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]
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]
^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. PMID30247930. S2CID52813510.