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{{Short description|Chemical compound}}
{{Use dmy dates|date=May 2024}}
{{cs1 config |name-list-style=vanc |display-authors=6}}
{{Infobox drug
{{Infobox drug
| drug_name =
| image =
⚫
| INN =
⚫
| type = <!-- empty -->
| image = Pegvaliase compact.png
| alt =
| alt =
| caption =
| caption =
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| DailyMedID = Pegvaliase
| DailyMedID = Pegvaliase
| pregnancy_AU = D
| pregnancy_AU = D
| pregnancy_AU_comment = <ref name="Palynziq APM summary" /><ref>{{cite web | title=Updates to the Prescribing Medicines in Pregnancy database | website=Therapeutic Goods Administration (TGA) | date=12 May 2022 | url=https://www.tga.gov.au/updates-prescribing-medicines-pregnancy-database | access-date=13 May 2022}}</ref>
| pregnancy_AU_comment = <ref name="Palynziq APM summary" /><ref>{{cite web | title=Updates to the Prescribing Medicines in Pregnancy database | website=Therapeutic Goods Administration (TGA) | date=12 May 2022 | url=https://www.tga.gov.au/resources/resource/guidance/updates-prescribing-medicines-pregnancy-database | access-date=13 May 2022 | archive-date=3 April 2022 | archive-url=https://web.archive.org/web/20220403064059/https://www.tga.gov.au/updates-prescribing-medicines-pregnancy-database | url-status=live }}</ref>
| pregnancy_category=
| pregnancy_category=
| routes_of_administration = [[Subcutaneous injection|Subcutaneous]]
| routes_of_administration = [[Subcutaneous injection|Subcutaneous]]
| class =
| class =
⚫
| ATCvet =
| ATC_prefix = A16
| ATC_prefix = A16
| ATC_suffix = AB19
| ATC_suffix = AB19
| ATC_supplemental =
| biosimilars =


<!-- Legal status -->
| legal_AU = S4
| legal_AU = S4
| legal_AU_comment = <ref name="Palynziq APM summary">{{cite web | title=Palynziq | website=Therapeutic Goods Administration (TGA) | date=23 July 2021 | url=https://www.tga.gov.au/apm-summary/palynziq | access-date=5 September 2021}}</ref>
| legal_AU_comment = <ref name="Palynziq APM summary">{{cite web | title=Palynziq | website=Therapeutic Goods Administration (TGA) | date=23 July 2021 | url=https://www.tga.gov.au/apm-summary/palynziq | access-date=5 September 2021 | archive-date=5 September 2021 | archive-url=https://web.archive.org/web/20210905213708/https://www.tga.gov.au/apm-summary/palynziq | url-status=live }}</ref>
| legal_BR = <!-- OTC, A1, A2, A3, B1, B2, C1, C2, C3, C4, C5, D1, D2, E, F-->
| legal_BR = <!-- OTC, A1, A2, A3, B1, B2, C1, C2, C3, C4, C5, D1, D2, E, F-->
| legal_BR_comment =
| legal_BR_comment =
| legal_CA = Rx-only
| legal_CA = Rx-only
| legal_CA_comment = /&nbsp;Schedule D<ref>{{cite web | title=Palynziq Product information | website=Health Canada | date=25 April 2012 | url=https://health-products.canada.ca/dpd-bdpp/info.do?lang=en&code=101536 | access-date=29 June 2022}}</ref>
| legal_CA_comment = /&nbsp;Schedule D<ref>{{cite web | title=Palynziq Product information | website=Health Canada | date=25 April 2012 | url=https://health-products.canada.ca/dpd-bdpp/info.do?lang=en&code=101536 | access-date=29 June 2022 | archive-date=29 June 2022 | archive-url=https://web.archive.org/web/20220629032328/https://health-products.canada.ca/dpd-bdpp/info.do?lang=en&code=101536 | url-status=live }}</ref>
| legal_DE = <!-- Anlage I, II, III -->
| legal_DE = <!-- Anlage I, II, III or Unscheduled -->
| legal_DE_comment =
| legal_NZ = <!-- Class A, B, C -->
| legal_UK = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM / Class A, B, C -->
| legal_NZ = <!-- Class A, B, C -->
| legal_NZ_comment =
| legal_UK = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM / Class A, B, C -->
| legal_UK_comment =
| legal_US = Rx-only
| legal_US = Rx-only
| legal_US_comment = <ref name="Palynziq FDA label">{{cite web | title=Palynziq- pegvaliase-pqpz injection, solution | website=DailyMed | date=21 December 2022 | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6dba844a-db02-44f8-8593-ce497ed9406c | access-date=12 December 2023}}</ref>
| legal_EU = Rx-only
| legal_EU = Rx-only
| legal_EU_comment = <ref name="Palynziq EPAR">{{cite web | title=Palynziq EPAR | website=European Medicines Agency | date=28 January 2010 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/palynziq | access-date=22 May 2024}}</ref>
| legal_UN = <!-- N I, II, III, IV / P I, II, III, IV-->
| legal_status = <!-- Free text -->
| legal_UN = <!-- N I, II, III, IV / P I, II, III, IV -->
| legal_UN_comment =
| legal_status = <!-- For countries not listed above -->


<!-- Pharmacokinetic data -->
<!-- Pharmacokinetic data -->
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<!-- Identifiers -->
<!-- Identifiers -->
| CAS_number = 1585984-95-7
| CAS_number = 1585984-95-7
| CAS_supplemental =
⚫
| PubChem = 86278362
⚫
| PubChem =
| DrugBank = DB12839
| DrugBank = DB12839
⚫
| ChemSpiderID =
| UNII = N6UAH27EUV
| UNII = N6UAH27EUV
| KEGG = D11077
| KEGG = D11077
⚫
| ChEBI =
⚫
| ChemSpiderID = 58172730
⚫
| ChEMBL =
| NIAID_ChemDB =
| PDB_ligand =
| synonyms = Pegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
| synonyms = Pegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
| SMILES = COCCOCCCCCC(=O)NCCCC[C@H](N)C(O)=O
| 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


<!-- Chemical and physical data -->
<!-- Chemical and physical data -->
| IUPAC_name = (2''S'')-2-amino-6-[6-(2-methoxyethoxy)hexanamido]hexanoic acid
| IUPAC_name =
| chemical_formula_ref = <ref>{{cite web | title=Pegvaliase | website=Inxight Drugs | url=https://drugs.ncats.io/substance/N6UAH27EUV | access-date=22 May 2024}}</ref><ref>{{cite web | title=Pegvaliase | website=GSRS | url=https://gsrs.ncats.nih.gov/ginas/app/beta/substances/N6UAH27EUV | access-date=22 May 2024}}</ref>
| C=15 | H=30 | N=2 | O=5
| C = 10872 | H = 17216 | O = 3300 | N = 3040 | S = 80
| SMILES =
| StdInChI =
| StdInChI_comment =
| StdInChIKey =
⚫
| density =
| density_notes =
| melting_point =
| melting_high =
| melting_notes =
| boiling_point =
| boiling_notes =
| solubility =
| sol_units =
| specific_rotation =
}}
}}


'''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 | s2cid = 52813510 }}</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>
'''Pegvaliase''', sold under the brand name '''Palynziq''', is a medication used for the treatment of the genetic disease [[phenylketonuria]].<ref name="Palynziq FDA label" /><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 | s2cid = 52813510 }}</ref> It is a phenylalanine (Phe)‑metabolizing enzyme.<ref name="Palynziq FDA label" /> Chemically, it is a [[pegylated]] [[derivative (chemistry)|derivative]] of the enzyme [[phenylalanine ammonia-lyase]] that metabolizes [[phenylalanine]] to reduce its blood levels.<ref name="Palynziq FDA label" />


The most common adverse events include injection site reactions, joint pain, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, pruritus (itchy skin), nausea, dizziness, abdominal pain, throat pain, fatigue, vomiting, cough and, diarrhea.<ref name=FDA2018Sub />
⚫
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 US [[Food and Drug Administration]] (FDA) 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 = U.S. [[Food and Drug Administration]] (FDA) | date = 24 May 2018 | access-date = 12 October 2020 | archive-date = 25 July 2021 | archive-url = https://web.archive.org/web/20210725044549/https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pku-rare-and-serious-genetic-disease | url-status = live }} {{PD-notice}}</ref> The 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 | archive-date=17 September 2020 | archive-url=https://web.archive.org/web/20200917055627/https://www.fda.gov/media/120357/download | url-status=live }}</ref>

== Medical uses ==
Pegvaliase is [[indicated]] to reduce blood Phe concentrations in adults with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.<ref name="Palynziq FDA label" />

== Adverse effects ==
The FDA label for pegvaliase includes a [[boxed warning]] for anaphylaxis.

== History ==
The safety and efficacy of pegvaliase were studied in two clinical trials in adult participants with PKU with blood phenylalanine concentrations greater than 600 μmol/L on existing management.<ref name=FDA2018Sub /> Most PKU participants in the pegvaliase trials were on an unrestricted diet prior to and during the trials.<ref name=FDA2018Sub /> The first trial was a randomized, open-label trial in participants treated with increasing doses of pegvaliase administered as a subcutaneous injection up to a target dose of either 20 mg once daily or 40 mg once daily.<ref name=FDA2018Sub /> The second trial was an 8-week, placebo-controlled, randomized withdrawal trial in participants who were previously treated with pegvaliase.<ref name=FDA2018Sub /> Participants treated with pegvaliase achieved statistically significant reductions in blood phenylalanine concentrations from their pre-treatment baseline blood Phe concentrations.<ref name=FDA2018Sub />

The FDA granted approval of Palynziq to BioMarin Pharmaceutical.<ref name=FDA2018Sub />


==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}}
{{Portal bar | Medicine}}
{{Authority control}}


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


{{gastrointestinal-drug-stub}}

Latest revision as of 03:53, 22 May 2024

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
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC10872H17216N3040O3300S80[7][8]
Molar mass245880.10 g·mol−1

Pegvaliase, sold under the brand name Palynziq, is a medication used for the treatment of the genetic disease phenylketonuria.[5][9][10] It is a phenylalanine (Phe)‑metabolizing enzyme.[5] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.[5]

The most common adverse events include injection site reactions, joint pain, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, pruritus (itchy skin), nausea, dizziness, abdominal pain, throat pain, fatigue, vomiting, cough and, diarrhea.[9]

It was approved by the US Food and Drug Administration (FDA) for use in the United States in 2018.[9] The FDA considers it to be a first-in-class medication.[11]

Medical uses

[edit]

Pegvaliase is indicated to reduce blood Phe concentrations in adults with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.[5]

Adverse effects

[edit]

The FDA label for pegvaliase includes a boxed warning for anaphylaxis.

History

[edit]

The safety and efficacy of pegvaliase were studied in two clinical trials in adult participants with PKU with blood phenylalanine concentrations greater than 600 μmol/L on existing management.[9] Most PKU participants in the pegvaliase trials were on an unrestricted diet prior to and during the trials.[9] The first trial was a randomized, open-label trial in participants treated with increasing doses of pegvaliase administered as a subcutaneous injection up to a target dose of either 20 mg once daily or 40 mg once daily.[9] The second trial was an 8-week, placebo-controlled, randomized withdrawal trial in participants who were previously treated with pegvaliase.[9] Participants treated with pegvaliase achieved statistically significant reductions in blood phenylalanine concentrations from their pre-treatment baseline blood Phe concentrations.[9]

The FDA granted approval of Palynziq to BioMarin Pharmaceutical.[9]

References

[edit]
  1. ^ a b "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. ^ "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 October 2023.
  4. ^ "Palynziq Product information". Health Canada. 25 April 2012. Archived from the original on 29 June 2022. Retrieved 29 June 2022.
  5. ^ a b c d e "Palynziq- pegvaliase-pqpz injection, solution". DailyMed. 21 December 2022. Retrieved 12 December 2023.
  6. ^ "Palynziq EPAR". European Medicines Agency. 28 January 2010. Retrieved 22 May 2024.
  7. ^ "Pegvaliase". Inxight Drugs. Retrieved 22 May 2024.
  8. ^ "Pegvaliase". GSRS. Retrieved 22 May 2024.
  9. ^ a b c d e f g h i "FDA approves a new treatment for PKU, a rare and serious genetic disease" (Press release). U.S. Food and Drug Administration (FDA). 24 May 2018. Archived from the original on 25 July 2021. Retrieved 12 October 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  10. ^ 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.
  11. ^ 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.
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