WS Investor
16 Mar 2026, 16:04
BioMarin Pharmaceutical said it will discontinue dosing and enrollment in Phase 2 trials of VOXZOGO for Turner syndrome, SHOX-deficiency and Aggrecan (ACAN) deficiency after several slipped capital femoral epiphysis (SCFE) events were reported in two investigator-sponsored studies.
The company noted that SCFE events were not observed in BioMarin’s own Phase 2 trials for these conditions, nor among more than 5,000 infants and children treated with VOXZOGO for achondroplasia across roughly 10,000 patient-years of safety data. No cases have also been reported in BioMarin’s clinical trials for hypochondroplasia.
Other VOXZOGO studies will continue as planned, including Phase 2 CANOPY trials in children with Noonan syndrome and in those with idiopathic short stature without ACAN deficiency, which accounts for about 95% of participants in the idiopathic short stature study.
The company noted that SCFE events were not observed in BioMarin’s own Phase 2 trials for these conditions, nor among more than 5,000 infants and children treated with VOXZOGO for achondroplasia across roughly 10,000 patient-years of safety data. No cases have also been reported in BioMarin’s clinical trials for hypochondroplasia.
Other VOXZOGO studies will continue as planned, including Phase 2 CANOPY trials in children with Noonan syndrome and in those with idiopathic short stature without ACAN deficiency, which accounts for about 95% of participants in the idiopathic short stature study.