NDAORALTABLETPriority Review
Approved
May 1996
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
6
Data completeness
5/8 — Partial
Priority Review
BUPHENYL (sodium phenylbutyrate) by Amgen is clinical pharmacology sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. First approved in 1996.
Drug data last refreshed 17h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
BUPHENYL (sodium phenylbutyrate) is an oral small-molecule pro-drug approved in 1996 that metabolizes to phenylacetate, which conjugates with glutamine to form phenylacetylglutamine for renal excretion. It provides an alternate nitrogen waste pathway comparable to urea on a molar basis. This mechanism makes it indicated for urea cycle disorders and hyperammonemic conditions where nitrogen accumulation is life-threatening.
$0.003BPart D
LOE ApproachingLow commercial scale (120 Part D claims, $3M spending) with lifecycle approaching LOE signals team focus is maintaining specialty niche rather than broad expansion.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (6)
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
A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate
Started May 2022
8 enrolled
Hyperammonemia
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
Started Feb 2018
16 enrolled
Urea Cycle Disorder
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
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Started Feb 2008
12 enrolled
Argininosuccinic AciduriaAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.