Sodium phenylbutyrate is a Small Molecule owned by Horizon Therapeutics, and is involved in 2 clinical trials, which were completed.

Sodium phenylbutyrate depletes glutamine which are involved in turning genes ‘on’ and ‘off’ within cells. They are responsible for the histone being acetylated or deacetylated and related to transcription, cell cycle progression, gene silencing, lymphocyte and muscle differentiation, regulation of neuronal phenotype, DNA replication, and the response to DNA damage. 

The revenue for Sodium phenylbutyrate is expected to reach a total of $76m through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Sodium phenylbutyrate NPV Report.

Sodium phenylbutyrate is currently owned by Horizon Therapeutics.

Sodium phenylbutyrate Overview

Sodium phenylbutyrate (Buphenyl, Ammonaps) is a sodium salt of fatty acid, acts as endocrine and metabolic agent. It is formulated as tablet, powder and granules intended for oral administration. Sodium phenylbutyrate is indicated for urea cycle disorders involving deficiencies of ornithine transcarbamylase (OTC), carbamylphosphate synthetase (CPS), or argininosuccinic acid synthetase (AS). It is indicated in all patients with neonatal-onset deficiency and in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy. Sodium phenylbutyrate was under development for the treatment of spinal muscular atrophy.

Horizon Therapeutics Overview

Horizon Therapeutics formerly Horizon Pharma, is a pharmaceutical company, which develops and commercializes medical products for the treatment of rare, autoimmune, arthritis and severe inflammatory diseases. It markets medicines through its orphan, primary care and rheumatology business units. A few of the company’s marketed products include Actimmune, Buphenyl, Duexis, Krystexxa, Pennsaid, Procysbi, Quinsair, Ravicti, Rayos, Tepezza, Uplinza, and Vimovo. Horizon Therapeutics provides products and services to patients and healthcare professionals in the US. The company operates through offices in the US, Switzerland, Luxembourg, Bermuda, Germany, Canada, and Israel. Horizon Therapeutics is headquartered in Dublin, Ireland.

The company reported revenues of (US Dollars) US$3,226.4 million for the fiscal year ended December 2021 (FY2021), an increase of 46.6% over FY2020. In FY2021, the company’s operating margin was 16.8%, compared to an operating margin of 20.8% in FY2020. In FY2021, the company recorded a net margin of 16.6%, compared to a net margin of 17.7% in FY2020. The company reported revenues of US$925.4 million for the third quarter ended September 2022, an increase of 5.6% over the previous quarter.

Quick View – Sodium phenylbutyrate

Report Segments
  • Innovator (NME)
Drug Name
  • Sodium phenylbutyrate
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.