Glycerol phenylbutyrate

Glycerol phenylbutyrate
Clinical data
Trade names Ravicti
AHFS/Drugs.com ravicti
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
    ECHA InfoCard 100.228.552 Edit this at Wikidata
    Chemical and physical data
    Formula C33H38O6
    Molar mass 530.651 g/mol
    3D model (JSmol)

    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 Theraputics 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.

    References

    1. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337639.htm
    2. Drugs@FDA: FDA Approved Drug Products. Accessdata.fda.gov. [accessed 2018 Feb 18]. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&applno=203284
    3. Herder M. Orphan drug incentives in the pharmacogenomic context: policy responses in the USA and Canada. Journal of Law and the Biosciences. 2016 [accessed 2018 Feb 18];3(1):158-166. DOI: 10.1093/jlb/lsv060 URL: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5033429/
    4. "Horizon Pharma to Acquire Hyperion Therapeutics for $1.1B". GEN.


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