Review Article

Drugs in R & D

, Volume 11, Issue 3, pp 227-249

First online:

Open Access This content is freely available online to anyone, anywhere at any time.

Clinical and Experimental Applications of Sodium Phenylbutyrate

  • Tommaso IannittiAffiliated withDepartment of Biological and Biomedical Sciences, Glasgow Caledonian University Email author 
  • , Beniamino PalmieriAffiliated withDepartment of General Surgery and Surgical Specialties, University of Modena and Reggio Emilia Medical School, Surgical Clinic


Histone acetyltransferase and histone deacetylase are enzymes responsible for histone acetylation and deacetylation, respectively, in which the histones are acetylated and deacetylated on lysine residues in the N-terminal tail and on the surface of the nucleosome core. These processes are considered the most important epigenetic mechanisms for remodeling the chromatin structure and controlling the gene expression. Histone acetylation is associated with gene activation. Sodium phenylbutyrate is a histone deacetylase inhibitor that has been approved for treatement of urea cycle disorders and is under investigation in cancer, hemoglobinopathies, motor neuron diseases, and cystic fibrosis clinical trials. Due to its characteristics, not only of histone deacetylase inhibitor, but also of ammonia sink and chemical chaperone, the interest towards this molecule is growing worldwide. This review aims to update the current literature, involving the use of sodium phenylbutyrate in experimental studies and clinical trials.