Sodium Phenylbutyrate
Sodium phenylbutyrate is an oral ammonia-scavenging agent used for long-term management of urea cycle disorders, serving as a prodrug that is beta-oxidized in the liver to phenylacetate, which then conjugates with glutamine for renal nitrogen excretion. It also acts as a histone deacetylase inhibitor at therapeutic concentrations, and is investigated for its ability to induce expression of fetal hemoglobin in sickle cell disease, reduce protein misfolding in inherited metabolic disorders, and as an antineoplastic agent in colorectal cancer and other tumors.
Molekularmasse
186,1800 g/mol
TPSA
40,10 Ų
Therapeutische Bereiche
Wirkmechanismus
Administered as an inactive precursor that is metabolically converted to its active form in the body. This prodrug design improves bioavailability, absorption, or targeted delivery compared to the active compound.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Administered as an inactive precursor that is metabolically converted to its active form in the body. This prodrug design improves bioavailability, absorption, or targeted delivery compared to the active compound.
2D-Struktur
Cite this structure
Embed this structure
SMILES
O=C([O-])CCCc1ccccc1.[Na+]
InChI
InChI=1S/C10H12O2.Na/c11-10(12)8-4-7-9-5-2-1-3-6-9;/h1-3,5-6H,4,7-8H2,(H,11,12);/q;+1/p-1
Molecular Formula
C10H11NaO2
HBD / HBA
- / 2
Rotierbare Bindungen
4
Schwere Atome
13
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
Häufig gestellte Fragen
Sodium phenylbutyrate is an oral ammonia-scavenging agent used for long-term management of urea cycle disorders, serving as a prodrug that is beta-oxidized in the liver to phenylacetate, which then conjugates with glutamine for renal nitrogen excretion. It also acts as a histone deacetylase inhibitor at therapeutic concentrations, and is investigated for its ability to induce expression of fetal hemoglobin in sickle cell disease, reduce protein misfolding in inherited metabolic disorders, and as an antineoplastic agent in colorectal cancer and other …
Administered as an inactive precursor that is metabolically converted to its active form in the body. This prodrug design improves bioavailability, absorption, or targeted delivery compared to the active compound.
Yes, Sodium Phenylbutyrate is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL1746. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 5258. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.
Medizinischer Haftungsausschluss
This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.
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