Phenylbutyric acid
- Generic Name
- Phenylbutyric acid
- Brand Names
- Ammonaps, Buphenyl, Olpruva 2 Gm Pack, Pheburane, Relyvrio
- Drug Type
- Small Molecule
- Chemical Formula
- C10H12O2
- CAS Number
- 1821-12-1
- Unique Ingredient Identifier
- 7WY7YBI87E
Overview
Phenylbutyric acid is a fatty acid and a derivative of butyric acid naturally produced by colonic bacteria fermentation. It demonstrates a number of cellular and biological effects, such as relieving inflammation and acting as a chemical chaperone. It is used to treat genetic metabolic syndromes, neuropathies, and urea cycle disorders.
Background
Phenylbutyric acid is a fatty acid and a derivative of butyric acid naturally produced by colonic bacteria fermentation. It demonstrates a number of cellular and biological effects, such as relieving inflammation and acting as a chemical chaperone. It is used to treat genetic metabolic syndromes, neuropathies, and urea cycle disorders.
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. Phenylbutyric acid must be combined with dietary protein restriction and, in some cases, essential amino acid supplementation. Phenylbutyric acid, as sodium phenylbutyrate, is used in combination with tauroursodeoxycholic acid to treat amyotrophic lateral sclerosis (ALS) in adults.
Associated Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Argininosuccinate Synthetase Deficiency (Citrullinemia)
- Carbamyl Phosphate Synthetase Deficiency
- Ornithine Transcarbamylase Deficiency
- Deficiencies in enzymes of the urea cycle
- Neonatal-onset deficiency
Research Report
Phenylbutyric Acid: A Comprehensive Review of its Pharmacology, Therapeutic Applications, and Clinical Profile
1. Introduction to Phenylbutyric Acid
1.1. Overview and Significance
Phenylbutyric acid, a derivative of the short-chain fatty acid butyric acid, is a pharmacologically active compound with established and emerging therapeutic applications.[1] It is recognized as being naturally produced by the fermentation processes of colonic bacteria.[1] The primary clinical significance of phenylbutyric acid lies in its role as an adjunctive therapy for the chronic management of urea cycle disorders (UCDs), a group of rare genetic metabolic conditions characterized by the inability to effectively eliminate waste nitrogen.[1] Beyond this well-defined indication, phenylbutyric acid exhibits a broader spectrum of biological activities. These include functioning as a histone deacetylase (HDAC) inhibitor and as a chemical chaperone, properties that form the basis for its investigation in a variety of other pathological states.[1] The diverse pharmacological profile of phenylbutyric acid suggests its potential utility extends beyond its primary metabolic role, encompassing inflammatory conditions, certain malignancies, and neurodegenerative disorders.
1.2. Initial DrugBank and User-Provided Information
The compound under review is Phenylbutyric acid, identified in the DrugBank database with the accession number DB06819.[1] Its Chemical Abstracts Service (CAS) registry number is 1821-12-1.[2] Phenylbutyric acid is classified as a Small Molecule drug.[1]
Its principal approved use is as an adjunctive therapeutic agent for the chronic management of UCDs. This includes enzyme deficiencies presenting in the neonatal period (neonatal-onset deficiency) as well as those manifesting later in life (late-onset disease) in individuals with a prior history of hyperammonemic encephalopathy.[1]
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