Glycerol phenylbutyrate

Chemical compound

Pharmaceutical compound

Glycerol phenylbutyrate

Clinical data
Trade names| Ravicti
AHFS/Drugs.com| Micromedex Detailed Consumer Information
License data|

* US DailyMed: Glycerol phenylbutyrate

Routes of
administration| By mouth
ATC code|

* A16AX09 (WHO)

Legal status
Legal status|

* CA: ℞-only[1]
* US: ℞-only[2]
* EU: Rx-only[3]

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

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]

## Medical uses

[edit]

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

[edit]

### Economics

[edit]

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

[edit]

1. ^ "Health Canada New Drug Authorizations: 2016 Highlights". Health Canada. 14 March 2017. Retrieved 7 April 2024.
2. ^ a b "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. Archived from the original on 1 August 2016. 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.

* v
* t
* e

Other alimentary tract and metabolism products (A16)

Amino acids and derivatives|

* Ademetionine
* Betaine
* Carglumic acid
* Glutamine
* Levocarnitine
* Mercaptamine
* Metreleptin

Enzymes|

* Amino acids: phenylalanine ammonia-lyase (Pegvaliase)

* Carbohydrate metabolism: sucrase (Sacrosidase)
* alpha-glucosidase (Alglucosidase alfa, Avalglucosidase alfa, Cipaglucosidase alfa)

* Glycolipid/sphingolipid: glucocerebrosidase (Alglucerase
* Imiglucerase
* Taliglucerase alfa
* Velaglucerase alfa)
* alpha-galactosidase (Agalsidase alfa
* Agalsidase beta
* Pegunigalsidase alfa)

* Glycosaminoglycan: iduronidase (Laronidase)
* arylsulfatase B (Galsulfase)
* iduronate-2-sulfatase (Idursulfase)
* idursulfase beta

* Lipid: lysosomal lipase (Sebelipase alfa)

* Phosphate: Asfotase alfa

* atidarsagene autotemcel
* cerliponase alfa
* eladocagene exuparvovec
* elosulfase alfa
* olipudase alfa
* pabinafusp alfa
* pegzilarginase
* vestronidase alfa

Other|

* Anethole trithione
* Eliglustat
* Givosiran
* Glycerol phenylbutyrate
* Leriglitazone
* Lumasiran
* Miglustat
* Nedosiran
* Nitisinone
* Sapropterin
* Sepiapterin
* Sodium benzoate (+sodium phenylacetate)
* Sodium phenylbutyrate
* Teduglutide
* Trientine
* Tioctic acid
* Triheptanoin
* Uridine triacetate
* Velmanase alfa
* Zinc acetate

Portal:

* Medicine

*[US]: United States
*[CA]: Canada
*[EU]: European Union
*[CID]: Compound ID
*[EPA]: U.S. Environmental Protection Agency
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