A Comprehensive Review of Pegvaliase, an Enzyme Substitution Therapy for the Treatment of Phenylketonuria
UMass Chan Affiliations
Commonwealth MedicineClinical Pharmacy Services
Department of Family Medicine and Community Health
Document Type
Journal ArticlePublication Date
2019-06-21Keywords
PKUPalynziq
pegvaliase
pegvaliase-pqpz
phenylalanine ammonia lyase
phenylketonuria
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Enzymes and Coenzymes
Medicinal and Pharmaceutical Chemistry
Nervous System Diseases
Nutritional and Metabolic Diseases
Therapeutics
Metadata
Show full item recordAbstract
Objective: To review the pharmacology, pharmacokinetics, efficacy, safety, and place in therapy of a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations, pegvaliase injection. Data Sources: Searches of MEDLINE (1946-September 1, 2018) were conducted using the terms pegvaliase and phenylalanine ammonia lyase (PAL). Additional data were obtained from the prescribing information, the product dossier obtained from the manufacturer, and Clinicaltrials.gov. Study Selection and Data Extraction: All English language articles related to pharmacology, pharmacokinetics, efficacy, or safety of the combination therapy in human subjects were reviewed. Data Synthesis: Pegvaliase is a pegylated PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. Blood phenylalanine levels were reduced by approximately 50% to 70% in patients receiving therapeutic doses of pegvaliase. However, most patients experienced adverse events. Conclusions and Relevance: The mainstay of therapy in phenylketonuria (PKU) has historically consisted of dietary restriction of phenylalanine. Pegvaliase injection is the first Food and Drug Administration (FDA)-approved enzyme substitution therapy for patients with PKU. The therapy may be a viable option for patients with documented blood phenylalanine > 600 micromol/L who have failed existing management strategies.Source
Drug Target Insights. 2019 Jun 21;13:1177392819857089. doi: 10.1177/1177392819857089. eCollection 2019. Link to article on publisher's site
DOI
10.1177/1177392819857089Permanent Link to this Item
http://hdl.handle.net/20.500.14038/41086PubMed ID
31258325Related Resources
Rights
Copyright © The Author(s) 2019. This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (http://www.creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (http://www.uk.sagepub.com/aboutus/openaccess.htm).Distribution License
http://creativecommons.org/licenses/by-nc/4.0/ae974a485f413a2113503eed53cd6c53
10.1177/1177392819857089
Scopus Count
Except where otherwise noted, this item's license is described as Copyright © The Author(s) 2019. This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (http://www.creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (http://www.uk.sagepub.com/aboutus/openaccess.htm).