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'''Pegvaliase''', sold under the brand name '''Palynziq''', is a medication for the treatment of the genetic disease [[phenylketonuria]].<ref name=FDA2018Sub/> Chemically, it is a [[pegylated]] [[derivative (chemistry)|derivative]] of the enzyme [[phenylalanine ammonia-lyase]] that metabolizes [[phenylalanine]] to reduce its blood levels.<ref>{{Cite web | url = https://www.palynziq.com/hcp/ | title = Palynziq | publisher = BioMarin Pharmaceutica}}</ref>
'''Pegvaliase''', sold under the brand name '''Palynziq''', is a medication for the treatment of the genetic disease [[phenylketonuria]].<ref name=FDA2018Sub/><ref name="pmid30247930">{{cite journal | vauthors = Mahan KC, Gandhi MA, Anand S | title = Pegvaliase: a novel treatment option for adults with phenylketonuria | journal = Current Medical Research and Opinion | volume = 35 | issue = 4 | pages = 647–651 | date = April 2019 | pmid = 30247930 | doi = 10.1080/03007995.2018.1528215 }}</ref> Chemically, it is a [[pegylated]] [[derivative (chemistry)|derivative]] of the enzyme [[phenylalanine ammonia-lyase]] that metabolizes [[phenylalanine]] to reduce its blood levels.<ref>{{Cite web | url = https://www.palynziq.com/hcp/ | title = Palynziq | publisher = BioMarin Pharmaceutica}}</ref>


It was approved by the [[Food and Drug Administration]] for use in the United States in 2018.<ref name=FDA2018Sub>{{cite press release | url = https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pku-rare-and-serious-genetic-disease | title = FDA approves a new treatment for PKU, a rare and serious genetic disease | publisher = [[Food and Drug Administration]] | date = May 24, 2018}}</ref> The U.S. [[Food and Drug Administration]] (FDA) considers it to be a [[first-in-class medication]].<ref>{{cite report | title=New Drug Therapy Approvals 2018 | website=U.S. [[Food and Drug Administration]] (FDA) | date=January 2019 | url=https://www.fda.gov/media/120357/download | format=PDF | access-date=16 September 2020}}</ref>
It was approved by the [[Food and Drug Administration]] for use in the United States in 2018.<ref name=FDA2018Sub>{{cite press release | url = https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pku-rare-and-serious-genetic-disease | title = FDA approves a new treatment for PKU, a rare and serious genetic disease | publisher = [[Food and Drug Administration]] | date = May 24, 2018}}</ref> The U.S. [[Food and Drug Administration]] (FDA) considers it to be a [[first-in-class medication]].<ref>{{cite report | title=New Drug Therapy Approvals 2018 | website=U.S. [[Food and Drug Administration]] (FDA) | date=January 2019 | url=https://www.fda.gov/media/120357/download | format=PDF | access-date=16 September 2020}}</ref>

Revision as of 04:30, 3 July 2021

Pegvaliase
Clinical data
Pronunciationpeg val' i ase
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
AHFS/Drugs.comMonograph
MedlinePlusa618057
License data
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.[2][3] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.[4]

It was approved by the Food and Drug Administration for use in the United States in 2018.[2] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]

References

  1. ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 Oct 2023.
  2. ^ a b "FDA approves a new treatment for PKU, a rare and serious genetic disease" (Press release). Food and Drug Administration. May 24, 2018.
  3. ^ 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.
  4. ^ "Palynziq". BioMarin Pharmaceutica.
  5. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
  • "Pegvaliase". Drug Information Portal. U.S. National Library of Medicine.


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