Alagille Syndrome
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Sign up freeTop drugs studied for this disease
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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 · 2026-04-22Clinical Trial Update: Management of Steatorrhea in Children and Young Adults With Alagille Syndrome After Liver Transplantation [NA]
Clinical trial update focusing on managing steatorrhea due to exocrine pancreatic insufficiency in children and young adults with Alagille s…
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EMA approval · 2026-04-17EMA Authorised: Livmarli (Maralixibat chloride) — Mirum Pharmaceuticals International B.V.
The European Medicines Agency has authorised Livmarli (maralixibat) for Alagille syndrome, with orphan medicine designation.
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CLINICAL_TRIALS trial_result · 2026-04-01Clinical Trial Update: Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome [Phase 3]
A Phase 3 trial evaluating the long-term safety and efficacy of odevixibat in Alagille syndrome; the study is currently recruiting. Sponsor:…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This clinical trial update notes the long-term effectiveness and safety assessment of odevixibat (Bylvay) in patients with Alagille syndrome…
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CLINICAL_TRIALS trial_result · 2026-03-27Clinical Trial Update: Atorvastatin Therapy on Xanthoma in Alagille Syndrome [Phase 4]
Phase 4 clinical trial evaluating atorvastatin for xanthomas in Alagille syndrome; the study has completed.
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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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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU) [Phase 4]
A Phase 4 long-term safety and clinical outcomes study of LivmArli in patients with Alagille Syndrome in the European Union is currently rec…
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Update indicating the LOGIC genetic study of intrahepatic cholestasis is currently suspended.
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EMA approval · 2025-07-11EMA Authorised: Kayfanda (odevixibat sesquihydrate) — Ipsen Pharma
The EMA authorised Kayfanda (odevixibat sesquihydrate) for pruritus and Alagille syndrome, manufactured by Ipsen Pharma.
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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 · 2025-04-10FDA Approved: LIVMARLI — Original Application (Mirum Pharmaceuticals Inc.)
The FDA approved LIVMARLI (maralixibat chloride) from Mirum Pharmaceuticals as an original application for the treatment of pruritus in pedi…
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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 trial_result · 2024-10-15Clinical Trial Update: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) [Phase 3]
A Phase 3 trial evaluating TAK-625 for Alagille Syndrome has completed, with results posted in October 2024.
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CLINICAL_TRIALS trial_result · 2024-08-28Clinical Trial Update: FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
This update reports on the FORCE trial evaluating FibroScan, a non-invasive liver fibrosis assessment tool, in pediatric cholestatic liver d…
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FDA approval · 2024-03-13FDA Approved: LIVMARLI — Supplemental Application (Mirum Pharmaceuticals Inc.)
The FDA approved a supplemental application for LIVMARLI (maralixibat chloride) from Mirum Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2023-11-02Clinical Trial Update: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome [Phase 3]
Phase 3 trial results update on the efficacy and safety of odevixibat in patients with Alagille syndrome.
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CLINICAL_TRIALS trial_result · 2021-11-19Clinical Trial Update: An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) [Phase 2]
Phase 2 extension study evaluating long-term safety and durability of LUM001 in Alagille Syndrome; trial completed with results posted in 20…
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CLINICAL_TRIALS trial_result · 2021-07-14Clinical Trial Update: Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) [Phase 2]
Phase 2 safety and efficacy study of LUM001 (maralixibat) in participants with Alagille Syndrome has completed and results were posted.
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CLINICAL_TRIALS trial_result · 2021-06-22Clinical Trial Update: An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome [Phase 2]
Phase 2 extension study evaluating long-term safety and durability of effect of LUM001 in pediatric Alagille syndrome; study completed with …
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CLINICAL_TRIALS trial_result · 2018-01-23Clinical Trial Update: Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome [Phase 2]
Phase 2 clinical trial evaluating LUM001 for reduction of pruritus in Alagille syndrome has completed with results posted.
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CLINICAL_TRIALS trial_result · 2015-12-07Clinical Trial Update: Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome [Phase 2]
Phase 2 clinical trial assessing safety and efficacy of LUM001 for cholestatic liver disease in Alagille Syndrome has completed with posted …
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CLINICAL_TRIALS trial_result · 2009-01-22Clinical Trial Update: Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease [NA]
A completed clinical trial evaluating magnesium sulfate in children with reduced bone density due to chronic cholestatic liver disease, incl…
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CLINICAL_TRIALS research · 2008-03-04Clinical Trial Update: Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
This completed clinical trial update reports the progress of positional cloning to identify the gene(s) responsible for Alagille syndrome. T…