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'''Pegvaliase''' (trade 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''' (trade 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>


It was approved by the [[Food and Drug Administration]] for use in the United States in 2018.<ref name=FDA2018Sub>{{cite web | url = https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm608835.htm | title = FDA approves a new treatment for PKU, a rare and serious genetic disease | publisher = [[Food and Drug Administration]] | date = May 24, 2018}}</ref>
It was approved by the [[Food and Drug Administration]] for use in the United States in 2018.<ref name=FDA2018Sub>{{cite web | url = https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm608835.htm | 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>


==References==
==References==
{{reflist}}
{{reflist}}

== External links ==
* {{cite web | url = https://druginfo.nlm.nih.gov/drugportal/name/pegvaliase | publisher = U.S. National Library of Medicine | work = Drug Information Portal | title = Pegvaliase }}


{{Other alimentary tract and metabolism products}}
{{Other alimentary tract and metabolism products}}
{{Portal bar | Medicine}}


[[Category:Drugs acting on the gastrointestinal system and metabolism]]
[[Category:Drugs acting on the gastrointestinal system and metabolism]]
[[Category:Recombinant proteins]]
[[Category:Recombinant proteins]]



{{gastrointestinal-drug-stub}}
{{gastrointestinal-drug-stub}}

Revision as of 06:42, 17 September 2020

Pegvaliase
Clinical data
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
Routes of
administration
Subcutaneous injection
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
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 (trade name Palynziq) is a medication for the treatment of the genetic disease phenylketonuria.[2] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.[3]

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.[4]

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". Food and Drug Administration. May 24, 2018.
  3. ^ "Palynziq". BioMarin Pharmaceutica.
  4. ^ 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.