Medicines
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Discover all available medicines and treatments
Brand: Buphenyl, Ammonaps, Ravicti
4-Phenylbutyric acid (4-PBA) is a small molecule drug primarily used as an ammonia scavenger in the treatment of urea cycle disorders (UCDs). It acts as a nitrogen-binding agent, helping to remove excess ammonia from the body. It is also being investigated for its potential in other conditions, including certain cancers and neurodegenerative diseases.
For treatment of urea cycle disorders and investigated for potential use in certain cancers and neurodegenerative diseases.
Outcome:
Increased 4-PBA levels
Mechanism:
Competition for renal excretion
Outcome:
Altered lithium levels
Mechanism:
Competition for renal excretion
Outcome:
Reduced absorption of 4-PBA
Mechanism:
Changes in gastric pH
Most likely new formulation: Sustained-release oral formulation (2025, 70% confidence)
Based on current clinical trial activity, there is a 60% likelihood of 4-PBA gaining approval for an additional indication within the next 5 years.
Ammonia Scavenger, Orphan Drug
Aromatic Fatty Acid