BioMarin Pharmaceutical Inc. announced discontinuation of dosing and enrollment in Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency, and Aggrecan (ACAN)-deficiency due to slipped capital femoral epiphysis (SCFE) events in investigator-sponsored trials. Phase 2 CANOPY trials in Noonan syndrome and idiopathic short stature (ISS) without ACAN-deficiency will continue as planned.
Key takeaways
Discontinued dosing and enrollment in Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency, and ACAN-deficiency.
Decision follows several SCFE events in two ongoing investigator-sponsored trials.
No SCFE events observed in BioMarin's Phase 2 trials for these conditions.
No SCFE cases in more than 5,000 infants and children treated with VOXZOGO for achondroplasia, including 10 years of clinical research and over 10,000 patient-years of safety data.
No SCFE cases in BioMarin's hypochondroplasia trials.
Phase 2 CANOPY trials continue in Noonan syndrome and ISS without ACAN-deficiency, representing approximately 95% of ISS trial enrollees.