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
  • (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)
  • COCCOCCCCCC(=O)NCCCC[C@H](N)C(O)=O
  • 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

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]

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. 1 2 "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. 1 2 "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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