Glycerol phenylbutyrate is a nitrogen-binding agent used to manage patients with urea cycle disorders who cannot be sufficiently managed amino acid supplementation and/or dietary restrictions. Chemically, it is a triglyceride in which three molecules of phenylbutyrate are linked to a glycerol backbone.
- Mechanism
- Curator reviewed
The toxic accumulation of ammonia in the blood and brain arise from urea cycle disorders in which patients are deficient in critical enzymes or transporters that are involved in the synthesis of urea from ammonia. Glycerol phenylbutyrate is a prodrug - the major metabolite, phenylacetate (PAA) is the molecule that binds to nitrogen. PAA conjugates with glutamine (which contains 2 molecules of nitrogen) via acetylation in the liver and kidneys to form phenylacetylglutamine (PAGN), which is excreted by the kidneys. PAGN, like urea, contains 2 moles of nitrogen and provides an alternate vehicle for waste nitrogen excretion.
- Primary indication
- Glycerol phenylbutyrate is a nitrogen-binding agent for the chronic management of adult and pediatric patients ≥2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid...Curator reviewed · 1 structured indication
- Formula / weight
- C33H38O6 · 530.6512 g/mol (avg)
- First approval
- Canada, 2016 · United States, 2013 · European Union, 2020
- Also known as
- GT4P
- Code names
- HPN-100
- Brand names
Resolves to
ZSDBFLMJVAGKOU-UHFFFAOYSA-NSMILESO=C(CCCC1=CC=CC=C1)OCC(COC(=O)CCCC1=CC=CC=C1)OC(=O)CCCC1=CC=CC=C1What you can answer from here — as of July 03, 2026
Which drugs share a target with Glycerol phenylbutyrate, and which of those have an active Phase 3 trial?
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