Glycerol phenylbutyrate

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Glycerol phenylbutyrate
Glycerol phenylbutyrate skeletal.svg
Clinical data
Trade names Ravicti
AHFS/Drugs.com Micromedex Detailed Consumer Information
License data
Routes of
administration 		By mouth
ATC code
Legal status
Legal status
Identifiers
  • 1,2,3-Propanetriyl tris(4-phenylbutanoate)
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard 100.228.552 OOjs UI icon edit-ltr-progressive.svg
Chemical and physical data
Formula C33H38O6
Molar mass 530.661 g·mol−1
3D model (JSmol)
  • C1=CC=C(C=C1)CCCC(=O)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3
  • 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

Glycerol phenylbutyrate, sold under the brand name Ravicti, is a nitrogen-binding agent medication used in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body. [4] It is an FDA-approved prescription drug in the US. [5] It was developed by Hyperion Therapeutics based on the existing medication sodium phenylbutyrate, and received approval in February 2013. [6]

Contents

Medical uses

Glycerol phenylbutyrate is indicated for chronic management of people with urea cycle disorders who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. [2]

Society and culture

Economics

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). [7]

References

  1. ↑ "Health Canada New Drug Authorizations: 2016 Highlights". Health Canada . 14 March 2017. Retrieved 7 April 2024.
  2. 1 2 "Ravicti- glycerol phenylbutyrate liquid". DailyMed. 13 September 2021. Archived from the original on 3 May 2023. Retrieved 4 July 2023.
  3. ↑ "Ravicti EPAR". European Medicines Agency (EMA). 10 June 2010. Retrieved 20 February 2025.
  4. ↑ "FDA approves new drug for the chronic management of some urea cycle disorders" (Press release). U.S. Food and Drug Administration (FDA). 1 February 2013. Archived from the original on 7 March 2013. Retrieved 1 April 2013.
  5. ↑ "Drug Approval Package: Ravicti (glycerol phenylbutyrate) Oral Liquid NDA #203284". U.S. Food and Drug Administration (FDA). 28 March 2013. Retrieved 20 February 2025.
  6. ↑ 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.
  7. ↑ "Horizon Pharma to Acquire Hyperion Therapeutics for $1.1B". Genetic Engineering & Biotechnology News. 30 March 2015. Archived from the original on 26 August 2017. Retrieved 18 February 2018.
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