BMRN News & Events

BioMarin announced its decision to discontinue dosing and enrollment in its Phase 2 trials for Voxzogo in Turner syndrome, SHOX-deficiency and aggrecan-deficiency following the occurrence of several slipped capital femoral epiphysis events in two ongoing investigator-sponsored trials. "SCFE events have not been observed in the Phase 2 BioMarin trials in these same conditions, nor have any cases been observed in the more than 5,000 infants and children who have received Voxzogo for achondroplasia, which includes 10 years of clinical research and more than 10,000 patient-years of safety data in clinical studies and post-marketing surveillance," the company added. There have also been no observed cases in BioMarin's clinical trials for hypochondroplasia. The Phase 2 CANOPY trials of Voxzogo in children with Noonan syndrome, as well as those living with idiopathic short stature without ACAN-deficiency, which represents approximately 95% of children enrolled in the ISS trial, will continue as planned, the company added. Shares of BioMarin are down 3% to $56.91 in premarket trading.

BioMarin announced positive new data from studies of Voxzogo - vosoritide - in children with achondroplasia in ongoing clinical trials and real-world studies. The latest findings reinforce the importance of early treatment and the established efficacy and safety of Voxzogo. The data will be presented at the 2026 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting in Baltimore. New data will be presented from a study of children who started Voxzogo before age 2, which showed that treatment was associated with durable, multi-year growth improvements, including body proportionality and arm span compared to untreated children, as well as improvements in height over time. Additional analyses further emphasize that these children showed durable improvements in height while maintaining stable arm-to-height ratios, consistent with arm span improvements compared to untreated children. Researchers also saw significant improvements in body mass index in children who initiated treatment when compared to untreated children. At ACMG, researchers will present findings on real-world evidence highlighting the impact of Voxzogo in children who were treated in Japan, Europe and the U.S.