BioMarin Announces Positive Phase 3 Data for PALYNZIQ in Adolescent PKU Treatment
AI-Generated Summary
BioMarin Pharmaceutical announced new Phase 3 data for PALYNZIQ, an enzyme replacement therapy for phenylketonuria (PKU) in adolescents. The PEGASUS study demonstrated statistically significant reductions in blood phenylalanine (Phe) levels compared to diet alone. Nearly half of the participants treated with PALYNZIQ achieved a 50% or more reduction from baseline, with many reaching guideline-recommended Phe target levels.
In a nutshell
This announcement offers a promising development for adolescents with PKU, a rare genetic disorder, by showing PALYNZIQ's significant efficacy in managing blood Phe levels. It underscores continued progress in developing therapeutic options for challenging rare diseases.
Source: Markets Insider
