Also known as PAH deficiency, phenylketonuria is a genetic condition. Credit: bangoland / Shutterstock.
BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its primary efficacy endpoint, showcasing a reduction in blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU) against only diet.
The adolescent individuals in the trial are aged between 12 and 17 years.
The open-label, randomised controlled trial is assessing the efficacy and safety of therapy against dietary management alone in 55 PKU adolescents of this age group.
Its primary endpoints were the change in blood Phe concentration and the characterisation of the safety profile.
Secondary endpoints of the trial included changes in the intake of total dietary protein and pharmacokinetics. The therapy’s safety outcomes were found to be consistent with its known profile.
According to BioMarin Pharmaceutical, the trial is being carried out in two parts.
Part one involves the primary treatment phase from weeks one to 73 while part two comprises an extension phase, involving up to 80 more weeks of monitoring for the therapy group and provides the option for participants in the diet-only group to cross over.
Later this year, the company plans to submit detailed trial outcomes to global health authorities, aiming for a label expansion that would include adolescent patients.
Palynziq functions by substituting the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with phenylalanine ammonia lyase’s PEGylated version, which helps break down Phe.
The treatment involves a dosing regimen tailored for tolerability, and its safety profile is primarily characterised by immune-mediated responses, including anaphylaxis.
BioMarin chief research and development officer and executive vice-president Greg Friberg said: “We are encouraged to see these positive data that build on that legacy and show how Palynziq can make an impact for adolescents as they begin their transition to adult living.”
Also known as PAH deficiency, PKU is a genetic condition where insufficient PAH enzyme levels lead to Phe buildup in the blood, causing various complications.
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