Pegvaliase

Pegvaliase, sold under the brand name Palynziq, is a medication for the treatment of the genetic disease phenylketonuria.[4][5] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.[6]

Pegvaliase
Clinical data
Pronunciationpeg val' i ase
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
AHFS/Drugs.comMonograph
MedlinePlusa618057
License data
Pregnancy
category
Routes of
administration		Subcutaneous
ATC code
Legal status
Legal status
Identifiers
IUPAC name
  • (2S)-2-amino-6-[6-(2-methoxyethoxy)hexanamido]hexanoic acid
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
Chemical and physical data
FormulaC15H30N2O5
Molar mass318.414 g·mol−1
3D model (JSmol)
SMILES
  • COCCOCCCCCC(=O)NCCCC[C@H](N)C(O)=O
InChI
  • InChI=1S/C15H30N2O5/c1-21-11-12-22-10-6-2-3-8-14(18)17-9-5-4-7-13(16)15(19)20/h13H,2-12,16H2,1H3,(H,17,18)(H,19,20)/t13-/m0/s1
  • Key:NPOCDVAOUKODSQ-ZDUSSCGKSA-N

It was approved by the Food and Drug Administration for use in the United States in 2018.[4] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]

References
  1. "Palynziq". Therapeutic Goods Administration (TGA). 23 July 2021. Archived from the original on 5 September 2021. Retrieved 5 September 2021.
  2. "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 12 May 2022. Archived from the original on 3 April 2022. Retrieved 13 May 2022.
  3. "Palynziq Product information". Health Canada. 25 April 2012. Archived from the original on 29 June 2022. Retrieved 29 June 2022.
  4. "FDA approves a new treatment for PKU, a rare and serious genetic disease" (Press release). Food and Drug Administration. May 24, 2018. Archived from the original on July 25, 2021. Retrieved October 12, 2020.
  5. Mahan KC, Gandhi MA, Anand S (April 2019). "Pegvaliase: a novel treatment option for adults with phenylketonuria". Current Medical Research and Opinion. 35 (4): 647–651. doi:10.1080/03007995.2018.1528215. PMID 30247930. S2CID 52813510.
  6. "Palynziq". BioMarin Pharmaceutica. Archived from the original on 2020-06-26. Retrieved 2018-06-21.
  7. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.
  • "Pegvaliase". Drug Information Portal. U.S. National Library of Medicine.


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