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pegvaliase (Palynziq)
Indications:
- phenylketonuria caused by phenylalanine hydroxylase deficiency
Dosage:
- 5-60 mg/day
Adverse effects:
- arthralgia (70%)
- injection-site reaction (62%)
- headache (47%)
- hypersensitivity (6%)
- 1/2 of individuals with hypersensitivity continue with treatmant
Mechanism of action:
- PEGylated recombinant Anabaena variabilis phenylalanine ammonia lyase
- converts Phe to trans-cinnamic acid & ammonia
- potential enzyme substitution therapy to lower blood Phe in adults with phenylketonuria
General
metabolic agent (metabolic modifier)
recombinant protein; chimer
References
- Thomas J, Levy H, Amato S
Pegvaliase for the treatment of phenylketonuria: Results of a
long-term phase 3 clinical trial program (PRISM).
Molecular Genetics & Metabolism, 124(1):27-38. May 2018
PMID: 29653686 Free full text
https://www.sciencedirect.com/science/article/pii/S1096719218300210