after 6 months of treatment. Enrolling by invitation --> Active, not recruiting | Trial completion date: Oct 2023 --> Mar 2024 | Trial primary completion date: Oct 2023 --> Feb 2024
- |||| Bylvay (odevixibat) / Ipsen
Enrollment closed, Trial completion date, Trial primary completion date: PEDFIC 2: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (clinicaltrials.gov) - Oct 12, 2022
P3, N=120, Active, not recruiting, Sponsor: Albireo
Enrolling by invitation --> Active, not recruiting | Trial completion date: Oct 2023 --> Mar 2024 | Trial primary completion date: Oct 2023 --> Feb 2024 Recruiting --> Active, not recruiting | Trial completion date: Dec 2023 --> May 2024 | Trial primary completion date: Dec 2023 --> Mar 2024
- | Bylvay (odevixibat) / Albireo
Journal: Validation of the PRUCISION Instruments in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis. (Pubmed Central) - Oct 5, 2022
P3
Recruiting --> Active, not recruiting | Trial completion date: Dec 2023 --> May 2024 | Trial primary completion date: Dec 2023 --> Mar 2024 The PRUCISION ObsRO instrument is reliable, valid, and sensitive to change, supporting its use as a tool to measure pruritus and sleep disturbance in patients with PFIC and other pediatric cholestatic liver diseases.
- Bylvay (odevixibat) / Albireo
Benign Recurrent Intrahepatic Cholestasis (BRIC) Managed With Plasmapheresis (Crown Ballroom) - Sep 4, 2022 - Abstract #ACG2022ACG_3421;
Our case highlights the potential usefulness of this promising modality in improving pruritus and shortening the duration of attacks in BRIC/PFIC patients. Table: Labs on admission versus post-plasmapheresis sessions
- Value of Real-World Evidence for Treatments for Ultra Orphan or Rare Diseases: Lessons and Insights From Review of NICE Highly Specialized Technology Assessment Reports (2016-2022) (Virtual) - Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_329;
To our knowledge this is the first report describing use of RWE for ultra-rare disease HTA submissions. This analysis provides valuable lessons and insights for manufacturers to leverage RWE for HTA submission.
- ||| Bylvay (odevixibat) / Albireo
P3 data, Journal: Positive phase III results for odevixibat for progressive familial intrahepatic cholestasis. (Pubmed Central) - Aug 31, 2022
This analysis provides valuable lessons and insights for manufacturers to leverage RWE for HTA submission. No abstract available
- ||| Bylvay (odevixibat) / Albireo
P3 data, Journal: Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. (Pubmed Central) - Aug 11, 2022
P3
In children with PFIC, odevixibat effectively reduced pruritus and serum bile acids versus placebo and was generally well tolerated. Odevixibat, administered as once a day oral capsules, is a non-surgical, pharmacological option to interrupt the enterohepatic circulation in patients with PFIC.
- || Bylvay (odevixibat) / Albireo
Odevixibat Scratches the Itch of Kids' Rare Liver Disease https://t.co/ZZRjWfIujx (Twitter) - Jul 12, 2022
- || Bylvay (odevixibat) / Albireo
Odevixibat may reduce pruritus in kids with progressive familial intrahepatic cholestasis: Lancet https://t.co/2FvgB2FFK1 (Twitter) - Jul 11, 2022
- |||||||| Bylvay (odevixibat) / Ipsen
New P3 trial: BOLD-EXT: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia (clinicaltrials.gov) - Jun 22, 2022
P3, N=180, Enrolling by invitation, Sponsor: Albireo
- ||| Bylvay (odevixibat) / Ipsen
Trial completion date, Trial primary completion date: PEDFIC 2: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (clinicaltrials.gov) - May 10, 2022
P3, N=120, Recruiting, Sponsor: Albireo
Odevixibat, administered as once a day oral capsules, is a non-surgical, pharmacological option to interrupt the enterohepatic circulation in patients with PFIC. Trial completion date: Sep 2023 --> Dec 2023 | Trial primary completion date: Sep 2023 --> Dec 2023
- ||| Bylvay (odevixibat) / Ipsen
Trial completion date, Trial primary completion date: PEDFIC 2: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC (clinicaltrials.gov) - May 5, 2022
P3, N=120, Recruiting, Sponsor: Albireo
Trial completion date: Sep 2023 --> Dec 2023 | Trial primary completion date: Sep 2023 --> Dec 2023 Trial completion date: Dec 2021 --> Sep 2023 | Trial primary completion date: Dec 2021 --> Sep 2023
- Bylvay (odevixibat) / Albireo
LONG-TERM TREATMENT WITH ODEVIXIBAT IMPROVES MULTIPLE SLEEP PARAMETERS IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: A POOLED RESPONDER ANALYSIS FROM THE PHASE 3 PEDFIC STUDIES (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_935;
These effects occurred rapidly and continued over time. The improvement in sleep is likely linked to the improved pruritus observed in patients who responded to odevixibat.
- Bylvay (odevixibat) / Albireo
ODEVIXIBAT EFFECTS ON CHOLESTASIS-RELATED PARAMETERS: ANALYSIS OF POOLED DATA FROM THE PEDFIC 1 AND PEDFIC 2 STUDIES IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_934;
In general, responders had greater mean changes from baseline (ie, improvements) vs nonresponders in these hepatic laboratory parameters with long-term odevixibat treatment (Table) that started as early as week 4 and increased over time. Conclusion s: Patients with PFIC who responded to odevixibat treatment had sustained improvements in cholestasis-related parameters that were not observed to the same extent in treatment nonresponders.
- Bylvay (odevixibat) / Albireo
ODEVIXIBAT THERAPY IMPROVES CLINICALLY MEANINGFUL ENDPOINTS IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM THE PEDFIC 1 AND PEDFIC 2 TRIALS (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_933;
P3
Four patients had TEAEs leading to treatment discontinuation. Conclusion s: In children with PFIC, odevixibat treatment for up to 48 weeks was well tolerated and associated with clinically meaningful effects on sBAs, pruritus, growth, and sleep parameters.
- Bylvay (odevixibat) / Albireo
EFFICACY AND SAFETY OF ODEVIXIBAT IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS WITH PRIOR PARTIAL EXTERNAL BILIARY DIVERSION (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_932;
These improvements were observed despite elevated sBAs and pruritus at baseline, suggesting that some patients with poor response to PEBD could potentially respond to odevixibat treatment. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Albireo
RELATIONSHIPS BETWEEN DECREASES IN SERUM BILE ACIDS, PRURITUS, AND SLEEP DISTURBANCE SCORES WITH UP TO 72 WEEKS OF ODEVIXIBAT TREATMENT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_931;
Odevixibat was generally well tolerated. Odevixibat may be a noninvasive treatment option for patients with PFIC, a disease with high unmet needs.
- Bylvay (odevixibat) / Albireo
DISEASE BURDEN AND NATURAL HISTORY OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: BASELINE CLINICAL CHARACTERISTICS AMONG ODEVIXIBAT-TREATED PATIENTS IN THE PHASE 3 PEDFIC STUDIES (ePoster - DDW Virtual) - Apr 25, 2022 - Abstract #DDW2022DDW_930;
Conclusion s: The baseline characteristics, including cholestasis and impaired hepatic function, growth, and sleep, of this patient population illustrate the heterogeneity and complexity of PFIC. In addition, PFIC had a considerable impact on the QoL of patients and their caregivers.
- |||| Bylvay (odevixibat) / Ipsen
Enrollment closed, Trial completion date, Trial primary completion date: ASSERT: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Mar 31, 2022
P3, N=63, Active, not recruiting, Sponsor: Albireo
In addition, PFIC had a considerable impact on the QoL of patients and their caregivers. Recruiting --> Active, not recruiting | Trial completion date: Jul 2022 --> Oct 2022 | Trial primary completion date: Jun 2022 --> Oct 2022
- Bylvay (odevixibat) / Albireo
Efficacy and safety of odevixibat over 72 weeks of treatment in patients with progressive familial intrahepatic cholestasis (Poster Area) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_920;
At baseline, 82% of these 27 patients were using ursodeoxycholic acid (UDCA), 56% were using rifampicin, and 89% were using UDCA and/or rifampicin. Odevixibat was well tolerated and demonstrated durable clinical treatment benefits for up to 72 weeks in improving sBA levels, pruritus scores, hepatic parameters, and growth in patients with PFIC who were treated for at least 72 weeks.
- Bylvay (odevixibat) / Albireo
Analysis of quality of life, hepatic biochemical markers, and sleep in patients with progressive familial intrahepatic cholestasis who had a pruritus response with odevixibat treatment (Poster Area) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_917;
Overall, patients with PFIC with a pruritus response following treatment with odevixibat experienced improvements in sBAs, QoL, hepatic biochemical markers, and sleep parameters that were sustained over time. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Albireo
Changes in hepatic parameters, growth, sleep, and biochemical markers with odevixibat treatment across patients with various types of progressive familial intrahepatic cholestasis (Poster Area) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_916;
In patients with PFIC treated with odevixibat assessed to date, growth improved, autotaxin levels decreased, and C4 levels increased over time; changes in hepatic and sleep parameters in some PFIC types were more variable (eg, PFIC3, PFIC6), but these conclusions may be limited by the small size of these subgroups, particularly at later timepoints. Odevixibat was generally well tolerated.
- Bylvay (odevixibat) / Albireo
Total, primary, and secondary serum bile acid changes and pruritus improvement during odevixibat treatment in patients with progressive familial intrahepatic cholestasis (Meeting Point 3) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_915;
A sBA PR was not associated with a mean decrease in pruritus with concomitant UDCA use. All sBA R groups included patients for whom pruritus R did not correlate with sBA R.
- Bylvay (odevixibat) / Albireo
Improvements in quality of life in odevixibat responders and nonresponders: an analysis of pooled data from the PEDFIC 1 and PEDFIC 2 studies (Poster Area) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_914;
Odevixibat Rs and, particularly, their families, experienced QoL improvements that were sustained over time; QoL for NRs and their families was largely unchanged with up to 72 weeks of treatment. Further analysis of specific domains within PedsQL and FI that were most highly impacted in Rs is ongoing.
- Bylvay (odevixibat) / Albireo
Correlations between Patient- and Caregiver-Reported Pruritus from PEDFIC 1: Evaluation of Prucision Data in Patients with Progressive Familial Intrahepatic Cholestasis (Virtual) - Mar 8, 2022 - Abstract #ISPOR2022ISPOR_1228;
P3
In the phase 3 PEDFIC 1 study (NCT03566238), odevixibat, an ileal bile acid transporter inhibitor, reduced pruritus compared with placebo based on caregiver report in children with PFIC using the PRUCISION scale... PRO pruritus scores significantly correlated with ObsRO pruritus scores at all time points in PEDFIC 1, highlighting the consistency between the PRUCISION instruments for measuring pruritus severity in patients with PFIC.
- || Korsuva IV (difelikefalin IV) / Cara Therap, Maruishi Pharma, Vifor
Review, Journal: 2021 FDA TIDES (Peptides and Oligonucleotides) Harvest. (Pubmed Central) - Feb 27, 2022
Importantly, this percentage has surpassed the figure in 2020 (10%), thus reflecting the remarkable success of TIDES. In this review, the approved TIDE-based drugs are analyzed on the basis of their chemical structure, medical target, mode of action, administration route, and adverse effects.
- | Bylvay (odevixibat) / Albireo
Journal: Odevixibat (Bylvay) for progressive familial intrahepatic cholestasis-associated pruritus. (Pubmed Central) - Feb 23, 2022
In this review, the approved TIDE-based drugs are analyzed on the basis of their chemical structure, medical target, mode of action, administration route, and adverse effects. No abstract available
- || Bylvay (odevixibat) / Albireo
NICE: $ALBO NICE Recommends Bylvay or All PFIC Types (Twitter) - Feb 22, 2022
- || Bylvay (odevixibat) / Albireo
Clinical, P2 data, Journal: Effects of odevixibat on pruritus and bile acids in children with cholestatic liver disease: Phase 2 study. (Pubmed Central) - Feb 8, 2022
No abstract available Orally administered odevixibat was well tolerated, reduced serum bile acids, and improved pruritus and sleep disturbance in children with cholestatic diseases.
- || Bylvay (odevixibat) / Albireo
Review, Journal: Odevixibat: First Approval. (Pubmed Central) - Feb 5, 2022
Odevixibat is also in clinical development for the treatment of other cholestatic diseases, including Alagille syndrome and biliary atresia, in various countries. This article summarizes the milestones in the development of odevixibat leading to this first approval for PFIC.
- || Bylvay (odevixibat) / Albireo
$ALBO Albireo Increases 2021 Bylvay™ (odevixibat) Sales Guidance https://t.co/4dhXmoO6li (Twitter) - Jan 5, 2022
- | Bylvay (odevixibat) / Albireo
Journal: AHFS®first Release™. (Pubmed Central) - Dec 16, 2021
This article summarizes the milestones in the development of odevixibat leading to this first approval for PFIC. No abstract available
- || Bylvay (odevixibat) / Albireo
$ALBO Albireo Presenting New Bylvay™ (odevixibat) Data at NASPGHAN 2021 https://t.co/JtRe8xgbgk (Twitter) - Dec 13, 2021
- || Bylvay (odevixibat) / Albireo
#Odevixibat reduces markers of progressive familial intrahepatic cholestasis in children #LiverMtg21 @AASLDtweets https://t.co/V6i5yRIvyo (Twitter) - Nov 15, 2021
- | Bylvay (odevixibat) / Albireo
$ALBO Q3 Bylvay net revenue $1.1M (Twitter) - Nov 4, 2021
- Bylvay (odevixibat) / Albireo
[VIRTUAL] Bylvay™: the First and Only Drug Therapy for the Treatment of Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC) () - Nov 2, 2021 - Abstract #AASLD2021AASLD_2466;
Supported by: Albireo Pharmaceuticals Dr. Ryan Fischer will be discussing a treatment option available for patients living with Progressive Familial Intrahepatic Cholestasis (PFIC).
- Bylvay (odevixibat) / Albireo
[VIRTUAL] NATURAL VARIATION OF SERUM BILE ACID LEVELS AND PRURITUS IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: PRETREATMENT DATA FROM PATIENTS IN THE PHASE 3 PEDFIC 1 STUDY () - Oct 21, 2021 - Abstract #AASLD2021AASLD_2191;
Prior to the start of PEDFIC 1, patients with PFIC1 and PFIC2 had considerable variations in clinical parameters including sBAs and pruritus. These data highlight that patients with PFIC experience natural fluctuations in clinical signs and symptoms as a result of their underlying disease .
- Bylvay (odevixibat) / Albireo
[VIRTUAL] HEPATIC PARAMETERS, GROWTH, AND SLEEP WITH RESPONDERS AND NONRESPONDERS TO ODEVIXIBAT TREATMENT: POOLED DATA FROM THE PEDFIC 1 AND PEDFIC 2 STUDIES IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS () - Oct 21, 2021 - Abstract #AASLD2021AASLD_2179;
Odevixibat treatment for up to 72 weeks in patients with PFIC was associated with improvement in hepatic health, quality of sleep, and growth, with greater improvement observed in Rs compared with NRs . Odevixibat was generally well tolerated in Rs and NRs .
- Bylvay (odevixibat) / Albireo
[VIRTUAL] EFFICACY AND SAFETY OUTCOMES WITH ODEVIXIBAT IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS DUE TO DEFICIENCIES IN MULTIDRUG RESISTANCE PROTEIN 3 (PFIC TYPE 3) OR MYOSIN 5B (PFIC TYPE 6) () - Oct 21, 2021 - Abstract #AASLD2021AASLD_2173;
Patients with PFIC3 and PFIC6 experienced clinical benefits with odevixibat, including reductions in sBAs and improvement in pruritus symptoms. Odevixibat treatment was generally well tolerated .
- Bylvay (odevixibat) / Albireo
[VIRTUAL] EFFECTS ON SERUM BILE ACIDS, PRURITUS, AND SAFETY WITH UP TO 72 WEEKS OF ODEVIXIBAT TREATMENT: POOLED DATA FROM THE PEDFIC 1 AND PEDFIC 2 STUDIES IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS () - Oct 21, 2021 - Abstract #AASLD2021AASLD_2172;
sBA Rs had larger improvements in pruritus than sBA NRs; improvements in the latter group may be explained by either continued effects in partial Rs or perceived treatment effects . Odevixibat treatment was generally well tolerated in both Rs and NRs .
- Bylvay (odevixibat) / Albireo
[VIRTUAL] RELATIONSHIPS BETWEEN CHANGES IN AUTOTAXIN, PRURITUS, AND SERUM BILE ACIDS AFTER ODEVIXIBAT TREATMENT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM A POOLED ANALYSIS () - Oct 7, 2021 - Abstract #AASLD2021AASLD_154;
Odevixibat was also generally well tolerated . Odevixibat may be a potential noninvasive treatment option to alleviate critical signs and symptoms of disease in patients with PFIC .
- |||| Bylvay (odevixibat) / Ipsen
Enrollment open: ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Sep 30, 2021
P3, N=63, Enrolling by invitation, Sponsor: Albireo
Odevixibat may be a potential noninvasive treatment option to alleviate critical signs and symptoms of disease in patients with PFIC . Not yet recruiting --> Enrolling by invitation
- || Bylvay (odevixibat) / Albireo
$ALBO Albireo Announces Bylvay® (odevixibat) Now Available in Germany https://t.co/qKqwLZ1c6s (Twitter) - Sep 15, 2021
- || Bylvay (odevixibat) / Albireo
$ALBO Albireo Receives UK MHRA Approval of Bylvay® (odevixibat) https://t.co/FX5y2Nowpv (Twitter) - Sep 8, 2021
- |||||||| Bylvay (odevixibat) / Ipsen
New P3 trial: ASSERT-EXT: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (clinicaltrials.gov) - Sep 5, 2021
P3, N=63, Not yet recruiting, Sponsor: Albireo
- || maralixibat (SHP625) / Mirum Pharma, odevixibat (A4250) / Albireo
Review, Journal: Ileal bile acid transporter inhibition as an anticholestatic therapeutic target in biliary atresia and other cholestatic disorders. (Pubmed Central) - Aug 18, 2021
By reducing return of bile acids to the cholestatic liver, IBAT inhibitors may potentially lessen or delay liver damage associated with the hepatotoxicity and cholangiopathy of bile acid accumulation. The clinical programs of 2 IBAT inhibitors in development for the treatment of pediatric cholestatic liver diseases, maralixibat and odevixibat, are highlighted.