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Pegvaliase: Difference between revisions

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| drug_name =
| drug_name =
| INN =
| INN =
| type =<!-- empty -->
| type = <!-- empty -->
| IUPAC_name = (2S)-2-amino-6-[6-(2-methoxyethoxy)hexanamido]hexanoic acid
| image =
| image =
| alt =
| alt =
| caption =
| caption =

<!-- Clinical data -->
<!-- Clinical data -->
| pronounce =
| pronounce = peg val' i ase
| tradename = Palynziq
| tradename = Palynziq
| Drugs.com = {{drugs.com|monograph|pegvaliase-pqpz}}
| Drugs.com = {{drugs.com|monograph|pegvaliase-pqpz}}
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| legal_UN = <!-- N I, II, III, IV / P I, II, III, IV-->
| legal_UN = <!-- N I, II, III, IV / P I, II, III, IV-->
| legal_status = <!-- Free text -->
| legal_status = <!-- Free text -->

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| CAS_number = 1585984-95-7
| CAS_number = 1585984-95-7
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| StdInChI=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
| StdInChI=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
| StdInChIKey = NPOCDVAOUKODSQ-ZDUSSCGKSA-N
| StdInChIKey = NPOCDVAOUKODSQ-ZDUSSCGKSA-N

<!-- Chemical and physical data -->
<!-- Chemical and physical data -->
| IUPAC_name = (2S)-2-amino-6-[6-(2-methoxyethoxy)hexanamido]hexanoic acid
| chemical_formula =
| chemical_formula =
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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''', 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>


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>
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>


==References==
==References==

Revision as of 07:54, 12 October 2020

Pegvaliase
Clinical data
Pronunciationpeg val' i ase
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
AHFS/Drugs.comMonograph
MedlinePlusa618057
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, sold under the brand 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" (Press release). 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.