Urea Cycle Disorder
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2025-10-06Clinical Trial Update: At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism [NA]
Clinical trial update on at-home ammonia monitoring for individuals with inborn errors of ammonia metabolism; enrollment is by invitation.
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CLINICAL_TRIALS trial_result · 2025-07-20Clinical Trial Update: Hepatic Histopathology in Urea Cycle Disorders
This clinical trial update indicates the study on hepatic histopathology in urea cycle disorders is actively recruiting, including Ornithine…
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CLINICAL_TRIALS research · 2025-04-17Clinical Trial Update: Liver Disease in Urea Cycle Disorders
A clinical trial update indicating ongoing recruitment for a study on liver disease in patients with urea cycle disorders, including OTC def…
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CLINICAL_TRIALS research · 2024-10-30Clinical Trial Update: MyRareDiet A Novel Diet Tracking Tool [NA]
This clinical trial update outlines MyRareDiet, a novel diet-tracking tool being studied for urea cycle disorders and related organic acidem…
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CLINICAL_TRIALS trial_result · 2024-05-29Clinical Trial Update: HepaStem Long-Term Safety Registry
This clinical trial update announces the termination of the HepaStem long-term safety registry and highlights conditions studied (urea cycle…
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FDA approval · 2024-01-19FDA Approved: CARBAGLU — Supplemental Application (Recordati Rare Diseases)
FDA approved a supplemental application for CARBAGLU (carbaglumic acid) submitted by Recordati Rare Diseases.
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CLINICAL_TRIALS trial_result · 2023-07-10Clinical Trial Update: Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) [Phase 4]
This Phase 4 study evaluated glycerol phenylbutyrate and sodium phenylbutyrate in phenylbutyrate-naïve patients with urea cycle disorders; t…
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FDA approval · 2023-05-04FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Supplemental Application (MAIA PHARMACEUTICALS, INC)
FDA approved a supplemental application for sodium phenylacetate and sodium benzoate from Maia Pharmaceuticals, indicating approval of an ex…
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CLINICAL_TRIALS trial_result · 2023-02-10Clinical Trial Update: Chronic Liver Disease in Urea Cycle Disorders
A completed clinical trial assessing chronic liver disease in individuals with urea cycle disorders; results posted in 2023.
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FDA approval · 2022-07-29FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
FDA approved a supplemental NDA for BUPHENYL (sodium phenylbutyrate) from Horizon Therapeutics USA, Inc., expanding its approved labeling fo…
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FDA approval · 2022-06-17FDA Approved: PHEBURANE — Original Application (Medunik USA)
FDA approved PHEBURANE (sodium phenylbutyrate) under Medunik USA's original NDA for the treatment of urea cycle disorders to prevent hyperam…
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FDA approval · 2021-06-10FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Original Application (MAIA PHARMACEUTICALS)
The FDA approved Maia Pharmaceuticals' original application for sodium phenylacetate and sodium benzoate, a therapy for managing hyperammone…
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CLINICAL_TRIALS trial_result · 2021-02-18Clinical Trial Update: Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
An observational trial sponsored by Amgen to collect information on patients with urea cycle disorders; the study is terminated and results …
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CLINICAL_TRIALS trial_result · 2019-03-05Clinical Trial Update: A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders [Phase 4]
Phase 4 clinical trial update assessing safety, efficacy and pharmacokinetics of glycerol phenylbutyrate in pediatric subjects under 2 years…
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EMA withdrawal · 2016-04-04EMA Refused: Heparesc (human heterologous liver cells) — Cytonet GmbH KG
The EMA refused the marketing authorization for Heparesc (human heterologous liver cells) by Cytonet GmbH KG for urea cycle disorders, Inbor…
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FDA approval · 2016-02-24FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Original Application (Omnivium Pharmaceuticals LLC)
FDA approved Omnivium's generic sodium phenylacetate and sodium benzoate (original application) for treatment of hyperammonemia due to urea …
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CLINICAL_TRIALS trial_result · 2015-05-29Clinical Trial Update: Neurologic Injuries in Adults With Urea Cycle Disorders
A completed clinical trial update reporting results on neurologic injuries in adults with urea cycle disorders, including ornithine transcar…
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CLINICAL_TRIALS trial_result · 2014-11-05Clinical Trial Update: Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder [Phase 2]
Phase 2 clinical trial update on arginine and buphenyl for argininosuccinic aciduria, a urea cycle disorder; trial completed with results po…
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FDA label_change · 2013-08-05FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
FDA approved a supplemental application for BUPHENYL (sodium phenylbutyrate) from Horizon Therapeutics USA, Inc.
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CLINICAL_TRIALS research · 2012-07-31Clinical Trial Update: Investigating Project of the Functional C13 Test "Pilot Study" [Phase 1, Phase 2]
Clinical trial update for a Phase 1/2 pilot study evaluating a Functional C13 Test for urea cycle disorders. The current status is unknown.
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FDA approval · 2009-03-31FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
FDA approved a supplemental application for BUPHENYL (sodium phenylbutyrate) submitted by Horizon Therapeutics USA, Inc.