BUPHENYL

LOE Approaching

sodium phenylbutyrate

Amgen
NDAORALTABLETPriority Review
Approved
May 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
DailyMed
Full prescribing information
FDA Drugs@FDA
FDA approval history
RxNorm
Drug nomenclature & identifiers