- Full-year 2020 Total Revenues Increased 9% to $1.86 billion
- Company Dose-escalates in PHEarless, a Phase 1/2 Study with BMN 307 Gene Therapy for the Treatment of Phenylketonuria (PKU); the Third Potential Therapeutic Modality in its PKU Franchise
- During the Fourth Quarter, BioMarin Announced Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study
- Marketing Applications for Vosoritide to Treat Children with Achondroplasia under Review in Europe and the U.S.; Committee for Medicinal Products for Human Use (CHMP) Opinion Expected in June 2021
- In January 2021, BioMarin Announced Positive Phase 3 Results with Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set; Mean Annualized Bleed Rate was 0.9 in Subset Dosed More than Two Years Ago