BioMarin Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria (PKU).

PKU is a rare, inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine. Untreated phenylketonuria can lead to brain damage, intellectual disabilities, behavioral symptoms or seizures.

The primary endpoints are the changes in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include changes in total dietary protein intake ...

https://www.benzinga.com/25/04/44613863/biomarin-seeks-label-expansion-for-palynziq-after-positive-phase-3-results-in-adolescent-patients