BioMarin discontinues VOXZOGO trials in Turner, SHOX, ACAN after SCFE safety events

BIOMARIN PHARMACEUTICAL INC

Discontinued dosing and enrollment in Phase 2 CANOPY trials for Turner syndrome, SHOX-deficiency, and ACAN-deficiency.
Decision follows slipped capital femoral epiphysis (SCFE) events in two investigator-sponsored trials; SCFE not seen in BioMarin's own trials or in >5,000 achondroplasia patients.
Phase 2 CANOPY trials for Noonan syndrome and idiopathic short stature (ISS) without ACAN-deficiency will continue as planned.
Over 10,000 patient-years of safety data in achondroplasia shows no SCFE cases; VOXZOGO remains approved for achondroplasia.

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.