Active Ingredient History
Sodium phenylbutyrate is a salt of an aromatic fatty acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. It is used as adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in carbamylphosphate (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase. It is indicated in all neonatal- onset efficiency presenting within the first 28 days of life. Also indicated in patients with late-onset, presenting after the first month of life with a history of hyperammonemic encephalopathy. Sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine. The kidneys then excrete Phenylacetylglutamine. PBA (phenylbutyric acid) is absorbed from the intestine and converted by way of β-oxidation to the active moiety, phenylacetic acid (PAA). PAA is conjugated with glutamine in the liver and kidney by way of N-acyl coenzyme A-l-glutamine N-acyltransferase to form phenylacetylglutamine (PAGN). Like urea, PAGN incorporates two waste nitrogens and is excreted in the urine. On a molar basis, it is comparable to urea (each containing two moles of nitrogen). Therefore, phenylacetylglutamine provides an alternate vehicle for waste nitrogen excretion. NCATS
Drug Pricing (per unit)
Note: This drug pricing data is preliminary, incomplete, and may contain errors.
Combination drugs
| Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
|---|
| Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
|---|
Adenocarcinoma (Phase 1/Phase 2)
alpha 1-Antitrypsin Deficiency (Phase 2)
Alzheimer Disease (Phase 2)
Amino Acid Metabolism, Inborn Errors (Phase 2)
Amyotrophic Lateral Sclerosis (Phase 3)
Argininosuccinic Aciduria (Phase 2)
Brain Neoplasms (Phase 2)
Central Nervous System Diseases (Phase 2)
Cholestasis, Intrahepatic (Phase 2/Phase 3)
Colonic Neoplasms (Phase 1/Phase 2)
Colorectal Neoplasms (Phase 1/Phase 2)
Cystic Fibrosis (Phase 1/Phase 2)
Deficiency Diseases (Phase 2)
Diabetes Mellitus (Phase 4)
Diabetes Mellitus, Type 2 (Phase 4)
Genetic Diseases, Inborn (Phase 2/Phase 3)
Head and Neck Neoplasms (Phase 2)
Healthy Volunteers (Phase 1)
Huntington Disease (Phase 2)
Hyperammonemia (Phase 1)
Insulin Resistance (Phase 4)
Intestinal Neoplasms (Phase 1)
Leukemia (Phase 2)
Lung Neoplasms (Phase 2)
Lymphoma (Phase 2)
Lymphoproliferative Disorders (Phase 2)
Machado-Joseph Disease (Phase 2)
Maple Syrup Urine Disease (Phase 2/Phase 3)
Motor Neuron Disease (Phase 2)
Multiple Myeloma (Phase 2)
Muscular Atrophy, Spinal (Phase 1/Phase 2)
Myelodysplastic Syndromes (Phase 2)
Myositis, Inclusion Body (Phase 1)
Neoplasms (Phase 1)
Nervous System Diseases (Phase 2)
Neuroblastoma (Phase 1)
Neurodegenerative Diseases (Phase 3)
Neuromuscular Diseases (Phase 2)
Parkinson Disease (Phase 3)
Premenstrual Dysphoric Disorder (Phase 3)
Prostatic Neoplasms (Phase 2)
Proteinuria (Phase 2/Phase 3)
Pyruvate Dehydrogenase Complex Deficiency Disease (Phase 1/Phase 2)
Spinal Cord Diseases (Phase 2)
Spinal Muscular Atrophies of Childhood (Phase 1/Phase 2)
Stomach Neoplasms (Phase 2)
Supranuclear Palsy, Progressive (Phase 3)
TDP-43 Proteinopathies (Phase 2)
Tuberculosis, Pulmonary (Phase 2)
Urea Cycle Disorders, Inborn (Phase 4)
Wolfram Syndrome (Phase 2)
| Trial | Phase | Start Date | Organizations | Indications |
|---|
Feedback
Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).
Report issue
