Product Description
Sodium phenylbutyrate is used together with a proper diet to help treat urea cycle disorders (including a specific liver enzyme deficiency) that help remove ammonia (nitrogen) from the body. (Sourced from: https://www.mayoclinic.org/drugs-supplements/sodium-phenylbutyrate-oral-route/description/drg-20066057)
Mechanisms of Action: HDAC Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Canada | Colombia | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Netherlands | New Zealand | Norway | Poland | Portugal | Romania | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: Amgen
Company Location: THOUSAND OAKS CA 91320
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Japan, United States
Active Clinical Trial Count: 3
Highest Development Phases
Phase 2: Lipid Metabolism, Inborn Errors|MELAS Syndrome
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| jRCT2071220110 | P3 |
Recruiting |
Unknown |
2026-03-31 |
|
| MCADD | P2 |
Recruiting |
Lipid Metabolism, Inborn Errors |
2025-06-01 |
|
| PHEMI | P2 |
Active, not recruiting |
MELAS Syndrome |
2020-07-16 |