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.comMicromedex Detailed Consumer Information
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
IUPAC name
  • 1,2,3-Propanetriyl tris(4-phenylbutanoate)
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.228.552
Chemical and physical data
FormulaC33H38O6
Molar mass530.661 g·mol−1
3D model (JSmol)
SMILES
  • C1=CC=C(C=C1)CCCC(=O)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3
InChI
  • InChI=1S/C33H38O6/c34-31(22-10-19-27-13-4-1-5-14-27)37-25-30(39-33(36)24-12-21-29-17-8-3-9-18-29)26-38-32(35)23-11-20-28-15-6-2-7-16-28/h1-9,13-18,30H,10-12,19-26H2
  • Key:ZSDBFLMJVAGKOU-UHFFFAOYSA-N

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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