sodium phenylbutyrate (buphenyl) Report issue

Small molecule Orphan Drug FDA Approved FDA Priority Review FDA

Active Ingredient History

NOW
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Sodium phenylbutyrate is a salt of an aromatic fatty acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. It is used as adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in carbamylphosphate (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase. It is indicated in all neonatal- onset efficiency presenting within the first 28 days of life. Also indicated in patients with late-onset, presenting after the first month of life with a history of hyperammonemic encephalopathy. 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. The kidneys then excrete Phenylacetylglutamine. PBA (phenylbutyric acid) is absorbed from the intestine and converted by way of β-oxidation to the active moiety, phenylacetic acid (PAA). PAA is conjugated with glutamine in the liver and kidney by way of N-acyl coenzyme A-l-glutamine N-acyltransferase to form phenylacetylglutamine (PAGN). Like urea, PAGN incorporates two waste nitrogens and is excreted in the urine. On a molar basis, it is comparable to urea (each containing two moles of nitrogen). Therefore, phenylacetylglutamine provides an alternate vehicle for waste nitrogen excretion.   NCATS

More Chemistry
  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Oral
  • Pro Drug: Yes

Drug Pricing (per unit)

United States

$20.4053 - $54.2361

Australia

$401.6442
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

4-pba | 4-phenylbutanoic acid | 4-phenylbutylamine | 4-phenylbutyrate | 4-phenylbutyric acid | 4-phenylbutyric acid, calcium salt | 4-phenylbutyric acid, sodium salt | 4-phenyl-n-butyric acid | ambutyrate | ammonaps | benzenebutyric acid | buphenyl | gamma-phenylbutanoic acid | pba | pheburane | phenylbutanoic acid | phenylbutyrate | phenylbutyric acid | sodium 4-phenylbutanoate | sodium 4-phenylbutyrate | sodium phenylbutyrate | γ-phenylbutyric acid | γ-phenyl-n-butyric acid | ω-phenylbutanoic acid | ω-phenylbutyric acid

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