BioMarin Pharmaceutical Inc. has stopped enrollment and dosing in its Phase 2 studies of Voxzogo (vosoritide) for Turner syndrome, SHOX‑deficiency and aggrecan (ACAN) deficiency after several patients experienced slipped capital femoral epiphysis (SCFE), a serious hip joint dislocation.
The company noted that SCFE events were observed in the investigator‑sponsored trials for these indications, but no such events have been reported in its own Phase 2 studies for achondroplasia or in the >5,000 children treated for achondroplasia, which represents more than 10,000 patient‑years of safety data.
By discontinuing these trials, BioMarin removes a potential future revenue stream and signals a setback in its strategy to expand Voxzogo beyond achondroplasia. Other ongoing studies, such as the CANOPY trials for Noonan syndrome and idiopathic short stature, will continue as planned.
Market reaction to the announcement was negative, with the company’s shares falling 4.6% in pre‑market trading before stabilizing near $58.01 at open, reflecting investor concern over the safety signal and its impact on the drug’s label expansion.
While BioMarin has not issued a direct statement on the long‑term implications, the company’s decision underscores the importance of safety data in rare‑disease development and may prompt a reassessment of its pipeline priorities.
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