Progressive Familial Intrahepatic Cholestasis
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CLINICAL_TRIALS trial_result · 2026-06-23Clinical Trial Results: An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) [Phase 3]
Phase 3 extension study results for maralixibat in Progressive Familial Intrahepatic Cholestasis (PFIC) have been completed with results pos…
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CLINICAL_TRIALS trial_result · 2026-06-02Clinical Trial Results: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). [Phase 2]
Phase 2 study evaluating safety and tolerability of maralixibat in infants with cholestatic liver diseases including PFIC and Alagille syndr…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: 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
This is an ongoing clinical trial update evaluating the effectiveness, safety, and long-term outcomes of odevixibat (Bylvay) in participants…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain
A recruiting clinical trial in Spain to study genetic variations in PFIC-related genes and their link to symptoms in adults with recurrent c…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASE [NA]
This clinical trial study aims to define the genetic drivers of adult-onset cholestatic liver disease and is currently in the recruiting sta…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: Retrospective Observational Study of Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
Ipsen is conducting a retrospective observational study of odevixibat in Progressive Familial Intrahepatic Cholestasis (PFIC) comparing outc…
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: Pediatric Evaluation and Registry for Liver Cholestasis in Canada
This clinical trial registry update describes a Pediatric Evaluation and Registry for Liver Cholestasis in Canada. It focuses on Progressive…
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CLINICAL_TRIALS trial_result · 2026-02-20Clinical Trial Update: A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
This clinical trial status update describes a database study evaluating maralixibat (TAK-625) in participants with Alagille syndrome and pro…
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FDA approval · 2025-12-19FDA Approved: BYLVAY — Supplemental Application (Ipsen Biopharmaceuticals, Inc.)
FDA approved a supplemental application for BYLVAY (odevixibat) from Ipsen Biopharmaceuticals, Inc.
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EMA approval · 2025-07-11EMA Authorised: Bylvay (odevixibat) — Ipsen Pharma
EMA authorised Bylvay (odevixibat) from Ipsen Pharma for intrahepatic cholestasis as an orphan medicine with accelerated assessment.
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CLINICAL_TRIALS trial_result · 2025-07-10Clinical Trial Update: Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC [Phase 3]
A4250 (odevixibat) was studied in a long-term Phase 3 trial in children with Progressive Familial Intrahepatic Cholestasis. The results asse…
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CLINICAL_TRIALS trial_result · 2025-06-29Clinical Trial Update: An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) [Phase 3]
Clinical trial update announcing completion of an extension study of maralixibat in patients with Progressive Familial Intrahepatic Cholesta…
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CLINICAL_TRIALS research · 2025-05-25Clinical Trial Update: Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
ClinicalTrials.gov update indicates that the long-term safety and clinical outcomes study of Livmarli (maralixibat) for Alagille syndrome an…
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FDA approval · 2025-03-20FDA Approved: BYLVAY — Supplemental Application (Ipsen Biopharmaceuticals, Inc.)
FDA approved a supplemental application for BYLVAY (odevixibat) from Ipsen Biopharmaceuticals, Inc.
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CLINICAL_TRIALS trial_result · 2025-02-06Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). [Phase 2]
Phase 2 trial evaluating the safety and tolerability of maralixibat in infant participants with progressive familial intrahepatic cholestasi…
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CLINICAL_TRIALS research · 2025-01-24Clinical Trial Update: Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)
The TreatFIC study is a prospective analysis of treatment approaches for Progressive Familial Intrahepatic Cholestasis and is currently recr…
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CLINICAL_TRIALS research · 2025-01-17Clinical Trial Update: NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion
This clinical trial update discusses the natural course and prognosis of Progressive Familial Intrahepatic Cholestasis and examines the impa…
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CLINICAL_TRIALS research · 2025-01-17Clinical Trial Update: Genotype-phenotype Relationship Between Cryptogenic Cholestasis and Familial Intrahepatic Cholestasis
This ClinicalTrials.gov entry reports an ongoing study examining the genotype-phenotype relationship between cryptogenic cholestasis and fam…
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CLINICAL_TRIALS research · 2025-01-16Clinical Trial Update: Familial Intrahepatic Cholestasis-related Genes Associated with Disease Susceptibility in Hepato-biliary Cancers
A clinical trial is exploring genetic factors related to Progressive familial intrahepatic cholestasis and their association with susceptibi…
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CLINICAL_TRIALS trial_result · 2024-09-24Clinical Trial Update: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) [Phase 3]
A Phase 3 study evaluating TAK-625 for Progressive Familial Intrahepatic Cholestasis has completed and posted results.
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FDA approval · 2024-07-24FDA Approved: LIVMARLI — Supplemental Application (Mirum Pharmaceuticals Inc.)
The FDA approved a supplemental application for LIVMARLI (maralixibat chloride) submitted by Mirum Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2023-12-11Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) [Phase 3]
Phase 3 trial evaluating maralixibat for progressive familial intrahepatic cholestasis has completed with results posted.
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FDA approval · 2023-06-13FDA Approved: BYLVAY — Supplemental Application (Ipsen Biopharmaceuticals, Inc.)
The FDA approved a supplemental application for BYLVAY (odevixibat) submitted by Ipsen Biopharmaceuticals, Inc. The action updates the label…
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FDA approval · 2023-03-13FDA Approved: LIVMARLI — Supplemental Application (Mirum Pharmaceuticals Inc.)
The FDA approved a supplemental application for LIVMARLI (maralixibat chloride) submitted by Mirum Pharmaceuticals Inc. The approval updates…
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FDA label_change · 2022-10-21FDA Approved: BYLVAY — Supplemental Application (Ipsen Biopharmaceuticals, Inc.)
The document notes a supplemental application submission for BYLVAY (odevixibat) by Ipsen Biopharmaceuticals, Inc., related to labeling chan…