Glycerol phenylbutyrate is an FDA-approved prescription medication used to reduce ammonia levels in the blood of adults and children with urea cycle disorders (UCDs). UCDs are rare inherited metabolic disorders in which the body is unable to break down certain proteins. As a result, toxic ammonia accumulates in the body and can cause serious health problems, including coma and death. Glycerol phenylbutyrate works by helping the body break down ammonia more efficiently and quickly. It is usually taken by mouth, although it can also be administered as an enema. Common side effects of glycerol phenylbutyrate include nausea, vomiting, abdominal pain, and diarrhea.
Glycerol phenylbutyrate, also sold under the brand name Ravicti, is a medication specifically used to manage a group of rare genetic conditions called urea cycle disorders (UCDs) [1].
Here's a breakdown of key points about Glycerol phenylbutyrate:
- Purpose: It helps control high levels of ammonia in the blood (hyperammonemia) caused by UCDs. Ammonia is a waste product normally removed by the body through the urea cycle. In UCDs, this process malfunctions, leading to ammonia buildup which can be harmful.
- Working Mechanism: Glycerol phenylbutyrate acts as a nitrogen-binding agent. Once broken down in the body, it binds with excess ammonia, converting it into a harmless substance that can be excreted in the urine [2].
- Use: It's prescribed for long-term management of UCDs in adults and children two years of age and older, when diet restrictions and/or amino acid supplements alone aren't sufficient [2].
Important things to know:
- Glycerol phenylbutyrate is a prescription medication and should only be taken under a doctor's supervision.
- It's typically provided as an oral liquid medication [1].
- This medication is generally well-tolerated compared to some other treatments for UCDs [5].
Here are some resources for further information:
- Ravicti (brand name): Manufacturer website: https://www.ravicti.com/
- Glycerol phenylbutyrate on Wikipedia: https://en.wikipedia.org/wiki/Glycerol_phenylbutyrate
- Glycerol phenylbutyrate for the chronic management of urea cycle disorders: Pubmed article: https://pubmed.ncbi.nlm.nih.gov/30736206/
Disclaimer: I cannot provide medical advice. If you have any questions or concerns about Glycerol phenylbutyrate or UCDs, please consult with a healthcare professional.