Sodium phenylbutyrate

Sodium phenylbutyrate

Systematic (IUPAC) name
Sodium 4-phenylbutanoate
Clinical data
AHFS/Drugs.com	Consumer Drug Information
Licence data	EMA:Link, US FDA:link
Pregnancy cat.	not to be used
Legal status	?
Pharmacokinetic data
Metabolism	to phenylacetic acid
Half-life	0.8 hours
Excretion	80% as phenylacetylglutamine
Identifiers
CAS number	1716-12-7 N
ATC code	A16AX03
PubChem	CID 9815454
ChemSpider	5068 N
ChEMBL	CHEMBL1746 N
Chemical data
Formula	C10H11NaO2
Mol. mass	186.2 g/mol
SMILES	eMolecules & PubChem
InChI InChI=1S/C10H12O2.Na/c1-2-9(10(11)12)8-6-4-3-5-7-8;/h3-7,9H,2H2,1H3,(H,11,12);/q;+1/p-1 Y Key:RHLFTMGPBSLHRS-UHFFFAOYSA-M Y
N (what is this?)  (verify)

Sodium phenylbutyrate is an orphan drug, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl and by Swedish Orphan International (Sweden) as Ammonaps.

Phenylbutyrate has been used to treat urea cycle disorders.

University of Colorado researchers Dr. Curt Freed and Wenbo Zhou demonstrated that phenylbutyrate stops the progression of Parkinson's disease in mice by turning on a gene called DJ-1 that can protect dopaminergic neurons in the midbrain from dying. As of July 2011 they plan on testing phenylbutyrate for the treatment of Parkinson's disease in humans.

Metabolism

Phenylbutyrate is a prodrug. In the human body it is metabolized by beta-oxidation to phenylacetate.

Phenylacetate conjugates with glutamine to phenylacetylglutamine, that is eliminated with the urine.

Nitrogen elimination

Research

Sodium phenylbutyrate is also under investigation for the treatment of some sickle-cell disorders (Blood Products Plasma Expanders and Haemostatics) and for use as a potential differentiation-inducing agent in malignant glioma and acute myeloid leukaemia.^{[citation needed]}

Phenylbutyrate has been associated with longer lifespans in Drosophila.