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pegvaliase
- Development stage
- Approved
- Lead developer
- BioMarin
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, PEGylated Peptides → Modified Peptides → Peptides
- Administration
- Subcutaneous
01
Overview
Pegvaliase is a PEGylated recombinant phenylalanine ammonia lyase (PAL) enzyme used as an enzyme substitution therapy for adults with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations despite current management. It works by converting excess phenylalanine into ammonia and trans-cinnamic acid, thereby reducing toxic levels of phenylalanine in the blood. Pegvaliase is administered as a subcutaneous injection and has demonstrated significant reductions in blood phenylalanine levels and improvements in neuropsychiatric symptoms in clinical trials. The drug was developed and is marketed under the brand name Palynziq[1][2][5][6][8].
- Brand names
- Palynziq
- Other names
- pegvaliase-pqpzPEG-PALRAvPAL-PEG
02
Targets
PAL (Phenylalanine ammonia-lyase)
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