pegvaliase (Palynziq)
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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
More general terms
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: https://www.ncbi.nlm.nih.gov/pubmed/29653686 Free full text https://www.sciencedirect.com/science/article/pii/S1096719218300210