BUPHENYL

LOE Approaching

sodium phenylbutyrate

Amgen
NDAORALPOWDERPriority Review
Approved
Apr 1996
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
4

Mechanism of Action

CLINICAL PHARMACOLOGY Sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine. Phenylacetylglutamine then is excreted by the kidneys. On a molar…

Indications (6)

all patients with neonatal-onset deficiency (complete enzymatic deficiencypresenting within the first 28 days of life)late-onset disease (partial enzymatic deficiencypresenting after the first month of life)mental deteriorationrecurrence of hyperammonemic encephalopathy if carefully adhered to

Clinical Trials (4)

NCT06773026Phase 2Recruiting

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Started Jun 2025
24 enrolled
Medium-chain Acyl-CoA Dehydrogenase Deficiency
NCT05349435Phase 1Terminated

A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate

Started May 2022
NCT03335488Phase 4Completed

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Started Feb 2018
16 enrolled
Urea Cycle Disorder
NCT00947544Phase 2Completed

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

Started Mar 2010
17 enrolled
Urea Cycle Disorders
Company
Amgen
Amgen
THOUSAND OAKS, CA
1215 open jobs
External Resources
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