Urea Cycle Disorders
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Sign up freeTop drugs studied for this disease
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FDA approval · 2026-04-15FDA Approved: GLYCEROL PHENYLBUTYRATE — Original Application (Teva Pharmaceuticals, Inc.)
FDA approved glycerol phenylbutyrate for urea cycle disorders through an original application. Teva Pharmaceuticals, Inc. is the sponsor.
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FDA approval · 2026-04-15FDA Approved: GLYCEROL PHENYLBUTYRATE — Original Application (Aurobindo Pharma Limited)
FDA approved the original ANDA for glycerol phenylbutyrate, a generic version developed by Aurobindo Pharma Limited.
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FDA approval · 2026-04-15FDA Approved: GLYCEROL PHENYLBUTYRATE — Original Application (Novadoz Pharmaceuticals LLC)
FDA approves an original application for glycerol phenylbutyrate from Novadoz Pharmaceuticals LLC, a generic option for the treatment of ure…
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FDA approval · 2026-02-17FDA Approved: SODIUM PHENYLBUTYRATE — Original Application (Zydus Pharmaceuticals USA Inc.)
FDA approves an original ANDA for sodium phenylbutyrate by Zydus Pharmaceuticals USA Inc., enabling a generic version of the drug.
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
A clinical trial update for glycerol phenylbutyrate in Chinese patients with urea cycle disorders, currently recruiting.
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CLINICAL_TRIALS research · 2026-01-08Clinical Trial Update: Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients [NA]
A clinical trial update evaluating ureagenesis in healthy subjects and in patients with urea cycle disorders; the study is currently recruit…
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EMA approval · 2025-12-05EMA Authorised: Ravicti (glycerol phenylbutyrate) — Immedica Pharma AB
The European Medicines Agency granted marketing authorization for Ravicti (glycerol phenylbutyrate) to treat inborn urea cycle disorders, wi…
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CLINICAL_TRIALS research · 2024-06-25Clinical Trial Update: Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
This ClinicalTrials.gov study assesses neuroimaging and neuropsychological outcomes in people with urea cycle disorders and is currently rec…
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CLINICAL_TRIALS trial_result · 2024-06-20Clinical Trial Update: A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group [Phase 1]
A Phase 1 study evaluated the safety and pharmacokinetics of glyceryl tri-(4-phenylbutyrate) given as a single dose and twice-daily dosing f…
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FDA approval · 2023-09-29FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Supplemental Application (Zydus Pharmaceuticals USA Inc.)
FDA approved a supplemental application for sodium phenylacetate and sodium benzoate from Zydus Pharmaceuticals USA Inc., updating its regul…
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FDA approval · 2023-07-14FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Original Application (Zydus Pharmaceuticals USA Inc.)
FDA approved an original application for sodium phenylacetate and sodium benzoate to treat hyperammonemia due to urea cycle disorders.
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FDA approval · 2022-11-01FDA Approved: SODIUM PHENYLBUTYRATE — Original Application (GLENMARK PHARMACEUTICALS INC., USA)
FDA approved Glenmark's generic sodium phenylbutyrate (Original Application) for use in treating patients with urea cycle disorders, expandi…
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FDA approval · 2022-07-29FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
FDA approved a supplemental application for BUPHENYL (sodium phenylbutyrate) from Horizon Therapeutics USA, Inc., resulting in updated label…
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CLINICAL_TRIALS trial_result · 2021-12-15Clinical Trial Update: Manipulating the Gut Microbiome Study [Phase 1, Phase 2]
A Phase 1/2 clinical trial investigating manipulation of the gut microbiome for urea cycle disorder; the study was terminated with results p…
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FDA approval · 2021-12-02FDA Approved: GLYCEROL PHENYLBUTYRATE — Original Application (Par Health USA, LLC)
FDA approved the original application for glycerol phenylbutyrate oral to treat patients with urea cycle disorders and help prevent elevated…
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FDA approval · 2021-09-03FDA Approved: GLYCEROL PHENYLBUTYRATE — Supplemental Application (Lupin Pharmaceuticals, Inc.)
The FDA approved a supplemental application for glycerol phenylbutyrate submitted by Lupin Pharmaceuticals, Inc.
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CLINICAL_TRIALS trial_result · 2021-01-26Clinical Trial Update: PCORI Urea Cycle Disorder Study
Clinical trial update indicating completion of a PCORI-funded study on urea cycle disorders, with results posted in 2021.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002) [Phase 2]
Phase 2 clinical trial evaluating the efficacy of HepaStem in paediatric patients with urea cycle disorders has completed.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) [Phase 1, Phase 2]
Phase 1/2 safety study of HepaStem for urea cycle disorders and Crigler-Najjar syndrome has completed.
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FDA approval · 2019-10-04FDA Approved: GLYCEROL PHENYLBUTYRATE — Supplemental Application (Lupin Pharmaceuticals, Inc.)
The FDA approved Lupin Pharmaceuticals' supplemental application for glycerol phenylbutyrate.
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FDA approval · 2018-12-21FDA Approved: GLYCEROL PHENYLBUTYRATE — Supplemental Application (Lupin Pharmaceuticals, Inc.)
FDA approved Lupin Pharmaceuticals' supplemental submission for glycerol phenylbutyrate, a nitrogen-scavenging therapy for urea cycle disord…
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CLINICAL_TRIALS trial_result · 2018-05-04Clinical Trial Update: To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) [Phase 4]
Phase 4 clinical trial evaluating the long-term safety of HPN-100 for managing urea cycle disorders has completed, with results posted in 20…
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CLINICAL_TRIALS trial_result · 2018-03-07Clinical Trial Update: Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
A completed pilot study assessing practice patterns and patient outcomes in urea cycle disorders. The study aims to inform broader managemen…
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CLINICAL_TRIALS trial_result · 2017-12-08Clinical Trial Update: Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders [NA]
A completed clinical trial comparing phenylbutyrate to benzoate for urea cycle disorders with results posted in 2017.
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FDA approval · 2017-05-08FDA Approved: SODIUM PHENYLACETATE AND SODIUM BENZOATE — Original Application (MAIA PHARMACEUTICALS, INC)
FDA approved an original application for the generic combination product sodium phenylacetate and sodium benzoate for the treatment of hyper…