BioMarin presents studies showing efficacy of Voxzogo, Palynziq

BioMarin (BMRN) announced positive new data from studies of Voxzogo in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as Palynziq in adults with phenylketonuria. The findings validate the long-term benefit and established safety profiles of both medicines. The data will be presented at the 2025 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting in Los Angeles. New data from a study in Japan showed strong treatment adherence with Voxzogo in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. New data from the OPAL study, a post-marketing observational trial to assess the real-world safety and efficacy of Palynziq, showed lowered blood Phe levels following treatment and positive health-related quality of life outcomes. Mean blood Phe level was 1029 mumol/L at baseline and lowered to 293 mumol/L at week 96, representing a 67.8% reduction. Additionally, secondary data analyses from the Phase 3 PRISM clinical trial program demonstrated that sustaining lowered blood Phe levels with Palynziq led to improvements in attention and mood. Results from a Phase 3 study of Palynziq in adolescents in PKU is expected later this year.