NDAORALTABLETPriority Review
Approved
May 1996
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
6
Data completeness
6/8 — Strong
Priority Review
BUPHENYL (sodium phenylbutyrate) by Amgen is clinical pharmacology sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Approved for hyperammonemia. First approved in 1996.
Drug data last refreshed 20h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
BUPHENYL (sodium phenylbutyrate) is an oral tablet that acts as a pro-drug, rapidly metabolizing to phenylacetate, which conjugates with glutamine to form phenylacetylglutamine for renal excretion. It treats hyperammonemia and related urea cycle disorders by providing an alternative pathway for waste nitrogen excretion. The drug is indicated for hyperammonemia, combined D,L-2-hydroxyglutaric aciduria, and medium-chain acyl-CoA dehydrogenase deficiency.
$3MPart D
LOE ApproachingProduct is in late-stage lifecycle with limited volume (120 annual claims), suggesting small specialized teams focused on legacy maintenance rather than growth.
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 (1)
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.