BioMarin Pharmaceutical Inc. has announced new data characterising the efficacy and safety of PALYNZIQ (pegvaliase-pqpz) for the treatment of adolescents with phenylketonuria (PKU).
The announcement was made at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan.
The Phase 3 PEGASUS study evaluating the efficacy and safety of PALYNZIQ in adolescents aged 12 to 17 demonstrated statistically significant blood phenylalanine (Phe) lowering compared to diet alone. The study enrolled 55 adolescents, randomized to receive PALYNZIQ or diet alone. At baseline, the mean age was 14.3 years, mean blood Phe was 1026.4 µmol/L, and 49.1% of participants had blood Phe levels above 1000 µmol/L. After the 72-week primary treatment phase, 45.2% of participants in the PALYNZIQ arm achieved reductions in blood Phe concentrations of 50% or more from baseline, with many reaching guideline-recommended and even normal Phe target levels.
The safety profile and overall efficacy trends in adolescents were consistent with the known profile of the medicine in adults. The vast majority of adverse events (AEs) in the study were manageable, with 5.6% representing serious AEs (anaphylaxis) leading to study discontinuation. The ongoing extension phase of the PEGASUS trial will continue to evaluate long-term results of PALYNZIQ treatment for adolescents.
“These data from the PEGASUS study underscore the impact PALYNZIQ can have in enabling adolescents to experience more freedom from the burden of their condition, particularly during this pivotal period of transition to adulthood,” said Greg Friberg, executive vice president and chief research & development officer at BioMarin.
“BioMarin has been deeply committed to advancing scientific progress for people living with PKU over the past two decades, and we look forward to sharing these results with global regulators with the aim of bringing the unequalled efficacy observed with PALYNZIQ to an even younger group of people.”
PALYNZIQ is the first and only enzyme substitution therapy approved to treat adults with PKU. The company is on track with its planned submission of the PEGASUS study data to global health authorities to expand the approved indication for PALYNZIQ to include the treatment of adolescents.
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