BioMarin Halts Voxzogo Trials After Safety Concerns

BioMarin has paused dosing and enrollment in Phase II trials of Voxzogo (vosoritide) for Turner Syndrome, SHOX-deficiency, and ACAN-deficiency following safety concerns. The decision came after cases of slipped capital femoral epiphysis (SCFE) were reported in two investigator-led studies.

SCFE is a condition where the head of the femur slips from the growth plate, potentially leading to serious complications. BioMarin noted that such events have not been observed in other Voxzogo trials or among over 5,000 patients treated for achondroplasia.

Ongoing trials in other conditions, including Noonan syndrome and idiopathic short stature, will continue as planned. Voxzogo remains approved by the U.S. Food and Drug Administration for achondroplasia and continues to perform strongly commercially.

However, competition is increasing, with new therapies emerging, including options with more convenient dosing schedules, potentially impacting Voxzogo’s future market position.

22-03-2026