Glycerol phenylbutyrate

Glycerol phenylbutyrate, sold under the brand 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.^[3] It is an FDA-approved prescription drug in the US.^[4] It was developed by Hyperion Therapeutics based on the existing medication sodium phenylbutyrate, and received approval in February 2013.^[5]

Glycerol phenylbutyrate

Clinical data
Trade names	Ravicti
AHFS/Drugs.com	Micromedex Detailed Consumer Information
Routes of administration	By mouth
ATC code	A16AX09 (WHO)
Legal status
Legal status	CA: ℞-only[1] US: ℞-only[2] EU: Rx-only
Identifiers
IUPAC name 1,2,3-Propanetriyl tris(4-phenylbutanoate)
CAS Number	611168-24-2
PubChem CID	10482134
DrugBank	DB08909
ChemSpider	8657541
UNII	ZH6F1VCV7B
ChEMBL	ChEMBL2105745
CompTox Dashboard (EPA)	DTXSID40210005
ECHA InfoCard	100.228.552
Chemical and physical data
Formula	C33H38O6
Molar mass	530.661 g·mol−1
3D model (JSmol)	Interactive image
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

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).^[6]

References

1. "Health Canada New Drug Authorizations: 2016 Highlights". Health Canada. 14 March 2017. Retrieved 7 April 2024.
2. "Ravicti- glycerol phenylbutyrate liquid". DailyMed. 13 September 2021. Archived from the original on 3 May 2023. Retrieved 4 July 2023.
3. "FDA approves new drug for the chronic management of some urea cycle disorders" (Press release). U.S. Food and Drug Administration. 1 February 2013. Archived from the original on 2013-03-07. Retrieved 2013-04-01.
4. "FDA Approved Drug Products: Ravicti". Drugs@FDA. U.S. Food and Drug Administration. Archived from the original on 29 June 2017. Retrieved 18 February 2018.
5. 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.
6. "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.