Phenylbutyric acid is an agent indicated for the adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in specific enzymes, including the neonatal-onset deficiency and late-onset disease with a history of hyperammonemic encephalopathy. Phenylbutyric acid is a fatty acid and a derivative of butyric acid naturally produced by colonic bacteria fermentation.
- Mechanism
- Curator reviewed · 3 references
Sodium phenylbutyrate is the most commonly used salt used in drug products of phenylbutyric acid. Sodium phenylbutyrate is a pro-drug that rapidly metabolizes to phenylacetate. Phenylacetate is conjugated with phenylacetyl-CoA, which in turn combines with glutamine via acetylation to form phenylacetylglutamine. Phenylacetylglutamine is then excreted by the kidneys, thus providing an alternate mechanism of waste nitrogen excretion to the urea cycle. Phenylacetylglutamine is comparable to urea, as each molecule contains two moles of nitrogen.
- Primary indication
- Phenylbutyric acid is used for the treatment of various conditions, including urea cycle disorders, neonatal-onset deficiency, late-onset deficiency disease in patients with a history of hyperammonemic encephalopathy.Curator reviewed · 7 structured indications
- Formula / weight
- C10H12O2 · 164.2011 g/mol (avg)
- First approval
- Canada, 2015 · United States, 1996 · European Union, 2016
- Also known as
- 4-phenylbutyrate · 4-phenylbutyric acid · Benzenebutyric acid · Phenylbutyrate · ω-Phenylbutanoic acid
- Code names
- NSC-295
- Brand names
Resolves to
OBKXEAXTFZPCHS-UHFFFAOYSA-NSMILESOC(=O)CCCC1=CC=CC=C1What you can answer from here — as of September 13, 2026
Which drugs share a target with Phenylbutyric acid, and which of those have an active Phase 3 trial?
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