Posted by Michael Wonder on 30 Oct 2025
FDA accepts BioMarin’s Palynziq (pegvaliase-pqpz) supplemental biologics license application for priority review to expand use to adolescents aged 12-17 with phenylketonuria
29 October 2025 - Application based on positive results from Phase 3 PEGASUS study, which demonstrated statistically significant blood phenylalanine lowering compared to diet alone.
BioMarin today announced that the US FDA has accepted for priority review the company's Palynziq (pegvaliase-pqpz) supplemental biologics license application to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU).
The FDA has set a PDUFA target action date of 28 February 2026.
