Glycerol phenylbutyrate

Glycerol phenylbutyrate (USAN), trade name Ravicti, is a medication used in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body.[1] It is an FDA-approved prescription drug in the US.[2] It is approved for anyone over 2 months of age. It was developed by Hyperion Therapeutics based on the existing drug Buphenyl, and received approval on February 1, 2013.[3] Hyperion has been criticized for setting a high price for the drug. The price was set at US$250,000–290,000. In 2014, the drug generated $30.8 million in net sales, far behind the older and less expensive Buphenyl ($113.6 million in sales).[4] In March 2015, Horizon Pharma acquired Hyperion Therapeutics and thus Raviciti.

Glycerol phenylbutyrate
Clinical data
Trade namesRavicti
AHFS/Drugs.comravicti
License data
Pregnancy
category
  • US: C (Risk not ruled out)
    Routes of
    administration    		Oral
    ATC code
    Legal status
    Legal status
    Identifiers
    CAS Number
    PubChem CID
    DrugBank
    ChemSpider
    ChEMBL
    CompTox Dashboard (EPA)
    ECHA InfoCard100.228.552
    Chemical and physical data
    FormulaC33H38O6
    Molar mass530.661 g·mol−1
    3D model (JSmol)

    References
    1. "FDA approves new drug for the chronic management of some urea cycle disorders". U.S. Food and Drug Administration. 1 February 2013. Archived from the original on 2013-03-07. Retrieved 2013-04-01.
    2. "FDA Approved Drug Products: Ravicti". Drugs@FDA. U.S. Food and Drug Administration. Retrieved 18 February 2018.
    3. Herder M (April 2016). "Orphan drug incentives in the pharmacogenomic context: policy responses in the USA and Canada". Journal of Law and the Biosciences. 3 (1): 158–166. doi:10.1093/jlb/lsv060. PMC 5033429. PMID 27774236.
    4. "Horizon Pharma to Acquire Hyperion Therapeutics for $1.1B". Genetic Engineering & Biotechnology News. 30 March 2015.
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