BioMarin discontinues enrollment in some Phase 2 Voxzogo trials

BioMarin (BMRN) 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.